65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCT2031210309 | 20/12/2022 | 13/09/2021 | RALPID | Phase 2 study of the efficacy and safety of sirolumus in patients with primary immunodeficiency | Primary immunodeficiency PID, ALPS, APDS, CTLA4, IPEX, lymphoproliferative disease, enterocolitis, cytopenia;D000081207 | Administer sirolimus tablets or granules once a daily. Initial dose: 2mg (tablet), 0.049 mg/kg or 0.035 mg/kg (granule) | Imai Kohsuke | NULL | Recruiting | >= 2month old | Not applicable | Both | 10 | Phase 2 | Japan |
| 2 | NCT05189925 (ClinicalTrials.gov) | March 24, 2022 | 12/1/2022 | NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) | NADPH Oxidase Correction in mRNA Transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) | Chronic Granulomatous Disease;Infection | Biological: gp91 Grans | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 18 Years | 75 Years | Male | 25 | Phase 1 | United States |
| 3 | NCT05104723 (ClinicalTrials.gov) | March 24, 2022 | 2/11/2021 | Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications | A Phase 1/2 Open-label Study to Evaluate the Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications | Chronic Granulomatous Disease;Inflammatory Gastrointestinal Disease;Inflammatory Skin Disease;Inflammatory Lung Disease | Drug: XELJANZ (tofacitinib) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States |
| 4 | NCT04370795 (ClinicalTrials.gov) | March 24, 2022 | 30/4/2020 | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Severe Combined Immunodeficiency (SCID) | Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1/Phase 2 | United States |
| 5 | EUCTR2021-000227-13-BG (EUCTR) | 07/03/2022 | 02/12/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom | ||
| 6 | NCT05259917 (ClinicalTrials.gov) | March 1, 2022 | 4/2/2022 | A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE) | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: Placebo;Drug: KVD900 600 mg;Drug: KVD900 300 mg | KalVista Pharmaceuticals, Ltd. | NULL | Not yet recruiting | 12 Years | N/A | All | 114 | Phase 3 | NULL |
| 7 | NCT05070455 (ClinicalTrials.gov) | March 1, 2022 | 23/9/2021 | An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD) | An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immune Deficiency | Biological: Asceniv™ | ADMA Biologics, Inc. | NULL | Not yet recruiting | 2 Years | 11 Years | All | 12 | Phase 4 | NULL |
| 8 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 9 | NCT05047185 (ClinicalTrials.gov) | March 2022 | 7/9/2021 | Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II) | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor Deficiency;C1 Inhibitor Deficiency | Drug: PHA-022121;Drug: Placebo | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | 75 Years | All | 30 | Phase 2 | United States;Canada;United Kingdom |
| 10 | EUCTR2021-003327-15-NL (EUCTR) | 21/02/2022 | 19/01/2022 | Thymosin-a1 in immunodeficiency associated mood disorders | An open-label, 8-week, proof of concept trial on thymosin-a1 (thymalfasin) in the treatment of primary antibody deficiency (PAD) associated mood disorders (TIDAM18). - TIDAM | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Thymalfasin Product Name: Thymafalsin INN or Proposed INN: THYMALFASIN Other descriptive name: Zadaxin | Erasmus MC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Netherlands | ||
| 11 | EUCTR2021-000227-13-PL (EUCTR) | 02/02/2022 | 18/11/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom | ||
| 12 | NCT04513002 (ClinicalTrials.gov) | February 2022 | 26/7/2020 | Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis | A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation | Ataxia Telangiectasia | Dietary Supplement: Triheptanoin | The University of Queensland | National Health and Medical Research Council, Australia | Not yet recruiting | N/A | N/A | All | 30 | Phase 2 | Australia |
| 13 | JPRN-jRCT2031210457 | 24/01/2022 | 27/11/2021 | A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID) | A Phase 3, Open-label, Non-controlled Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of TAK-771 in Japanese Subjects with Primary Immunodeficiency Diseases (PID) | Primary Immunodeficiency Diseases (PID) | Epoch 1: TAK-771 Ramp up Period TAK-771 includes Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants will receive SC infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI will be increased from 1/3 of full dose to full dose in 3 weeks for participants who will receive TAK-771 once every 3 week, or from 1/4 of full dose to full dose in 6 weeks for participants who will receive TAK-771 once every 4 week. Epoch 2: TAK-771 Treatment Period TAK-771 includes Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants will receive subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 3, or 4 weeks for up to Week 24. | Nishizawa Atsushi | NULL | Recruiting | >= 2age old | Not applicable | Both | 15 | Phase 3 | Japan |
| 14 | NCT05150340 (ClinicalTrials.gov) | January 24, 2022 | 24/11/2021 | A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID) | A Phase 3, Open-label, Non-controlled Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of TAK-771 in Japanese Subjects With Primary Immunodeficiency Diseases (PID) | Primary Immunodeficiency Diseases (PID) | Drug: TAK-771 | Takeda | NULL | Recruiting | 2 Years | N/A | All | 15 | Phase 3 | Japan |
| 15 | NCT05256017 (ClinicalTrials.gov) | December 30, 2021 | 17/1/2022 | Safety and Tolerability Study of Acoziborole in g-HAT Seropositive Subjects | Safety and Tolerability Study of Acoziborole in g-HAT Seropositive Non-parasitologically Confirmed Subjects: a Multicentre Randomised Double-blind Placebo-controlled Study | Trypanosomiasis, African;Trypanosoma Brucei Gambiense; Infection;Sleeping Sickness | Drug: Acoziborole;Drug: Placebo | Drugs for Neglected Diseases | NULL | Recruiting | 15 Years | N/A | All | 1200 | Phase 2/Phase 3 | Congo, The Democratic Republic of the;Guinea |
| 16 | EUCTR2021-005001-26-NL (EUCTR) | 24/12/2021 | 08/12/2021 | The effect of high dose versus low dose antibody supplementation on airway disease in primary antibody defciency | Influencing Progression of Airway Disease in Primary Antibody Deficiency - IPAD Trial | Primary antibody deficiency:- Unclassified antibody deficiency (unPAD)- IgA deficiency- Specific polysaccharide antibody deficiency (SPAD)- IgG subclass deficiency (IgSD)- Common variable immunodeficiency (CVID)- Agammaglobulinemia (X-linked or otherwise);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Human normal immunoglobulin | UMC Utrecht | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 4 | Netherlands | ||
| 17 | EUCTR2020-004734-37-DE (EUCTR) | 14/12/2021 | 31/05/2021 | Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases | Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases | Primary Immunodeficiency Diseases MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Newnorm INN or Proposed INN: Newnorm Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 1;Phase 3 | United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany | ||
| 18 | NCT05078879 (ClinicalTrials.gov) | November 16, 2021 | 14/10/2021 | Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency | A Phase 1 Study of Empagliflozin as Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency | Crohn's Disease;Glycogen Metabolism;Inflammatory Bowel Disease (IBD) | Drug: Empagliflozin | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 18 Years | N/A | All | 10 | Phase 1 | United States |
| 19 | EUCTR2021-000136-59-BG (EUCTR) | 12/11/2021 | 05/10/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 20 | EUCTR2021-000136-59-IT (EUCTR) | 21/10/2021 | 27/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II - N/A | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Compresse a rilascio modificato Product Code: [N/A] Other descriptive name: KVD824 hydrochloride | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 21 | EUCTR2021-000136-59-CZ (EUCTR) | 13/10/2021 | 25/08/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 22 | NCT05055258 (ClinicalTrials.gov) | September 27, 2021 | 14/9/2021 | A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II | Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of 3 Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Angioedema, Hereditary, Types I and II | Drug: KVD824;Drug: Placebo to KVD824 | KalVista Pharmaceuticals, Ltd. | NULL | Recruiting | 18 Years | N/A | All | 48 | Phase 2 | United States;Australia;Canada;Czechia;France;Germany;Hungary;Italy;New Zealand;Puerto Rico;United Kingdom |
| 23 | EUCTR2021-000136-59-HU (EUCTR) | 24/09/2021 | 16/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 24 | EUCTR2021-000136-59-DE (EUCTR) | 20/09/2021 | 22/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 25 | NCT04638517 (ClinicalTrials.gov) | September 7, 2021 | 4/11/2020 | The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis | The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis | Pulmonary Fibrosis;Telomere Shortening;Telomere Disease;Dyskeratosis Congenita | Drug: Danazol;Drug: Placebo | The University of Queensland | NULL | Recruiting | 5 Years | N/A | All | 50 | Phase 2 | Australia |
| 26 | NCT03597594 (ClinicalTrials.gov) | September 2, 2021 | 19/6/2018 | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Severe Combined Immunodeficiency | Device: CliniMACS;Other: Donor Lymphocyte Infusion;Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1/Phase 2 | United States |
| 27 | NCT04640142 (ClinicalTrials.gov) | August 4, 2021 | 17/11/2020 | Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases | Prospective, Open-label, Single-arm, Multicentre Phase 3 Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency | Biological: Newnorm | Octapharma | NULL | Recruiting | 2 Years | 75 Years | All | 50 | Phase 3 | United States;Germany;Poland;Slovakia;Ukraine |
| 28 | NCT04986735 (ClinicalTrials.gov) | August 1, 2021 | 27/7/2021 | Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin | Evaluation of Safety and Efficacy of Empagliflozin for Neutropenia and Neutrophil Dysfunction in Children With Glycogen Storage Disease Type 1b (GSD1b) | Glycogen Storage Disease Type IB | Drug: Empagliflozin | Hong Kong Children's Hospital | NULL | Not yet recruiting | 6 Months | 18 Years | All | 11 | NULL | |
| 29 | NCT02860559 (ClinicalTrials.gov) | August 2021 | 26/7/2016 | Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency | Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency | Severe Combined Immunodeficiency | Biological: TBX-1400 | Taiga Biotechnologies, Inc. | NULL | Not yet recruiting | 1 Month | 4 Years | All | 8 | Phase 1 | Israel |
| 30 | NCT04925375 (ClinicalTrials.gov) | July 14, 2021 | 11/5/2021 | Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease | Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease | Interstitial Lung Disease;Common Variable Immunodeficiency | Drug: Abatacept;Other: Placebo | Children's Hospital Medical Center, Cincinnati | Bristol-Myers Squibb | Recruiting | 4 Years | N/A | All | 60 | Phase 2 | United States |
| 31 | NCT04554914 (ClinicalTrials.gov) | July 14, 2021 | 14/9/2020 | A Study to Evaluate Tabelecleucel in Participants With Epstein-barr Virus-associated Diseases | An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects With Epstein-Barr Virus-associated Diseases | Epstein-Barr Virus (EBV)-Associated Diseases;EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (PID LPD);EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (AID LPD);EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (CNS PTLD);EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD);Solid Organ Transplant Complications;Lymphoproliferative Disorders;Allogeneic Hematopoietic Cell Transplant;Stem Cell Transplant Complications;EBV+ Sarcomas;Leiomyosarcoma;Chronic Active Epstein-Barr Virus (CAEBV);Chronic Active Epstein-Barr Virus With Hemophagocytic Lymphohistiocytosis (HLH);Lymphohistiocytosis, Hemophagocytic | Biological: Tabelecleucel | Atara Biotherapeutics | NULL | Recruiting | N/A | N/A | All | 228 | Phase 2 | United States;Austria;Belgium;France;Italy;Spain;United Kingdom |
| 32 | NCT04887311 (ClinicalTrials.gov) | July 2021 | 5/5/2021 | MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia | An Open Label Study to Assess the Safety and Efficacy of MBM-01 for the Treatment of Ataxia Telangiectasia | Ataxia Telangiectasia Louis-Bar;Ataxia Telangiectasia in Children;Ataxia Telangiectasia | Drug: MBM-01 | Matrix Biomed, Inc. | The University of Texas Health Science Center, Houston | Not yet recruiting | 4 Years | N/A | All | 20 | Phase 2 | United States |
| 33 | NCT04902807 (ClinicalTrials.gov) | June 2021 | 21/5/2021 | Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation | Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation | Autoimmune Lymphoproliferative Syndrome;Autoimmune Cytopenia;Autoimmune Diseases;Autoimmune Anemia;Autoimmune Thrombocytopenia;Autoimmune Hepatitis;Autoimmune Diabetes;Autoimmune Rheumatologic Disease;Systemic Lupus Erythematosus;Juvenile Idiopathic Arthritis;Hemophagocytic Lymphohistiocytoses;EBV Lymphoproliferation;RAS-Associated Autoimmune Leucoproliferative Disease;Primary Immunodeficiency;APECED;IPEX;BENTA;Enteropathy, Autoimmune;Combined Immunodeficiency;IBD | Biological: Collection of samples | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Not yet recruiting | 1 Year | 18 Years | All | 500 | NULL | |
| 34 | EUCTR2018-000338-36-IT (EUCTR) | 12/05/2021 | 04/06/2021 | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study. | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study. - OLE-IEDAT | Neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate (soluzione 25mg/ml) Product Code: [Dexamethasone sodium phosphate] INN or Proposed INN: DEXAMETHASONE SODIUM PHOSPHATE | ERYDEL S.P.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Norway;Germany;Tunisia;United Kingdom;Italy;India | ||
| 35 | EUCTR2020-001496-32-PT (EUCTR) | 11/05/2021 | 11/08/2020 | Study, in which all know the treatment assigned to the patient, conducted in more sites, to evaluate the efficaty, safety, and metabolism of Kedrion in venous Human Normal Immunoglobulin (IVIg) 10% in child with Primary Immunodeficiency Disease (PID) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID) - PID-PED study | Pediatric Patients Affected by Primary Immunodeficiency Disease. MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: KIg10 Product Code: KIg10 INN or Proposed INN: Human Immunoglobulin | KEDRION S.P.A | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;Hungary;Slovakia;Russian Federation;Italy | ||
| 36 | NCT04842643 (ClinicalTrials.gov) | April 27, 2021 | 11/4/2021 | A Phase 3, Open-label, Non-controlled, Multi-dose, Extension Study to Evaluate the Long-term Safety and Tolerability of IGSC, 20% in Japanese Subjects With Primary Immunodeficiency Disease (PID) | A Phase 3, Open-label, Non-controlled, Multi-dose, Extension Study to Evaluate the Long-term Safety and Tolerability of IGSC, 20% in Japanese Subjects With Primary Immunodeficiency Disease (PID) | Primary Immunodeficiency Disease | Biological: IGSC 20% infusion | Takeda | NULL | Recruiting | 2 Years | N/A | All | 10 | Phase 3 | Japan |
| 37 | EUCTR2020-003445-11-BG (EUCTR) | 14/04/2021 | 02/03/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands | ||
| 38 | EUCTR2020-003445-11-NL (EUCTR) | 14/04/2021 | 01/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany | ||
| 39 | NCT04944979 (ClinicalTrials.gov) | March 31, 2021 | 11/6/2021 | Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID) | Primary Immunodeficiency Disease | Biological: Kedrion IVIG 10% | Kedrion S.p.A. | NULL | Recruiting | 2 Years | 16 Years | All | 30 | Phase 3 | Hungary;Italy;Slovakia |
| 40 | EUCTR2020-003445-11-DE (EUCTR) | 26/02/2021 | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands | ||
| 41 | EUCTR2020-003445-11-PL (EUCTR) | 23/02/2021 | 03/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands | ||
| 42 | EUCTR2021-000515-24-NL (EUCTR) | 15/02/2021 | 05/02/2021 | Vaccination Against Covid in Primary Immune Deficiencies | Vaccination Against Covid in Primary Immune Deficiencies - VACOPID | Patients with primary immunodeficiency disease, also known as inborn errors pf immunity, are vaccinated against COVID-19 using COVID-19 Vaccine Moderna. The vaccine has been approved for use in the Netherlands and all adults in the Netherlands are vaccinated according the the National Vaccination Campaign that started in January 2021 MedDRA version: 20.0;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: COVID-19 Vaccine Moderna | Erasmus University Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 700 | Phase 4 | Netherlands | ||
| 43 | ChiCTR2100043049 | 2021-02-04 | 2021-02-04 | The application of T/B cell antigen-treated shear ring (Trecs/Krecs) quantitative PCR detection in neonatal screening for primary immunodeficiency disease | The application of T/B cell antigen-treated shear ring (Trecs/Krecs) quantitative PCR detection in neonatal screening for primary immunodeficiency disease | Severe combined immunodeficiency | Gold Standard:TRECs: 40 copies / µ l whole blood Krecs: 40 copies / µ l whole blood RNase P: 2000 copies / µ l whole blood;Index test:Quantitative detection of TRECs / krecs and housekeeping gene plasmid by PCR; | Chengdu Women's and Children's Center Hospital | NULL | Recruiting | Both | Target condition:20000;Difficult condition:0 | China | |||
| 44 | NCT04618211 (ClinicalTrials.gov) | February 3, 2021 | 26/10/2020 | Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema | A Phase II, Double-blind, Placebo-controlled, Randomized, Cross-over, Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-inhibitor Deficiency Type I and II | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor Deficiency;C1 Inhibitor Deficiency | Drug: PHA-022121;Drug: Placebo | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | 75 Years | All | 72 | Phase 2 | United States;Bulgaria;Canada;Czechia;France;Germany;Hungary;Israel;Italy;Netherlands;Poland;Spain;United Kingdom |
| 45 | EUCTR2020-003445-11-HU (EUCTR) | 27/01/2021 | 30/11/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 46 | EUCTR2016-000468-41-FR (EUCTR) | 12/01/2021 | 02/12/2020 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d- activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;Belarus;France;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 47 | NCT04566692 (ClinicalTrials.gov) | January 5, 2021 | 22/9/2020 | A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency | A Multi-center, Single-Sequence, Open-label Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Subjects and Loading/Maintenance Dosing in Treatment-Naïve Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: IGSC 20% | Grifols Therapeutics LLC | NULL | Active, not recruiting | 6 Years | 75 Years | All | 33 | Phase 4 | United States |
| 48 | EUCTR2020-001496-32-IT (EUCTR) | 21/12/2020 | 21/10/2020 | Study, in which all know the treatment assigned to the patient, conducted in more sites, to evaluate the efficaty, safety, and metabolism of Kedrion in venous Human Normal Immunoglobulin (IVIg) 10% in child with Primary Immunodeficiency Disease (PID) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID) - PID-PED study | Pediatric Patients Affected by Primary Immunodeficiency Disease MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: KIg10 Product Code: [KIg10] INN or Proposed INN: IMMUNOGLOBULINA UMANA NORMALE | KEDRION S.P.A | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;Hungary;Slovakia;Italy | ||
| 49 | EUCTR2019-001153-10-GB (EUCTR) | 21/12/2020 | 29/06/2020 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 50 | EUCTR2020-003445-11-FR (EUCTR) | 18/12/2020 | 22/10/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Czech Republic;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 51 | EUCTR2018-000338-36-NO (EUCTR) | 06/11/2020 | 04/02/2020 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Tunisia;Germany;Norway;United Kingdom;Italy;India | ||
| 52 | NCT04691622 (ClinicalTrials.gov) | October 26, 2020 | 28/12/2020 | Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts | Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts | Viral Infection;Hematopoietic Stem Cell Transplantation (HSCT);Primary Immunodeficiency Disorders (PID) | Biological: Norovirus -specific T-cell (NST) therapy | Children's National Research Institute | NULL | Recruiting | 3 Months | 80 Years | All | 48 | Phase 1 | United States |
| 53 | EUCTR2019-001153-10-DE (EUCTR) | 20/10/2020 | 26/03/2020 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 3 | Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Korea, Republic of;United States;Spain;Turkey;Austria;Israel | ||
| 54 | NCT04154488 (ClinicalTrials.gov) | October 16, 2020 | 4/11/2019 | A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia Disorders | A Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia Disorders | Neutropenia | Drug: Mavorixafor | X4 Pharmaceuticals | NULL | Recruiting | 12 Years | N/A | All | 25 | Phase 1 | United States |
| 55 | EUCTR2019-001153-10-AT (EUCTR) | 16/10/2020 | 24/03/2020 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 56 | NCT04561115 (ClinicalTrials.gov) | October 6, 2020 | 10/9/2020 | A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency | A Phase 3, Multicenter, Open-label, Single-sequence, Cross-over, Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of IVIG-PEG Compared to Gamunex-C in Subjects With Primary Humoral Immunodeficiency | Primary Immunodeficiency | Biological: Gamunex-C;Biological: IVIG-PEG | Grifols Therapeutics LLC | NULL | Active, not recruiting | 18 Years | 75 Years | All | 36 | Phase 3 | United States |
| 57 | EUCTR2020-001496-32-SK (EUCTR) | 01/10/2020 | 02/07/2020 | Study, in which all know the treatment assigned to the patient, conducted in more sites, to evaluate the efficaty, safety, and metabolism of Kedrion in venous Human Normal Immunoglobulin (IVIg) 10% in child with Primary Immunodeficiency Disease (PID) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID) - PID-PED study | Pediatric Patients Affected by Primary Immunodeficiency Disease. MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: KIg10 Product Code: KIg10 INN or Proposed INN: Human Immunoglobulin | KEDRION S.P.A | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;Hungary;Slovakia;Russian Federation;Italy | ||
| 58 | EUCTR2019-002343-14-NL (EUCTR) | 25/09/2020 | 26/07/2019 | GENE THERAPY IN CHILDREN WITH RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY | PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY | Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RAG1 LV CD34+ cells Product Code: RAG1 LV CD34+ cells | Leiden University Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Netherlands | ||
| 59 | NCT04339777 (ClinicalTrials.gov) | September 22, 2020 | 8/4/2020 | Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency Diseases | A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency Diseases | Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency Disorders | Drug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irradiation;Procedure: Allogeneic HSCT;Drug: Tacrolimus (Tacro);Drug: Mycophenolate mofetil (MMF);Drug: Cyclophosphamide (Cytoxan) | National Cancer Institute (NCI) | NULL | Suspended | 4 Years | 69 Years | All | 10 | Phase 2 | United States |
| 60 | EUCTR2019-001153-10-NL (EUCTR) | 09/09/2020 | 06/04/2020 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 61 | EUCTR2020-001496-32-HU (EUCTR) | 09/09/2020 | 02/07/2020 | Study, in which all know the treatment assigned to the patient, conducted in more sites, to evaluate the efficaty, safety, and metabolism of Kedrion in venous Human Normal Immunoglobulin (IVIg) 10% in child with Primary Immunodeficiency Disease (PID) | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID) - PID-PED study | Pediatric Patients Affected by Primary Immunodeficiency Disease. MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: KIg10 Product Code: KIg10 INN or Proposed INN: Human Immunoglobulin | KEDRION S.P.A | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;Hungary;Slovakia;Russian Federation;Italy | ||
| 62 | ChiCTR2000032139 | 2020-09-01 | 2020-04-20 | Newborn screening and precision medical system construction for primary immunodeficiency disease | Newborn screening and precision medical system construction for primary immunodeficiency disease | primary immunodeficiency | Gold Standard:1. Whether there is immune deficiency; 2. Primary or secondary, persistent or temporary; 3. The part and degree of the defect of immune system; 4. Molecular diagnosis: sequence the first / second generation genes to detect whether there is pathogenic gene mutation; detection of relative protein molecular.;Index test:PID newborn screening technology system based on TRECs/KRECs combined with quantitative PCR detection; | Children's Hospital of Chongqing Medical University | NULL | Recruiting | 0 | 1 | Both | Target condition:50000;Difficult condition:0 | China | |
| 63 | NCT04528355 (ClinicalTrials.gov) | August 20, 2020 | 18/8/2020 | Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC) | Primary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory Conditions | Drug: data collection | Paul Szabolcs | NULL | Recruiting | 2 Months | 60 Years | All | 50 | United States | |
| 64 | NCT04346108 (ClinicalTrials.gov) | August 11, 2020 | 10/4/2020 | A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID) | A Phase 3, Open-label, Non-controlled, Multi-dose Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Subjects With Primary Immunodeficiency Diseases (PID) | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Intravenous (IGIV);Biological: Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%) | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 17 | Phase 3 | Japan |
| 65 | NCT04414046 (ClinicalTrials.gov) | July 22, 2020 | 24/5/2020 | TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Primary Immune Deficiency Disorders;Metabolic Disease | Biological: Haploidentical Hematopoietic Cell Transplantation | Johns Hopkins All Children's Hospital | NULL | Recruiting | N/A | 21 Years | All | 17 | Phase 2 | United States |
| 66 | EUCTR2020-000517-33-GB (EUCTR) | 14/07/2020 | 21/02/2020 | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137;Term: Genetic anomalies of leukocytes;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1;Phase 2 | United States;Spain;United Kingdom | ||
| 67 | EUCTR2018-004407-39-DE (EUCTR) | 26/06/2020 | 05/02/2020 | A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia. | Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study. | Ataxia-Telangiectasia (A-T) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Spain;Germany;United Kingdom | ||
| 68 | EUCTR2018-000338-36-GB (EUCTR) | 27/05/2020 | 20/01/2020 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT, version 5.0 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 51 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India | ||
| 69 | NCT04400045 (ClinicalTrials.gov) | May 22, 2020 | 19/5/2020 | Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome | Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome | Nijmegen Breakage Syndrome | Drug: Treosulfan | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 3 Months | 21 Years | All | 10 | Phase 2 | Russian Federation |
| 70 | NCT03879876 (ClinicalTrials.gov) | May 13, 2020 | 15/3/2019 | Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients | A Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients | Pediatric Patients;Any Type of Severe Combined Immunodeficiency (SCID);Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) | Drug: Human T Lymphoid Progenitor (HTLP) injection | Assistance Publique - Hôpitaux de Paris | NULL | Recruiting | 2 Years | 18 Years | All | 12 | Phase 1/Phase 2 | France |
| 71 | EUCTR2019-001153-10-PL (EUCTR) | 27/04/2020 | 09/03/2020 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 72 | EUCTR2019-001153-10-ES (EUCTR) | 02/03/2020 | 02/12/2019 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;United States;Spain;Turkey;Austria;Israel;Italy;United Kingdom;France;Hungary;European Union;Canada;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 73 | EUCTR2019-001153-10-IT (EUCTR) | 18/02/2020 | 17/06/2021 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome MedDRA version: 20.0;Level: LLT;Classification code 10020986;Term: Hypogammaglobulinemia, unspecified;System Organ Class: 100000004870;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: [X4P-001] INN or Proposed INN: Mavorixafor | X4 PHARMACEUTICALS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Korea, Republic of;Germany;Netherlands;Denmark;Australia;Poland | ||
| 74 | EUCTR2019-001693-28-DE (EUCTR) | 06/02/2020 | 02/09/2019 | Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older | Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with congenital C1-INH deficiency and hereditary angioedema - CONE-01 | Hereditary angioedema type I and type II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: C1 Esterase Inhibitor Human Product Code: OCTA-C1-INH Other descriptive name: C1 ESTERASE INHIBITOR (HUMAN) | Octapharma Pharmazeutika Produktionsges.m.b.H. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Belarus;Ukraine;Russian Federation;Germany | ||
| 75 | EUCTR2019-001153-10-FR (EUCTR) | 05/02/2020 | 16/10/2019 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome MedDRA version: 20.0;Level: LLT;Classification code 10010512;Term: Congenital immunodeficiency;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Portugal;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Hungary;European Union;Canada;Belgium;Poland;Romania;Australia;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 76 | NCT04275479 (ClinicalTrials.gov) | January 10, 2020 | 10/2/2020 | Diabetes/ Endocrine Surveillance in SDS | Endocrine Diabetes Screening in Patients With Shwachman-Diamond Syndrome DIABETES/ ENDOCRINE SURVEILLANCE IN SDS | Shwachman-Diamond Syndrome | Diagnostic Test: Oral Glucose Tolerance Test;Other: Modified Oral Glucose Tolerance Test;Other: Modified Mixed Meal Tolerance Test;Device: Continuous Glucose Monitor;Other: Food Diary;Other: Medical History Questionnaires | Washington University School of Medicine | Shwachman Diamond Syndrome Foundation;Barnes-Jewish Hospital | Recruiting | 3 Years | N/A | All | 60 | United States | |
| 77 | NCT03759678 (ClinicalTrials.gov) | January 8, 2020 | 27/11/2018 | N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) | Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study | Ataxia Telangiectasia;Louis Bar Syndrome | Drug: IB1001 | IntraBio Inc | NULL | Recruiting | 6 Years | N/A | All | 39 | Phase 2 | United States;Germany;Spain;United Kingdom |
| 78 | EUCTR2019-001153-10-HU (EUCTR) | 08/01/2020 | 08/11/2019 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 3 | United States;Portugal;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Hungary;European Union;Canada;Poland;Belgium;Romania;Australia;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 79 | EUCTR2019-001153-10-DK (EUCTR) | 16/12/2019 | 21/10/2019 | A treatment study in patients with WHIM Syndrome. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome. | WHIM Syndrome;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Mavorixafor Product Code: X4P-001 INN or Proposed INN: Mavorixafor | X4 Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 3 | United States;Spain;Austria;Israel;Russian Federation;Italy;United Kingdom;France;Hungary;Poland;Denmark;Australia;Germany;Netherlands;Korea, Republic of | ||
| 80 | NCT03814798 (ClinicalTrials.gov) | December 2019 | 22/1/2019 | Study Evaluating IGSC 20% Flexible Dosing in Treatment-Experienced and Treatment-Naive Subjects With Primary Immunodeficiency | A Multicenter, Randomized, Cross-over, Open-label Study to Evaluate IGSC 20% Flexible Dosing Including Daily Push Dosing In Treatment-Experienced Subjects With Primary Immunodeficiency (PI) and Evaluation of Loading/Maintenance IGSC 20% in Treatment-Naïve Subjects With PI | Primary Immunodeficiency | Biological: IGSC 20% daily push versus every 2 weeks pump;Biological: IGSC 20% daily push versus once a week pump;Biological: IGSC 20% daily push versus 2 times per week pump;Biological: IGSC 20% 150 mg/kg | Grifols Therapeutics LLC | NULL | Withdrawn | 2 Years | 75 Years | All | 0 | Phase 3 | United States |
| 81 | NCT04371939 (ClinicalTrials.gov) | November 5, 2019 | 29/4/2020 | Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome | Randomized, Two-arm Single-center Phase II Clinical Trial Comparing the Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Drug: Romiplostim;Drug: Eltrombopag | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | N/A | 18 Years | All | 30 | Phase 2 | Russian Federation |
| 82 | NCT03910452 (ClinicalTrials.gov) | October 28, 2019 | 9/4/2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cell | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Suspended | 4 Years | 65 Years | All | 4 | Early Phase 1 | United States |
| 83 | NCT03995108 (ClinicalTrials.gov) | October 17, 2019 | 19/6/2019 | Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension | WHIM Syndrome | Drug: Mavorixafor;Drug: Placebo | X4 Pharmaceuticals | NULL | Active, not recruiting | 12 Years | N/A | All | 28 | Phase 3 | United States;Australia;Austria;Denmark;France;Hungary;Israel;Italy;Korea, Republic of;Netherlands;Russian Federation;Spain;Turkey |
| 84 | NCT03939533 (ClinicalTrials.gov) | October 17, 2019 | 3/5/2019 | Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases | Clinical Phase 3 Study to Monitor the Safety, Tolerability, and Efficacy of Subcutaneous Human Immunoglobulin (CUTAQUIG®) Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder | Drug: CUTAQUIG | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 64 | Phase 3 | United States |
| 85 | NCT04140539 (ClinicalTrials.gov) | October 15, 2019 | 24/10/2019 | A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 | A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy (OTL-101) in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Severe Combined Immunodeficiency Due to ADA Deficiency | Biological: OTL-101 | Orchard Therapeutics | University of California, Los Angeles | Terminated | N/A | 17 Years | All | 3 | Phase 2/Phase 3 | United States |
| 86 | EUCTR2018-003297-27-DE (EUCTR) | 15/10/2019 | 28/03/2019 | Therapeutic use of Tadekinig alfa in NLRC4 mutation and XIAP deficiency | Multicenter, double-blind, placebo-controlled, randomized withdrawal trial with Tadekinig alfa (r-hIL-18BP) in patients with IL-18 driven monogenic autoinflammatory conditions: NLRC4 mutation and XIAP deficiency | NLRC4 mutationXIAP deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Tadekinig alfa Product Code: r-hIL-18BP INN or Proposed INN: Tadekinig alfa Other descriptive name: TADEKINIG ALFA | AB2 Bio Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 3 | United States;Canada;Germany | ||
| 87 | EUCTR2018-004489-32-AT (EUCTR) | 02/10/2019 | 07/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 88 | NCT03974178 (ClinicalTrials.gov) | September 29, 2019 | 23/5/2019 | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense: a Multicentre, Open-label Clinical Trial | Trypanosoma Brucei Rhodesiense; Infection | Drug: Fexinidazole | Drugs for Neglected Diseases | European and Developing Countries Clinical Trials Partnership (EDCTP) | Recruiting | 6 Years | N/A | All | 50 | Phase 2/Phase 3 | Malawi;Uganda |
| 89 | NCT04049084 (ClinicalTrials.gov) | September 26, 2019 | 6/8/2019 | An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID | An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID) | Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) | Orchard Therapeutics | University of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation Trust | Enrolling by invitation | N/A | N/A | All | 70 | United States;United Kingdom | |
| 90 | EUCTR2018-004407-39-GB (EUCTR) | 18/09/2019 | 06/08/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia | Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study. - IB1001-203 | To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning, and quality of life in patients with Ataxia-Telangiectasia (A-T);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB 1001 Other descriptive name: N-Acetyl-L-Leucine | IntraBio Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Germany;United Kingdom | ||
| 91 | NCT03812263 (ClinicalTrials.gov) | August 30, 2019 | 18/1/2019 | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 3 Months | N/A | All | 9 | Phase 1/Phase 2 | United States;Spain;United Kingdom |
| 92 | EUCTR2018-004489-32-PL (EUCTR) | 29/08/2019 | 29/05/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 93 | EUCTR2016-003438-26-HU (EUCTR) | 28/08/2019 | 20/06/2019 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Hungary;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 94 | NCT03984890 (ClinicalTrials.gov) | August 1, 2019 | 5/12/2018 | Vitamin D3 For CGD Patients With BCGosis/Itis | Effect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/Itis | Vitamin D3;Chronic-granulomatous Disease;BCG | Drug: Vitamin D3;Drug: Traditional treatment of CGD and TB | Children's Hospital of Fudan University | NULL | Recruiting | N/A | 18 Years | All | 50 | Phase 2/Phase 3 | China |
| 95 | EUCTR2018-004489-32-IT (EUCTR) | 23/07/2019 | 05/01/2021 | Uno studio per a) valutare la tollerabilità e i livelli ematici di KVD900 quando somministrato come dose singola ai pazienti e b) per valutare se KVD900 è efficace nel trattamento di attacchi di gonfiore nei pazienti con la malattia genetica , angioedema ereditario. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II - KVD900-201 | Hereditary Angioedema Type I or II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet Product Code: [KVD900 100 mg Film Coated Tablet] | KalVista Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | France;United States;Czechia;Hungary;Poland;Austria;Netherlands;Germany;United Kingdom;Italy | ||
| 96 | NCT04208412 (ClinicalTrials.gov) | July 2, 2019 | 20/12/2019 | A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | A Randomized, Double-blind, Placebo-controlled, Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: KVD900;Drug: Placebo | KalVista Pharmaceuticals, Ltd. | NULL | Completed | 18 Years | N/A | All | 68 | Phase 2 | United States;Austria;Czechia;Germany;Hungary;Italy;Netherlands;North Macedonia;Poland;United Kingdom |
| 97 | NCT03878069 (ClinicalTrials.gov) | June 25, 2019 | 12/3/2019 | Registry Study of Revcovi Treatment in Patients With ADA-SCID | Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: elapegademase-lvlr | Chiesi Farmaceutici S.p.A. | NULL | Recruiting | N/A | 65 Years | All | 20 | United States | |
| 98 | EUCTR2018-004489-32-DE (EUCTR) | 24/06/2019 | 12/02/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Poland;Austria;North Macedonia;Netherlands;Germany;Italy;United Kingdom | ||
| 99 | EUCTR2018-004489-32-NL (EUCTR) | 19/06/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Poland;Austria;North Macedonia;Germany;Netherlands;Italy;United Kingdom | ||
| 100 | NCT04870866 (ClinicalTrials.gov) | June 5, 2019 | 23/4/2021 | NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia | NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia | Ataxia Telangiectasia | Drug: Nicotinamide ribonucleoside | University Hospital, Akershus | The Bergesen Foundation;South-Eastern Norway Regional Health Authority;Sykehuset Innlandet HF;Oslo University Hospital;St. Olavs Hospital;Haukeland University Hospital;University Hospital of North Norway;University of Bergen | Active, not recruiting | 3 Years | N/A | All | 13 | Phase 2 | Norway |
| 101 | EUCTR2018-004489-32-HU (EUCTR) | 21/05/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 102 | NCT03961009 (ClinicalTrials.gov) | April 30, 2019 | 21/5/2019 | Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients | A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety and Pharmacokinetics of Kedrion Intravenous Immunoglobulin (IVIg) 10% in Primary Immunodeficiency Disease (PID) Patients | Primary Immunodeficiency Disease | Biological: Kedrion IVIG 10% | Kedrion S.p.A. | NULL | Completed | 2 Years | 70 Years | All | 47 | Phase 3 | United States;Canada |
| 103 | NCT03825783 (ClinicalTrials.gov) | April 15, 2019 | 25/1/2019 | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene. | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 3 Months | N/A | All | 2 | Phase 1 | Spain |
| 104 | NCT03547830 (ClinicalTrials.gov) | April 13, 2019 | 24/5/2018 | Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients | A Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Plerixafor;Drug: Gcsf | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 1 Month | 24 Years | All | 17 | Phase 2 | Russian Federation |
| 105 | EUCTR2018-004191-35-BE (EUCTR) | 12/04/2019 | 05/03/2019 | Use of an antidiabetic drug (Empagliflozin) to lower the blood level of 1,5-anhydroglucitol in patients deficient in the glucose-6-phosphate transporter (GSD1b) and the phosphatase G6PC3, both of the endoplasmic reticulum, to treat their recurrent infections by normalizing their blood neutrophil counts. Neutrophils are the most abundant white blood cells in our blood that are essential to help fighting infections. 1,5-anhydroglucitol is a sugar derivative with no known function. | Evaluation of the safety and efficacy of administration of Empagliflozin in a new treatment for neutropenia in patients with Glycogen Storage Disease type 1b (GSD1b) and G6PC3 deficiency. - GLYCO-1B | (1) Severe Congenital Neutropenia type 4 (SNC4) due to a deficiency in G6PC3, a phosphatase of the endoplasmic also known a Ubiquitous glucose-6-phosphatase and (2) the neutropenia in Glycogen Storage Disease type 1b due to a deficiency in the glucose-6-phosphate transporter (G6PT / SLC37A4) of the endoplasmic reticulum.;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Jardiance Product Name: Empagliflozin Product Code: A10BK03 | Cliniques universitaires Saint-Luc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 4 | Belgium | ||
| 106 | NCT03962114 (ClinicalTrials.gov) | March 18, 2019 | 11/4/2019 | Effects of Vitamin B3 in Patients With Ataxia Telangiectasia | Effects of Nicotinamide Riboside (Vitamin B3) in Patients With Ataxia Telangiectasia. | Ataxia Telangiectasia;ATM Gene Mutation | Dietary Supplement: Vitamin B3 | Radboud University | A-T childrens project;Twan foundation | Enrolling by invitation | 2 Years | N/A | All | 24 | Phase 2 | Netherlands |
| 107 | EUCTR2018-004489-32-GB (EUCTR) | 13/03/2019 | 27/12/2018 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom | ||
| 108 | EUCTR2016-000468-41-GB (EUCTR) | 25/02/2019 | 12/03/2018 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patientswith APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d- activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Italy;United Kingdom | ||
| 109 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/2019 | 19/11/2018 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) | A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. | Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematological Agents Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence Trade Name: Busilvex INN or Proposed INN: BUSULFAN Other descriptive name: NA Trade Name: Fludarabina Accord INN or Proposed INN: FLUDARABINE Other descriptive name: NA Trade Name: MabThera INN or Proposed INN: RITUXIMAB Other descriptive name: NA Trade Name: Mozobil, INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR Trade Name: MYELOSTIM Product Name: granulocyte colony stimulating factor (G-CSF) INN or Proposed INN: | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 110 | NCT03601286 (ClinicalTrials.gov) | December 21, 2018 | 22/2/2018 | Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan | Severe Combined Immunodeficiency, X-Linked | Drug: Lentiviral vector transduced CD34+ cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Recruiting | 8 Weeks | 5 Years | Male | 5 | Phase 1 | United Kingdom |
| 111 | EUCTR2018-002680-26-ES (EUCTR) | 27/11/2018 | 02/08/2018 | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137;Term: Genetic anomalies of leukocytes;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 2 | Phase 1 | Spain | ||
| 112 | EUCTR2016-000468-41-IE (EUCTR) | 25/10/2018 | 27/02/2018 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d- activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 113 | EUCTR2018-001029-14-FR (EUCTR) | 25/10/2018 | 02/07/2018 | HTLP | A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients - HTLP NECKER | SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT. MedDRA version: 20.0;Level: PT;Classification code 10010099;Term: Combined immunodeficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HTLP Product Code: HTLP | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | France | ||
| 114 | NCT03619551 (ClinicalTrials.gov) | October 22, 2018 | 16/7/2018 | Conditioning SCID Infants Diagnosed Early | A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRaß+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) | SCID | Drug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletion | Michael Pulsipher, MD | NULL | Recruiting | N/A | 2 Years | All | 64 | Phase 2 | United States |
| 115 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 116 | NCT03716700 (ClinicalTrials.gov) | September 24, 2018 | 31/8/2018 | Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN) | Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN) | Primary Immunodeficiency Diseases (PID) | Biological: CUVITRU | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Completed | 3 Years | N/A | All | 126 | Canada | |
| 117 | EUCTR2018-000338-36-DE (EUCTR) | 20/09/2018 | 21/06/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India | ||
| 118 | NCT03677557 (ClinicalTrials.gov) | September 19, 2018 | 6/7/2018 | Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment | Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment in Patients Who Did Not Tolerate Other 20% Subcutaneous Immunoglobulin Product(s) | Primary Immunodeficiency Disease;Secondary Immunodeficiency | Drug: 16,5% Cutaquig | Ottawa Hospital Research Institute | Octapharma | Recruiting | 18 Years | N/A | All | 30 | Phase 4 | Canada |
| 119 | NCT03663933 (ClinicalTrials.gov) | September 4, 2018 | 7/9/2018 | Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation | Phase II Trial of Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation | Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Primary T-cell Immunodeficiency Disorders;Immune System Diseases;Common Variable Immunodeficiency | Drug: Immunosuppression Only Conditioning;Drug: Reduced Intensity Conditioning;Drug: GVHD Prophylaxis;Procedure: Allogeneic HSC | National Cancer Institute (NCI) | NULL | Recruiting | 4 Years | N/A | All | 177 | Phase 2 | United States |
| 120 | EUCTR2018-000338-36-BE (EUCTR) | 23/08/2018 | 28/06/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 51 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Poland;Spain;Belgium;Australia;Israel;Germany;India | ||
| 121 | EUCTR2018-001489-41-SE (EUCTR) | 02/07/2018 | 08/05/2018 | A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions. | Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease | Dyskeratosis congenita / telomere disease MedDRA version: 20.0;Level: PT;Classification code 10001756;Term: Allogenic bone marrow transplantation therapy;System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lemtrada INN or Proposed INN: Alemtuzumab Other descriptive name: ALEMTUZUMAB Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE | Karolinska Universitetssjukhuset | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Sweden | ||
| 122 | NCT03366142 (ClinicalTrials.gov) | July 2, 2018 | 7/12/2017 | Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology | Use of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology | LAD1 | Drug: Ustekinumab | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 12 Years | 65 Years | All | 20 | Phase 1/Phase 2 | United States |
| 123 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders | Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;Adrenoleukodystrophy | Drug: Thiotepa--single daily dose;Drug: Thiotepa--escalated dose | University of Florida | Live Like Bella Pediatric Cancer Research | Recruiting | 3 Months | 39 Years | All | 40 | Phase 1/Phase 2 | United States |
| 124 | NCT03576469 (ClinicalTrials.gov) | June 13, 2018 | 15/6/2018 | A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | CVI - Common Variable Immunodeficiency | Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) | IMMUNOe Research Centers | NULL | Completed | 18 Years | N/A | All | 20 | Phase 4 | United States |
| 125 | EUCTR2016-000468-41-IT (EUCTR) | 01/06/2018 | 02/09/2021 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Product Name: Leniolisib Product Code: [CDZ173] INN or Proposed INN: leniolisib | NOVARTIS PHARMA SERVICES AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy | ||
| 126 | EUCTR2018-000338-36-ES (EUCTR) | 31/05/2018 | 05/04/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 61 | Phase 3 | United States;Poland;Belgium;Spain;Australia;Israel;Norway;Germany;Italy;United Kingdom;India | ||
| 127 | NCT03538899 (ClinicalTrials.gov) | May 31, 2018 | 3/5/2018 | Autologous Gene Therapy for Artemis-Deficient SCID | A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells | Severe Combined Immunodeficiency | Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan | University of California, San Francisco | NULL | Recruiting | 2 Months | N/A | All | 25 | Phase 1/Phase 2 | United States |
| 128 | JPRN-jRCTs031180398 | 30/05/2018 | 20/03/2019 | A phase II study of RIC-SCT for CGD | Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial - CGD-RIST2 | Chronic granulomatous disease Primary immunodeficiency;D006105 | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | Sakaguchi Hirotoshi | NULL | Recruiting | Not applicable | < 25age old | Both | 22 | Phase 2 | None (Japan only);Japan |
| 129 | NCT03548818 (ClinicalTrials.gov) | May 16, 2018 | 20/3/2018 | Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease | Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Interferon Gamma-1B | University of Colorado, Denver | NULL | Completed | 5 Years | 60 Years | All | 9 | United States | |
| 130 | NCT03232203 (ClinicalTrials.gov) | April 12, 2018 | 25/7/2017 | Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs) | Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment | Severe Combined Immunodeficiency Due to ADA Deficiency | Drug: STRIMVELIS | Orchard Therapeutics | NULL | Completed | 18 Years | N/A | All | 16 | Italy | |
| 131 | NCT03512314 (ClinicalTrials.gov) | January 24, 2018 | 9/4/2018 | Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension | Open-label Extension Study With Tadekinig Alfa (r-hIL-18BP) to Monitor Safety and Tolerability in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency | XIAP Deficiency;NLRC4-MAS | Drug: Tadekinig alfa | AB2 Bio Ltd. | NULL | Recruiting | N/A | N/A | All | 10 | Phase 3 | United States;Canada;Germany |
| 132 | NCT03311503 (ClinicalTrials.gov) | January 19, 2018 | 12/10/2017 | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Severe Combined Immunodeficiency, X Linked;Gene Therapy | Biological: autologous CD34+ cell transduced with G2SCID vector | David Williams | NULL | Recruiting | N/A | 5 Years | Male | 10 | Phase 1/Phase 2 | United States;United Kingdom |
| 133 | JPRN-UMIN000030806 | 2018/01/17 | 15/01/2018 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | Wiskott-Aldrich syndrome | WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan. 1. Rituximab (day-22) 375 mg/m2 2. Preconditioning chemotherapy Fludarabine 30mg/m2 x 2 (day-3, day-2) Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours) 3. Infusion of WASP cDNA-transduced CD34 positive HSC 5 x 10^6/kg (at least 3 x 10^6/kg) | National Center for Child Heath and Development | NULL | Complete: follow-up continuing | Not applicable | Not applicable | Male | 3 | Phase 1,2 | Japan |
| 134 | NCT03765632 (ClinicalTrials.gov) | January 3, 2018 | 8/8/2018 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Active, not recruiting | N/A | 17 Years | All | 13 | Phase 1/Phase 2 | United Kingdom |
| 135 | JPRN-UMIN000030647 | 2017/12/30 | 31/12/2017 | Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial (CGD-RIST2) | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | NULL | Recruiting | Not applicable | 25years-old | Male and Female | 22 | Phase 2 | Japan | |
| 136 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | NULL | Recruiting | 1 Year | 75 Years | All | 58 | Phase 2 | United States |
| 137 | NCT03330795 (ClinicalTrials.gov) | December 1, 2017 | 30/10/2017 | Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant | Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003) | Primary Immunodeficiency;PID | Biological: CD3/CD19 neg allogeneic BMT | National Institute of Allergy and Infectious Diseases (NIAID) | University of Pittsburgh;PPD | Active, not recruiting | 10 Years | 45 Years | All | 5 | Phase 1/Phase 2 | United States |
| 138 | EUCTR2016-003438-26-GR (EUCTR) | 13/11/2017 | 11/10/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 139 | EUCTR2017-001731-39-IT (EUCTR) | 12/10/2017 | 02/07/2020 | Retroviral insertion site methodology study | Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy - Retroviral insertion site methodology study | Adenosine deaminase (ADA) deficiency severe combined immunodeficiency MedDRA version: 20.1;Level: LLT;Classification code 10066367;Term: Adenosine deaminase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Strimvelis Product Name: Strimvelis Product Code: [NA] | Orchard Therapeutics (Europe) Ltd | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 4 | Turkey;Switzerland;Italy | ||
| 140 | NCT03828279 (ClinicalTrials.gov) | October 1, 2017 | 23/1/2019 | Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and II | The Global Registry on Hereditary Angioedema Type I and II | Hereditary Angioedema Type I and II | Diagnostic Test: functional and antigenic C1 inhibitor | HAE Global Registry Foundation | NULL | Recruiting | N/A | N/A | All | 220 | Italy | |
| 141 | JPRN-jRCT2031200236 | 28/09/2017 | 10/12/2020 | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis | chronic granulomatous disease-associated colitis primary immunodeficiency diseases; inflammatory bowel disease;D006105 | 1) Oral administration of thalidomide (1.5-3mg/kg), once a day 2) Oral administration of placebo (1.5-3mg/kg), once a day | Kawai Toshinao | Kawai Toshinao;Japan Agency for Medical Research and Development | Recruiting | >= 1age old | Not applicable | Both | 8 | Phase 2-3 | Japan |
| 142 | JPRN-UMIN000029324 | 2017/09/28 | 01/10/2017 | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis | Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis - Clinical trial of thalidomide for CGD colitis | Chronic granulomatous disease associated colitis | Oral administration of thalidomide (1.5-3mg/kg), once a day Oral administration of placebo (1.5-3mg/kg), once a day | National Center for Child Heath and Development | NULL | Recruiting | 1years-old | Not applicable | Male and Female | 8 | Not selected | Japan |
| 143 | NCT03277313 (ClinicalTrials.gov) | September 25, 2017 | 7/9/2017 | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: GAMMAGARD LIQUID | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Active, not recruiting | 2 Years | 15 Years | All | 44 | Phase 3 | United States |
| 144 | EUCTR2017-001275-23-GB (EUCTR) | 21/09/2017 | 03/07/2017 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells Product Code: OTL-101 INN or Proposed INN: There is no recommended INN Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | ||
| 145 | NCT03080480 (ClinicalTrials.gov) | September 1, 2017 | 27/2/2017 | Pioglitazone Therapy for Chronic Granulomatous Disease | Efficacy and Safety of Pioglitazone Therapy for Chronic Granulomatous Disease Patients With Severe Infection. | Chronic Granulomatous Disease | Drug: Pioglitazone | Children's Hospital of Fudan University | NULL | Terminated | 1 Month | 18 Years | All | 3 | Phase 1/Phase 2 | China |
| 146 | EUCTR2015-003290-15-HU (EUCTR) | 01/09/2017 | 12/05/2017 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Czech Republic;Hungary;Poland;Spain;Australia;Germany;United Kingdom;Sweden | ||
| 147 | NCT03113760 (ClinicalTrials.gov) | July 21, 2017 | 28/2/2017 | Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency | Multicenter, Double-blind, Placebo-controlled, Randomized Withdrawal Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency | NLRC4-MAS;XIAP Deficiency | Drug: Tadekinig alfa;Other: 0.9% sodium chloride | AB2 Bio Ltd. | NULL | Recruiting | N/A | N/A | All | 10 | Phase 3 | United States;Canada;Germany |
| 148 | NCT03217617 (ClinicalTrials.gov) | July 15, 2017 | 3/7/2017 | Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | SCID, X Linked | Biological: TYF-IL-2Rg gene-modified autologous stem cells | Shenzhen Geno-Immune Medical Institute | NULL | Recruiting | 1 Month | 10 Years | Male | 10 | Phase 1/Phase 2 | China |
| 149 | NCT03116347 (ClinicalTrials.gov) | June 13, 2017 | 12/4/2017 | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD Subjects | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: KIOVIG;Biological: Cuvitru | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Completed | 2 Years | 17 Years | All | 42 | Phase 4 | Czechia;Denmark;France;Greece;Slovakia;Sweden;United Kingdom |
| 150 | EUCTR2016-003438-26-CZ (EUCTR) | 18/05/2017 | 27/01/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 151 | EUCTR2016-003438-26-DK (EUCTR) | 08/05/2017 | 19/01/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 152 | EUCTR2016-003438-26-FR (EUCTR) | 18/04/2017 | 21/03/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 153 | EUCTR2016-003438-26-SE (EUCTR) | 04/04/2017 | 08/11/2016 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 154 | NCT03988426 (ClinicalTrials.gov) | March 7, 2017 | 13/6/2019 | Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases | Clinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases. | Primary Immune Deficiency Disorder | Biological: Octanorm | Octapharma | NULL | Completed | 18 Years | 70 Years | All | 25 | Phase 3 | Russian Federation |
| 155 | NCT02770807 (ClinicalTrials.gov) | March 2, 2017 | 2/5/2016 | EDS in Ataxia Telangiectasia Patients | Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With Ataxia Telangiectasia | Nervous System Disease;Genetic Syndrome | Drug: EDS-EP dose range of ~5-10 mg DSP/infusion;Drug: EDS-EP dose range of ~14-22 mg DSP/infusion;Drug: Placebo | Erydel | NULL | Completed | 6 Years | N/A | All | 175 | Phase 3 | United States;Australia;Belgium;Germany;India;Israel;Italy;Norway;Poland;Spain;Tunisia;United Kingdom |
| 156 | NCT03112655 (ClinicalTrials.gov) | February 24, 2017 | 31/3/2017 | Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: Early Test-of-cure | Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: WP4 Early Test-of-cure | African Trypanosomiasis;African; Trypanosomiasis, West;Sleeping Sickness; West African;Trypanosoma Brucei Gambiense; Infection | Diagnostic Test: RNA and neopterin detection | Institut de Recherche pour le Developpement | Institute of Tropical Medicine, Belgium;Institut National de Recherche Biomédicale. Kinshasa, République Démocratique du Congo;Ministry of Public Health, Democratic Republic of the Congo | Completed | 15 Years | N/A | All | 88 | N/A | Congo, The Democratic Republic of the |
| 157 | EUCTR2016-003438-26-GB (EUCTR) | 14/02/2017 | 01/11/2016 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 158 | EUCTR2015-003652-52-GB (EUCTR) | 02/02/2017 | 08/08/2016 | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | Replacement therapy in patients with primary immunodeficiency disease (PID) MedDRA version: 19.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: IgG Next Generation Product Code: BT595 INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Spain;Russian Federation;Germany;United Kingdom | ||
| 159 | NCT03033745 (ClinicalTrials.gov) | February 1, 2017 | 23/1/2017 | Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra) in Subjects With Primary Immunodeficiency (PID) | An Open-label Multicenter Study to Evaluate the Safety and Tolerability of Higher Infusion Parameters of Immune Globulin Subcutaneous (Human), 20% Liquid (Hizentra®) in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Drug: IgPro20 | CSL Behring | NULL | Completed | 2 Years | N/A | All | 49 | Phase 4 | United States;Canada |
| 160 | EUCTR2013-003877-87-SK (EUCTR) | 13/01/2017 | 17/03/2016 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1;Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland;Russian Federation | ||
| 161 | NCT03733249 (ClinicalTrials.gov) | January 2017 | 2/11/2018 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Drug: Rimiducid;Biological: rivogenlecleucel | Bellicum Pharmaceuticals | NULL | Enrolling by invitation | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;Saudi Arabia;United Kingdom |
| 162 | NCT03054181 (ClinicalTrials.gov) | December 22, 2016 | 13/2/2017 | Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO) | Facilitated Immunoglobulin Administration Registry and Outcomes Study | Primary Immunodeficiency;Secondary Immune Deficiency | Biological: HyQvia | GWT-TUD GmbH | NULL | Completed | N/A | N/A | All | 156 | France;Germany;Italy | |
| 163 | NCT02999984 (ClinicalTrials.gov) | December 16, 2016 | 19/12/2016 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles | Completed | N/A | 17 Years | All | 10 | Phase 1/Phase 2 | United States |
| 164 | NCT03037359 (ClinicalTrials.gov) | December 2016 | 20/1/2017 | A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin Products | A Multicenter, Non-interventional, Observational, Prospective Study to Assess Hypotension in Patients With Primary Immunodeficiency Disease Treated With Bivigam™ (Human 10%) or Other Commercial Human 10% Immune Globulin (Intravenous) (IGIV) Products During Infusion and up to 72 Hours Post Infusion | Primary Immune Deficiency Disorder | Biological: Bivigam;Biological: Other | ADMA Biologics, Inc. | NULL | Recruiting | N/A | N/A | All | 200 | United States | |
| 165 | NCT03005327 (ClinicalTrials.gov) | December 2016 | 20/12/2016 | A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome | A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome | WHIM Syndrome | Drug: X4P-001 | X4 Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 15 | Phase 2 | United States;Australia |
| 166 | EUCTR2015-005241-31-NO (EUCTR) | 21/11/2016 | 02/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;Norway;United Kingdom;Italy;India | ||
| 167 | NCT03025789 (ClinicalTrials.gov) | November 17, 2016 | 11/11/2016 | Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | An Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole in Patients With Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | Trypanosomiasis, African;Sleeping Sickness;Trypanosomiasis; Gambian | Drug: Fexinidazole | Drugs for Neglected Diseases | Sanofi | Completed | 6 Years | N/A | All | 174 | Phase 3 | Congo, The Democratic Republic of the;Guinea |
| 168 | NCT02996448 (ClinicalTrials.gov) | November 17, 2016 | 16/12/2016 | Safety, Tolerability, and Immunogenicity of One Dose of NDV 3A Vaccine in People With STAT3-Mutated Hyper-IgE Syndrome | A Phase 2a Study to Evaluate the Safety, Tolerability, and Immunogenicity of One Dose of NDV-3A Vaccine in Patients With STAT3-Mutated Hyper-IgE Syndrome | Autosomal-dominant Hyper-IgE Syndrome | Drug: NDV-3A | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 18 Years | 55 Years | All | 3 | Phase 2 | United States |
| 169 | NCT02881437 (ClinicalTrials.gov) | November 11, 2016 | 17/8/2016 | IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQvia | Assessment of the IgG Trough Level in Subjects With Primary Immunodeficiency Switching From Standard Subcutaneous Immunoglobulin (SCIG) to Every Other Week HyQvia | Primary Immunodeficiency | Drug: IgHy10 | University Hospital, Lille | Shire | Completed | 18 Years | N/A | All | 22 | Phase 4 | France |
| 170 | EUCTR2016-000468-41-NL (EUCTR) | 03/11/2016 | 19/09/2016 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy | ||
| 171 | EUCTR2015-005241-31-IT (EUCTR) | 02/11/2016 | 28/02/2018 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexametasone Fosfato Sodico INN or Proposed INN: Dexametasone Fosfato Sodico Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | ERYDEL S.P.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | Tunisia;United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;Belgium;Poland;Australia;Germany;Norway | ||
| 172 | EUCTR2015-005241-31-BE (EUCTR) | 21/10/2016 | 01/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;United Kingdom;Italy;India;Norway | ||
| 173 | NCT03087955 (ClinicalTrials.gov) | October 11, 2016 | 7/3/2017 | Prospective Study on Efficacy and Safety of Acoziborole (SCYX-7158) in Patients Infected by Human African Trypanosomiasis Due to T.b. Gambiense | Efficacy and Safety Study of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective Study | Trypanosomiasis, African;Gambiense Trypanosomiasis;Sleeping Sickness | Drug: Acoziborole (SCYX-7158) | Drugs for Neglected Diseases | NULL | Completed | 15 Years | N/A | All | 260 | Phase 2/Phase 3 | Congo, The Democratic Republic of the;Guinea |
| 174 | NCT02810444 (ClinicalTrials.gov) | October 4, 2016 | 15/6/2016 | Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID | An Open-label, Prospective, Multicenter Study Investigating Clinical Efficacy, Safety, and Pharmacokinetic Properties of the Human Normal Immunoglobulin for Intravenous Administration BT595 as Replacement Therapy in Patients With Primary Immunodeficiency Disease (PID) | Primary Immunodeficiency Disease | Biological: IgG Next Generation (BT595) | Biotest | Syneos Health | Completed | 2 Years | 75 Years | All | 81 | Phase 3 | United States;Germany;Hungary;Russian Federation;Spain |
| 175 | EUCTR2015-003652-52-DE (EUCTR) | 29/09/2016 | 18/05/2016 | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | Replacement therapy in patients with primary immunodeficiency disease (PID) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: IgG Next Generation Product Code: BT595 INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;Germany;United Kingdom | ||
| 176 | NCT02733679 (ClinicalTrials.gov) | September 29, 2016 | 31/3/2016 | Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone | Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone | Ataxia-Telangiectasia | Drug: Metformin;Drug: Pioglitazone | NHS Tayside | University of Dundee | Completed | 18 Years | 30 Years | All | 27 | Phase 4 | United Kingdom |
| 177 | EUCTR2015-003652-52-ES (EUCTR) | 19/09/2016 | 15/07/2016 | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID) | Replacement therapy in patients with primary immunodeficiency disease (PID) MedDRA version: 19.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: IgG Next Generation Product Code: BT595 INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Hungary;Spain;Russian Federation;Germany;United Kingdom | ||
| 178 | EUCTR2015-005241-31-DE (EUCTR) | 14/09/2016 | 27/05/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Norway;Germany;United Kingdom;Italy;India | ||
| 179 | NCT01512888 (ClinicalTrials.gov) | August 17, 2016 | 13/1/2012 | Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants | A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells | Severe Combined Immunodeficiency Disease, X-linked | Genetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacs | St. Jude Children's Research Hospital | National Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM) | Recruiting | N/A | 24 Months | Male | 28 | Phase 1/Phase 2 | United States |
| 180 | EUCTR2016-000468-41-CZ (EUCTR) | 19/07/2016 | 25/05/2016 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib Other descriptive name: CDZ173 Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 181 | EUCTR2015-003290-15-DE (EUCTR) | 12/07/2016 | 16/03/2016 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Hungary;Czech Republic;Poland;Spain;Australia;Germany;United Kingdom;Sweden | ||
| 182 | EUCTR2016-001480-36-FR (EUCTR) | 11/07/2016 | 22/11/2016 | Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQvia | Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQvia - HyQvia | Primary immunodeficiencies MedDRA version: 19.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia Other descriptive name: HyQvia | Centre Hospitalier Régional et Universitaire de Lille | NULL | Not Recruiting | Female: yes Male: yes | 22 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France | ||
| 183 | EUCTR2015-003369-27-DE (EUCTR) | 01/07/2016 | 18/05/2016 | A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID) | A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID) - REGAIN: REGulatory T cells and Aldesleukin for Immunodeficiency- associated eNteropathy | autoimmune enteropathy (AIE) in common variable immunodeficiency (CVID). MedDRA version: 19.0;Level: PT;Classification code 10021449;Term: Immunodeficiency common variable;System Organ Class: 10021428 - Immune system disorders MedDRA version: 19.0;Level: LLT;Classification code 10017922;Term: Gastroenteropathy NOS;System Organ Class: 100000004856;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: PROLEUKIN® S Product Name: Aldesleukin INN or Proposed INN: Aldesleukin Other descriptive name: ALDESLEUKIN | Universitätsklinikum Freiburg | NULL | Not Recruiting | Female: yes Male: yes | 6 | Phase 2 | Germany | ||
| 184 | NCT02609932 (ClinicalTrials.gov) | July 2016 | 11/11/2015 | Effect of IFN-? on Innate Immune Cells | Effect of Interferon-gamma 1-b on Innate Immune Cells | Chronic Granulomatous Disease | Drug: Administration of drug (Interferon-gamma 1-b) subcutaneously | University of Colorado, Denver | NULL | Completed | 18 Years | 60 Years | All | 20 | Phase 1 | United States |
| 185 | NCT02822495 (ClinicalTrials.gov) | July 2016 | 30/6/2016 | Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or Malignancies | Expanded Access Protocol for Providing Tabelecleucel to Patients With Epstein-Barr Virus-Associated Viremia or Malignancies for Whom There Are No Appropriate Alternative Therapies | Epstein-Barr Virus (EBV) Infections;Lymphoproliferative Disorders;EBV+ Associated Lymphoma;EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD);Epstein-Barr Viremia;Lymphoma, AIDS-related;Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Primary Immunodeficiency (PID);Leiomyosarcoma (LMS);Nasopharyngeal Carcinoma (NPC);Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Acquired Immunodeficiency (AID);Solid Organ Transplant Complications;Stem Cell Transplant Complications | Biological: tabelecleucel | Atara Biotherapeutics | NULL | Available | N/A | N/A | All | United States | ||
| 186 | EUCTR2015-003290-15-FR (EUCTR) | 29/06/2016 | 28/09/2018 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.1;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom | ||
| 187 | EUCTR2015-003290-15-CZ (EUCTR) | 22/06/2016 | 01/02/2016 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.1;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom | ||
| 188 | NCT02737384 (ClinicalTrials.gov) | June 14, 2016 | 8/4/2016 | Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID) | Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID): Selective Depletion of Naive Cells From the Graft | Combined Immunodeficiencies | Biological: Depletion in CD45RA graft donor | Assistance Publique - Hôpitaux de Paris | NULL | Terminated | 12 Months | 18 Years | All | 4 | Phase 2 | France |
| 189 | JPRN-UMIN000022688 | 2016/06/10 | 13/06/2016 | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease - A pilot study of RIC-SCT for CGD | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | NULL | Complete: follow-up complete | Not applicable | 25years-old | Male and Female | 9 | Not selected | Japan |
| 190 | NCT03019809 (ClinicalTrials.gov) | June 2016 | 11/1/2017 | A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients | A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome. | Wiskott-Aldrich Syndrome;Hematopoietic Stem Cell Transplantation;Graft Failure | Biological: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT. | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Unknown status | 1 Month | 19 Years | All | 30 | Phase 2 | Russian Federation |
| 191 | NCT02806986 (ClinicalTrials.gov) | June 2016 | 3/6/2016 | Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency | A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: IGSC 20% | Grifols Therapeutics LLC | NULL | Completed | 2 Years | 75 Years | All | 61 | Phase 3 | Australia;Czechia;France;Germany;Hungary;Poland;Spain;Sweden;United Kingdom;Czech Republic |
| 192 | EUCTR2015-005241-31-ES (EUCTR) | 31/05/2016 | 09/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 19.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Spain;Costa Rica;Turkey;Israel;United Kingdom;Italy;Belgium;Poland;Australia;Tunisia;Germany;Norway | ||
| 193 | EUCTR2015-003290-15-GB (EUCTR) | 12/05/2016 | 13/01/2016 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Australia;Germany;United Kingdom;Sweden | ||
| 194 | EUCTR2015-002491-24-DE (EUCTR) | 11/05/2016 | 30/03/2016 | Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease | Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease - SAIL | CVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs MedDRA version: 18.1;Level: PT;Classification code 10022611;Term: Interstitial lung disease;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders MedDRA version: 18.1;Classification code 10021449;Term: Immunodeficiency common variable;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: ORENCIA® 125 mg solution for injection in pre-filled syringe Product Name: Abatacept Product Code: BMS-188667 | Universitätsklinikum Freiburg, vertreten durch den Leitenden Ärztlichen Direktor | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | Germany | ||
| 195 | NCT02789397 (ClinicalTrials.gov) | May 2, 2016 | 16/5/2016 | Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease in Patients With Common Variable Immunodeficiency | Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID) | Granulomatous and Lymphocytic Interstitial Lung Disease | Drug: Rituximab (RTX) and Azathioprine (AZA);Drug: Placebos | Medical College of Wisconsin | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | NULL |
| 196 | EUCTR2015-003290-15-ES (EUCTR) | 12/04/2016 | 22/02/2016 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 18.1;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 18.1;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom | ||
| 197 | NCT03907241 (ClinicalTrials.gov) | March 1, 2016 | 7/12/2018 | CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL | Title for SCGAM-03: CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES WHO HAVE COMPLETED THE SCGAM-01 TRIAL Title for SCGAM-03 in Canada: CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL | Primary Immunodeficiency | Drug: Octanorm 16.5% | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 27 | Phase 3 | United States;Canada |
| 198 | NCT02627300 (ClinicalTrials.gov) | March 2016 | 3/12/2015 | Study of Octanorm Subcutaneous IG in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Clinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Primary Immunodeficiency Disease | Drug: Octanorm 16.5% | Octapharma | NULL | Completed | N/A | N/A | All | 21 | Phase 3 | United States;Canada |
| 199 | NCT02269163 (ClinicalTrials.gov) | January 26, 2016 | 16/10/2014 | Study of ProMetic BioTherapeutics Immune Globulin Intravenous (Human) 10% | A Phase 3, Multicenter, Open-Label Study of the Safety, Tolerability, Efficacy, and PK of ProMetic BioTherapeutics IGIV (Human) 10% in Adults and Children With Primary Immunodeficiency Diseases | Primary Immunodeficiency | Biological: Immune Globulin Intravenous;Biological: Prometic's Immune Globulin Intravenous 10% | Prometic Biotherapeutics, Inc. | Atlantic Research Group | Completed | 2 Years | 80 Years | All | 82 | Phase 3 | United States |
| 200 | NCT02123615 (ClinicalTrials.gov) | January 2016 | 11/4/2014 | ASIS for GAMMAGARD in Primary Immunodeficiency | ASIS for GAMMAGARD in Primary Immunodeficiency | Primary Immunodeficiency | Drug: Gadolinium For abdomen;Drug: Gadolinium For lower back;Drug: Efficacy of Gammagard subcutaneously at Week 12;Drug: Efficacy of Gammagard subcutaneously at Week 24;Drug: Efficacy of Gammagard subcutaneously at Week 36;Drug: Efficacy of Gammagard subdermally at Week 36;Drug: Efficacy of Gammagard subdermally at Week 12;Drug: Efficacy of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 12;Drug: Adverse Reactions of Gammagard subcutaneously at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 36;Drug: Adverse Reactions of Gammagard subdermally at Week 12;Drug: Adverse Reactions of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subdermally at Week 36 | ASIS Corporation | NULL | Not yet recruiting | 21 Years | 65 Years | Both | 60 | Phase 1/Phase 2 | United States |
| 201 | NCT02604810 (ClinicalTrials.gov) | January 2016 | 6/11/2015 | Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency | An Open-label, Multi-center Study to Evaluate the Safety and Pharmacokinetics of IGSC 20% Administered for 6 Months in Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: IGIV-C 10%;Biological: IGSC 20% | Grifols Therapeutics LLC | NULL | Completed | 2 Years | 75 Years | All | 53 | Phase 3 | United States;Canada |
| 202 | NCT02629120 (ClinicalTrials.gov) | December 17, 2015 | 10/12/2015 | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease Transplant | Drug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 50 | Phase 1/Phase 2 | United States |
| 203 | JPRN-UMIN000019532 | 2015/12/01 | 01/12/2015 | Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases | Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases - HSCT for ataxia telangiectasia and related diseases | ataxia telangiectasiaDNA damage response disorder | fludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4 | Graduate Schoool,Dept. of Community Pediatrics,Perinatal and Maternal Medicine,Tokyo Medical and Dental University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 5 | Phase 2 | Japan |
| 204 | NCT02579967 (ClinicalTrials.gov) | November 19, 2015 | 16/10/2015 | Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies | Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies | Primary T-cell Immunodeficiency Disorders;Common Variable Immunodeficiency;Immune System Diseases;Autoimmune Lymphoproliferative;Lymphoproliferative Disorders | Drug: Immunosuppression Only Conditioning -Closed with amendment L;Drug: Reduced Intensity Conditioning;Drug: Myeloablative Conditioning-Closed with amendment L;Drug: GVHD Prophylaxis;Procedure: Allo BMT | National Cancer Institute (NCI) | NULL | Recruiting | 4 Years | 75 Years | All | 224 | Phase 2 | United States |
| 205 | NCT02593188 (ClinicalTrials.gov) | November 12, 2015 | 29/10/2015 | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA | Baxalta now part of Shire | NULL | Active, not recruiting | 16 Years | N/A | All | 264 | United States | |
| 206 | NCT03055247 (ClinicalTrials.gov) | November 2015 | 21/7/2016 | Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD | A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD | Chronic Granulomatous Disease X-linked (X-CGD) | Drug: Ibuprofen;Drug: Myelostim;Drug: Mozobil | IRCCS San Raffaele | Fondazione Telethon | Recruiting | 18 Years | 45 Years | Male | 3 | Phase 2 | Italy |
| 207 | NCT02234934 (ClinicalTrials.gov) | October 29, 2015 | 4/9/2014 | Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease | A Two-Part, Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic, X-linked | Biological: Lentiviral G1XCGD Gene Therapy | University of California, Los Angeles | Boston Children's Hospital;National Heart, Lung, and Blood Institute (NHLBI);Genethon;California Institute for Regenerative Medicine (CIRM) | Recruiting | 23 Months | N/A | Male | 16 | Phase 1/Phase 2 | United States |
| 208 | NCT02199496 (ClinicalTrials.gov) | October 19, 2015 | 23/7/2014 | Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | An Open-Label Phase I/II Pilot Study to Assess the Safety/Tolerability and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Gastrointestinal Inflammation Associated With CVID;CVID Enteropathy | Biological: Stelara (ustekinumab) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Years | 75 Years | All | 5 | Phase 1/Phase 2 | United States |
| 209 | EUCTR2015-002356-27-IT (EUCTR) | 16/10/2015 | 29/07/2015 | Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGD | A multicentric, exploratory, non-randomised, non-controlled, prospective, open-label phase II, study evaluating safety and efficacy of IBU, G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by X-CGD. | X-linked chronic granulomatous disease MedDRA version: 18.0;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ibuprofen INN or Proposed INN: IBUPROFEN Other descriptive name: NA Trade Name: MYELOSTIM 34 milions UI/ml, powder and solvent for solution for injection or infusion Product Name: MYELOSTIM 34 milions UI/ml - powder and solvent for solution for injection/infusion INN or Proposed INN: LENOGRASTIM Trade Name: Mozobil 20mg/mL vial (injectable solution for subcutaneous use) Product Name: Mozobil 20mg/mL vial (injectable solution, subcutaneous use) INN or Proposed INN: Plerixafor Other descriptive name: Plerixafor Product Name: Pantoprazolo 20 mg gastro-resistant tablets INN or Proposed INN: Pantoprazole Other descriptive name: PANTOPRAZOLE | Ospedale San Raffaele | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 3 | Phase 2 | Italy | ||
| 210 | NCT02787486 (ClinicalTrials.gov) | October 2015 | 26/5/2016 | Expanded Noninvasive Genomic Medical Assessment: The Enigma Study | A Clinical Study to Evaluate the Relative Clinical Sensitivity, Specificity, and Performance of the a Laboratory Developed Test as a Screening Test for Fetal Chromosomal Aneuploidy, Infectious and Other Diseases, and RhD Genotyping in the General Population of Pregnant Women | Down Syndrome;Edwards Syndrome;Patau Syndrome;Klinefelter Syndrome;Turner Syndrome;DiGeorge Syndrome;Chromosome Deletion;Aneuploidy | Other: Blood sampling for Laboratory Developed Test (LDT) analysis | Progenity, Inc. | NULL | Completed | 18 Years | 54 Years | Female | 760 | United States | |
| 211 | NCT04136028 (ClinicalTrials.gov) | September 25, 2015 | 21/10/2019 | IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease | A Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Kineret | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Completed | N/A | 18 Years | All | 13 | Early Phase 1 | Russian Federation |
| 212 | NCT02435173 (ClinicalTrials.gov) | August 24, 2015 | 24/2/2015 | Study of Efficacy of CDZ173 in Patients With APDS/PASLI | An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor-blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 in Patients With APDS/PASLI | Common Variable Immunodeficiency (CVID), APDS / PASLI | Drug: CDZ173;Other: Placebo | Novartis Pharmaceuticals | NULL | Completed | 12 Years | 75 Years | All | 37 | Phase 2/Phase 3 | United States;Belarus;Czechia;Germany;Ireland;Italy;Netherlands;Russian Federation;United Kingdom;Czech Republic;France |
| 213 | NCT01884311 (ClinicalTrials.gov) | August 20, 2015 | 14/6/2013 | Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Subgam-VF in Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyperimmunoglobulin M Syndrome | Biological: Subgam | Bio Products Laboratory | NULL | Completed | 2 Years | 75 Years | All | 38 | Phase 3 | United States |
| 214 | NCT02369978 (ClinicalTrials.gov) | August 2015 | 17/2/2015 | CHICAMOCHA 3 - Equivalence of Usual Interventions for Trypanosomiasis (EQUITY) | Cardiovascular Health Investigation and Collaboration From Countries of America to Assess the Markers and Outcomes of Chagas Disease (CHICAMOCHA-3) - EQUITY (Equivalence of Usual Interventions for Trypanosomiasis) | Chagas Disease | Drug: Nifurtimox;Drug: Benznidazole;Drug: Placebo | Universidad Autónoma de Bucaramanga | Fundación Cardioinfantil Instituto de Cardiología;Instituto Nacional de Salud (Colombia);Instituto Nacional de Parasitologia Dr. Mario Fatala Chaben | Recruiting | 20 Years | 55 Years | All | 500 | Phase 2/Phase 3 | Colombia |
| 215 | NCT02503293 (ClinicalTrials.gov) | July 29, 2015 | 1/7/2015 | A Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL Administered With Two Different Delivery Devices: Injections Using Pump or Rapid Push | A Randomised, Cross-over Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL Administered With Two Different Delivery Devices: Injections Using Pump or Rapid Push | Primary Immunodeficiency | Device: Chrono Super PID then Generic Syringe-Gammanorm;Device: Generic Syringe then Chrono Super PID-Gammanorm | Octapharma | NULL | Completed | 18 Years | N/A | All | 30 | Phase 4 | Australia;Germany;Italy;United Kingdom |
| 216 | EUCTR2013-005508-33-IT (EUCTR) | 13/07/2015 | 21/05/2015 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 18.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 18.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 18.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Italy;Germany;Austria;Poland;Czech Republic | ||
| 217 | EUCTR2014-003746-27-GB (EUCTR) | 16/04/2015 | 14/01/2015 | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using an infusion pump or syringe | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push - Gammanorm in immunodeficient patients: quality of life using infusion by pump or rapid push | immunodeficiency syndrome MedDRA version: 19.0;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: Gammanorm 165 mg/mL | Octapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, Austria | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3;Phase 4 | Australia;Germany;United Kingdom | ||
| 218 | EUCTR2014-003746-27-DE (EUCTR) | 15/04/2015 | 01/12/2014 | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using an infusion pump or syringe | A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push - Gammanorm in immunodeficient patients: quality of life using infusion by pump or rapid push | immunodeficiency syndrome MedDRA version: 19.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: Gammanorm 165 mg/mL | Octapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, Austria | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3;Phase 4 | Australia;Germany;Italy;United Kingdom | ||
| 219 | NCT02490956 (ClinicalTrials.gov) | April 2015 | 30/3/2015 | Diagnostic Immunization With Rabies Vaccine in Patients With PID | Diagnostic Immunization With Rabies Vaccine in Patients With Primary Immunodeficiency Disorders | Primary Immunodeficiency | Biological: Verorab® (PVRV; Purified Vero Cell Vaccine) | Chulalongkorn University | NULL | Recruiting | 12 Months | 60 Years | Both | 40 | Phase 4 | Thailand |
| 220 | NCT02349906 (ClinicalTrials.gov) | April 2015 | 26/1/2015 | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases | Primary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure Syndromes | Drug: Treosulfan;Drug: Busilvex | medac GmbH | Celerion;Venn Life Sciences;Syneos Health | Active, not recruiting | N/A | 17 Years | All | 106 | Phase 2 | Czechia;Germany;Italy;Poland;Austria;Czech Republic |
| 221 | EUCTR2013-003877-87-PL (EUCTR) | 11/03/2015 | 04/12/2014 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency MedDRA version: 20.0;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland | ||
| 222 | NCT03198195 (ClinicalTrials.gov) | March 10, 2015 | 22/6/2017 | Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome | Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Procedure: cyclophosphamide | Capital Research Institute of Pediatrics | NULL | Enrolling by invitation | 5 Months | 10 Years | All | 5 | N/A | NULL |
| 223 | NCT02571062 (ClinicalTrials.gov) | March 2015 | 24/6/2015 | Bioequivalence Study - Reference Clinical Fexinidazole Tablet Versus Proposed Market Formulation | A Bioequivalence Study of the Reference Clinical Fexinidazole Tablet vs Proposed Market Formulation in Healthy Male Volunteers of African Sub-Saharan Origin:an Open-label,Randomized,Two-treatment,Single Dose,Replicate Design,Fed Condition | Trypanosomiasis, African | Drug: Fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 18 Years | 45 Years | Male | 30 | Phase 1 | France |
| 224 | NCT02972281 (ClinicalTrials.gov) | March 2015 | 21/11/2016 | Systematic Search for Primary Immunodeficiency in Adults With Infections | Systematic Search for Primary Immunodeficiency in Adults With Unexplained Recurrent and/or Severe Infections With Encapsulated Bacteria | Complement Deficiency;Antibody Deficiency;Chronic Sinus Infection;Meningitis, Bacterial;Pneumonia, Bacterial;Otitis Media;Streptococcal Infection;Neisseria Infections;Haemophilus Influenza;Pneumococcal Infections | Biological: Immunological diagnosis tests | University Hospital, Lille | Imagine Institute;Octapharma;CSL Behring;Laboratoire français de Fractionnement et de Biotechnologies;Air Liquide SA;The Binding Site Ltd | Terminated | 18 Years | 65 Years | All | 120 | N/A | France |
| 225 | NCT02909244 (ClinicalTrials.gov) | February 2015 | 5/9/2016 | Study of Gut Microbiota in Primary Immune Deficiency, Possibly Associated With Inflammatory Bowel Disease | Study of Gut Microbiota in Primary Immune Deficiencies, Possibly Associated With Inflammatory Bowel Disease (Chronic Granulomatous Disease, XIAP Gene Deficiency, or TTC7A Gene Deficiency) | Primary Immune Deficiencies | Biological: Biological sampling | Imagine Institute | Saint Antoine University Hospital;Assistance Publique - Hôpitaux de Paris | Completed | N/A | N/A | All | 51 | N/A | France |
| 226 | NCT02162420 (ClinicalTrials.gov) | January 2015 | 10/6/2014 | Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia | Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia | Dyskeratosis Congenita;Aplastic Anemia | Drug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulin | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 70 Years | All | 50 | N/A | United States |
| 227 | NCT02244450 (ClinicalTrials.gov) | December 2014 | 2/9/2014 | Generalized Neonatal Screening of Severe Combined Immunodeficiencies | Evaluation of the Clinical Utility and Cost Effectiveness Ratio of Generalized Neonatal Screening for Severe Combined Immunodeficiencies (SCID) by Quantification of TRECs on Guthrie Cards | Severe Combined Immunodeficiency, Atypical | Biological: SCID screening | Nantes University Hospital | NULL | Completed | N/A | 18 Months | All | 190539 | N/A | France |
| 228 | EUCTR2013-005508-33-PL (EUCTR) | 17/11/2014 | 08/08/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Czech Republic;Poland;Austria;Germany;Italy | ||
| 229 | EUCTR2013-005508-33-CZ (EUCTR) | 12/11/2014 | 29/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 mg, powder for solution for infusion Product Name: Ovastat 1000 INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 mg, powder for solution for infusion Product Name: Ovastat 5000 INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 230 | NCT02282904 (ClinicalTrials.gov) | October 23, 2014 | 4/11/2014 | Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | 65 Years | All | 7 | Phase 1/Phase 2 | United States |
| 231 | NCT02231879 (ClinicalTrials.gov) | October 14, 2014 | 3/9/2014 | Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome | A Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome. | Myelokathexis;Infections;Neutropenia;Warts;Hypogammaglobulinemia | Drug: Plerixafor;Drug: G-CSF | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 10 Years | 75 Years | All | 20 | Phase 2/Phase 3 | United States |
| 232 | EUCTR2013-002290-21-HU (EUCTR) | 13/10/2014 | 14/07/2014 | Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases - Study to compare Gammaplex 10% & 5% in Primary Immunodeficiency | Primary immunodeficiency diseases MedDRA version: 17.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Gammaplex Product Name: Gammaplex INN or Proposed INN: Human Normal Immunoglobulin Product Name: Gammaplex 10 INN or Proposed INN: Human Normal Immunoglobulin | Bio Products Laboratory Limited | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;Hungary;United Kingdom | ||
| 233 | EUCTR2013-005508-33-DE (EUCTR) | 29/09/2014 | 06/06/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 234 | NCT02179359 (ClinicalTrials.gov) | September 2, 2014 | 27/6/2014 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 25 | N/A | United States |
| 235 | EUCTR2013-005508-33-AT (EUCTR) | 08/08/2014 | 08/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 19.1;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 19.1;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 19.1;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft fuer klinische Spezialpräparate mbH | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 236 | NCT02498782 (ClinicalTrials.gov) | July 2014 | 20/8/2014 | Study to Evaluate Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chagas Disease | Phase 2, Randomized, Multicenter, Placebo-controlled, Safety and Efficacy Study to Evaluate Six Oral Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chronic Indeterminate Chagas Disease. | Chagas Disease;Trypanosomiasis, South American;South American Trypanosomiasis;Disease, Chagas | Drug: Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | NULL | Recruiting | 18 Years | 50 Years | Both | 140 | Phase 2 | Bolivia |
| 237 | NCT02177760 (ClinicalTrials.gov) | July 2014 | 19/6/2014 | Sirolimus Prophylaxis for aGVHD in TME SCID | Sirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT) | Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell Transplant | Drug: Sirolimus | University of California, San Francisco | NULL | Withdrawn | N/A | 1 Year | Both | 0 | Phase 2 | United States |
| 238 | EUCTR2014-000274-20-GB (EUCTR) | 14/05/2014 | 12/03/2014 | Long-term follow-up of the WAS gene therapy study | LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08) - Long-term follow-up of the WAS gene therapy study, version 2.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiencycaused by mutations in a single gene ,the Wiskott-Aldrich SyndromeProtein (WASP). WAS is characterised by micro-thrombocytopenia,recurrent infections,eczema and associated with a high incidence ofauto-immunity and of lymphoid malignancies. Over 150 uniquemutations in the WAS gene have been identified.Loss-of-functionmutations in this gene have widespread consequences on hematopoieticlineages. MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 10 | Phase 2 | United Kingdom | ||
| 239 | NCT02184689 (ClinicalTrials.gov) | May 3, 2014 | 19/6/2014 | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 6 Years | 14 Years | All | 125 | Phase 2/Phase 3 | Congo, The Democratic Republic of the |
| 240 | EUCTR2013-003257-20-GB (EUCTR) | 02/05/2014 | 12/03/2014 | TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATION | Evaluation of Treosulfan pharmacokinetics (PK) in children undergoing allogeneic haematopoietic stem cell transplantation (HSCT) - Treosulfan PK in children | Any paediatric disease with an indication to an allogeneic stem cell transplantation (inclusing leukaemia, primary immunodeficiencies, metabolic disorders and autoimmune or genetic inflammatory bowel disorders).;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Treosulfan Product Name: TREOSULFAN INN or Proposed INN: Treosulfan | GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | United Kingdom | ||
| 241 | NCT02169557 (ClinicalTrials.gov) | April 30, 2014 | 15/5/2014 | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: Fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 15 Years | N/A | All | 230 | Phase 2/Phase 3 | Congo, The Democratic Republic of the |
| 242 | EUCTR2013-002290-21-GB (EUCTR) | 30/04/2014 | 21/10/2013 | Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases - Study to compare Gammaplex 10% & 5% in Primary Immunodeficiency | Primary immunodeficiency diseases MedDRA version: 17.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Gammaplex Product Name: Gammaplex INN or Proposed INN: Human Normal Immunoglobulin Product Name: Gammaplex 10 INN or Proposed INN: Human Normal Immunoglobulin | Bio Products Laboratory Limited | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;Hungary;Israel;United Kingdom | ||
| 243 | NCT02510404 (ClinicalTrials.gov) | April 2014 | 13/7/2015 | Multivirus-specific Cytotoxic T Lymphocytes (mCTL) | Treatment of EBV, CMV, and Adenovirus Infections in Primary Immunodeficiency Disorders With Viral-specific Cytotoxic T-Lymphocytes | Refractory Viral Infections | Biological: mCTLs | Catherine Bollard | Children's National Research Institute | Completed | N/A | 45 Years | All | 1 | Phase 1 | United States |
| 244 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 18 Years | All | 193 | Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
| 245 | NCT02180763 (ClinicalTrials.gov) | April 2014 | 23/6/2014 | Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or Pumps | A Randomized, Cross-over Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL According to the Delivery Device: Injections Using Pump or Rapid Push. | Primary Immunodeficiency (PID) | Biological: Gammanorm | Octapharma | NULL | Completed | 18 Years | N/A | All | 31 | Phase 4 | France |
| 246 | EUCTR2013-003877-87-HU (EUCTR) | 14/03/2014 | 07/01/2014 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 3 | United States;Czech Republic;Hungary;Slovakia;Canada;Poland | ||
| 247 | EUCTR2013-003877-87-CZ (EUCTR) | 12/03/2014 | 19/12/2013 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1;Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland | ||
| 248 | JPRN-UMIN000013102 | 2014/03/10 | 01/03/2014 | Efficacy of Zoledronate for RAS associated ALPS like disease (RALD) | RAS associated ALPS like disease (RALD) | intravenous drip infusion of Zoledronate | Department of Pediatrics, Tokyo Medical and Dental University | NULL | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 5 | Not selected | Japan | |
| 249 | NCT01888484 (ClinicalTrials.gov) | March 2014 | 21/6/2013 | Study of Octanorm Subcutaneous IG in Patients With PID | Clinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder | Biological: octanorm 16.5% | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 75 | Phase 3 | United States;Canada;Czechia;Hungary;Poland;Russian Federation;Slovakia;Czech Republic |
| 250 | NCT01962415 (ClinicalTrials.gov) | February 4, 2014 | 10/10/2013 | Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults =55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation | Primary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory Conditions | Drug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: Thiotepa | Paul Szabolcs | NULL | Recruiting | 2 Months | 55 Years | All | 100 | Phase 2 | United States |
| 251 | NCT01814800 (ClinicalTrials.gov) | February 2014 | 6/3/2013 | Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) | An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immune Deficiency Disorder | Biological: RI-002 | ADMA Biologics, Inc. | NULL | Completed | 2 Years | 75 Years | All | 59 | Phase 3 | United States |
| 252 | NCT01963143 (ClinicalTrials.gov) | February 2014 | 13/9/2013 | Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyper-IgM Syndrome | Biological: Gammaplex (5%);Biological: Gammaplex 10 | Bio Products Laboratory | NULL | Completed | 2 Years | 55 Years | All | 48 | Phase 3 | United States;Hungary;United Kingdom |
| 253 | NCT01420627 (ClinicalTrials.gov) | January 24, 2014 | 18/8/2011 | EZN-2279 in Patients With ADA-SCID | A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immunodeficiency | ADA-SCID;Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: EZN-2279;Biological: Adagen | Leadiant Biosciences, Inc. | NULL | Completed | N/A | N/A | All | 7 | Phase 3 | United States |
| 254 | EUCTR2013-000620-34-GB (EUCTR) | 10/01/2014 | 09/08/2013 | A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes | A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes | Primary Immunofediciency (PID) syndromes MedDRA version: 14.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Human Normal Immunoglobulin for subcutaneous administration Product Code: LFB-IgSC INN or Proposed INN: Human Normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | LFB Biotechnologies | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom | ||
| 255 | NCT01917708 (ClinicalTrials.gov) | January 2014 | 24/7/2013 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases | Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell Disease | Drug: Abatacept | Emory University | NULL | Completed | N/A | 21 Years | All | 10 | Phase 1 | United States |
| 256 | EUCTR2012-000242-35-DE (EUCTR) | 23/12/2013 | 29/04/2013 | Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease (X-CGD) | A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients | X-linked Chronic Granulomatous Disease MedDRA version: 14.1;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector Product Code: G1XCGD transduced CD34+ cells Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR | GENETHON | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 1/2 | France;Germany;United Kingdom;Switzerland | ||
| 257 | NCT01998633 (ClinicalTrials.gov) | December 2013 | 29/10/2013 | Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204) | Reduced-Intensity Conditioning for Children and Adults With Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (RICHI) (BMT CTN #1204) | Hemophagocytic Lymphohistiocytosis;Chronic Active Epstein-Barr Virus Infection;Chronic Granulomatous Disease;HIGM-1;Leukocyte Adhesion Deficiency;IPEX | Biological: Hematopoietic Stem Cell Transplant | Medical College of Wisconsin | National Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program | Completed | 4 Months | 45 Years | All | 47 | Phase 2 | United States;Canada |
| 258 | NCT01985373 (ClinicalTrials.gov) | December 2013 | 31/10/2013 | Pharmacokinetics and Safety of IVIG Nanogam 100 mg/ml | Pharmacokinetics and Safety of the Intravenous Human Immunoglobulin Product Nanogam 100 mg/ml | Primary Immunodeficiency | Drug: Intravenous immunoglobulin infusion | Sanquin | NULL | Completed | 18 Years | N/A | Both | 23 | Phase 3 | Netherlands |
| 259 | EUCTR2013-000961-36-DE (EUCTR) | 15/11/2013 | 05/08/2013 | See the full title | Tolerability and safety evaluation of the administration of Ig VENA at high infusion rates. Open label phase III study. - Ig VENA Infusion Speed | Patients affected by primary or secondary immunodeficiency (ID) or patients affected by Primary Immune Thrombocytopenia (ITP) MedDRA version: 16.1;Level: PT;Classification code 10054979;Term: Secondary immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 16.1;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1;Classification code 10021245;Term: Idiopathic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ig VENA 50 g/l solution for infusion 100 ml vial + infusion set | Kedrion SpA | NULL | Not Recruiting | Female: yes Male: yes | 35 | Phase 3 | Germany;Italy | ||
| 260 | NCT02054832 (ClinicalTrials.gov) | November 2013 | 31/1/2014 | Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch | A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch | Glycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0 | Dietary Supplement: Glycosade | John Mitchell | NULL | Completed | 2 Years | 50 Years | Both | 11 | N/A | Canada |
| 261 | EUCTR2013-000620-34-DE (EUCTR) | 07/10/2013 | 05/08/2013 | A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes | A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes | Primary Immunofediciency (PID) syndromes MedDRA version: 14.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Human Normal Immunoglobulin for subcutaneous administration Product Code: LFB-IgSC INN or Proposed INN: Human Normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | LFB Biotechnologies | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom | ||
| 262 | NCT01946906 (ClinicalTrials.gov) | October 2013 | 12/9/2013 | The Rifaximin Study in CVID | Effects of Rifaximin, by Modulation of the Gut Microbiota, on Markers of Systemic Inflammation in Patients With Common Variable Immunodeficiency - An Exploratory Open-label Randomized Controlled Trial | Common Variable Immunodeficiency (CVID) | Drug: Rifaximin | Oslo University Hospital | NULL | Completed | 18 Years | 74 Years | Both | 40 | Phase 4 | Norway |
| 263 | EUCTR2013-000620-34-HU (EUCTR) | 19/09/2013 | 01/08/2013 | A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes | A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes | Primary Immunofediciency (PID) syndromes MedDRA version: 16.0;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Human Normal Immunoglobulin for subcutaneous administration Product Code: LFB-IgSC INN or Proposed INN: Human Normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | LFB Biotechnologies | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom | ||
| 264 | EUCTR2013-000883-27-NO (EUCTR) | 16/08/2013 | 20/06/2013 | The effects of the local acting antibiotic Rifaximin on markers of inflammation in the blood by changing the bacterial composition in the gut, in patients with common variable immunodeficiency. | EFFECTS OF RIFAXIMIN, BY MODULATION OF THE GUT MICROBIOTA, ON MARKERS OF SYSTEMIC INFLAMMATION IN PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY - AN EXPLORATORY OPEN-LABEL RANDOMIZED CONTROLLED TRIAL | Adult patients with the diagnosis of Common variable deficiency and fulfill the inclusion and exclusion criteria, will be invited to participate in the study.;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Xifaxan | Oslo University hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Norway | ||||
| 265 | EUCTR2013-000620-34-IT (EUCTR) | 05/08/2013 | 30/07/2013 | A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes | A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes - NA | Primary Immunofediciency (PID) syndromes MedDRA version: 14.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Human Normal Immunoglobulin for subcutaneous administration Product Code: LFB-IgSC INN or Proposed INN: Human Normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | LFB Biotechnologies | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;Hungary;Poland;Ukraine;Germany;United Kingdom;Italy | ||
| 266 | NCT01852071 (ClinicalTrials.gov) | August 2, 2013 | 7/5/2013 | Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene | Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | ADA-SCID | Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles | Completed | 1 Month | 17 Years | All | 20 | Phase 1/Phase 2 | United States |
| 267 | EUCTR2012-005727-32-NL (EUCTR) | 25/07/2013 | 23/05/2013 | The pharmacokinetics (the behaviour in the body) and safety of the intravenous immunoglobulin product Nanogam 100 mg/ml | Pharmacokinetics and safety of the intravenous human immunoglobulin product Nanogam 100 mg/ml - PK and safety of Nanogam 100 mg/ml | Primary a- or hypogammaglobulinemia MedDRA version: 14.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 100000004870 MedDRA version: 14.1;Classification code 10010509;Term: Congenital hypogammaglobulinemia;System Organ Class: 100000004850 MedDRA version: 14.1;Classification code 10071133;Term: Congenital agammaglobulinemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Nanogam 100 mg/ml INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: IVIG Trade Name: Nanogam® 50 mg/ml INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV) Other descriptive name: IVIG | Sanquin Blood Supply Foundation | NULL | Not Recruiting | Female: yes Male: yes | Netherlands | ||||
| 268 | EUCTR2011-004154-25-IT (EUCTR) | 25/06/2013 | 20/02/2013 | Clinical study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiency | A prospective, open-label, phase I/II study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiency | Patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia. MedDRA version: 14.1;Level: PT;Classification code 10016075;Term: Factor I deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Fibrinogen Concentrate from Human Plasma Product Code: BT524 INN or Proposed INN: Human Fibrinogen Concentrate Other descriptive name: Human Fibrinogen Concentrate | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 1;Phase 2 | Egypt;Lebanon;Germany;Italy | ||
| 269 | NCT01852370 (ClinicalTrials.gov) | June 20, 2013 | 26/4/2013 | Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric Donors | Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric Donors | Severe Combined Immunodeficiency (SCID);Immunodeficiency With Predominant T-cell Defect, Unspecified;Severe Chronic Neutropenia;Chronic Granulomatous Disease (CGD);Hyper IgE Syndromes;Hyper IgM Deficiencies;Wiskott-Aldrich Syndrome;Mendelian Susceptibility to Mycobacterial Disease;Common Variable Immune Deficiency (CVID) | Biological: CD3/CD19 negative allogeneic hematopoietic stem cells | Paul Szabolcs | NULL | Enrolling by invitation | 5 Years | 45 Years | All | 16 | Phase 1/Phase 2 | United States |
| 270 | EUCTR2010-023483-41-FR (EUCTR) | 24/04/2013 | 04/01/2011 | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | a primary immunodeficiency as defined by the ESID and validated by a reference centre :• X-linked agammaglobulinemia (XLA)• Autosomal recessive inherited agammaglobulinemia (including autosomal recessive hyper-IgM syndrome)• Common variable immunodeficiency (CVID)• IgG subclass deficiency (at least 2 subclasses) with clinical manifestations of immunodeficiency MedDRA version: 12.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Hungary | ||
| 271 | EUCTR2012-001725-26-DE (EUCTR) | 12/03/2013 | 18/12/2012 | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector - gene therapy for X-CGD | Chronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to kill phagocytized bacteria or fungi. MedDRA version: 14.1;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: genetically modified autologous blood stem cells Product Code: somatic gene-therapy by X-CGD INN or Proposed INN: G1XCG Other descriptive name: Genetically modified autologous blood stem cells | Johann Wolfgang Goethe-University | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Germany | ||
| 272 | NCT01966367 (ClinicalTrials.gov) | March 2013 | 17/10/2013 | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant Disease | Bone Marrow Failure Syndrome;Severe Aplastic Anemia;Severe Congenital Neutropenia;Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Schwachman Diamond Syndrome;Primary Immunodeficiency Syndromes;Acquired Immunodeficiency Syndromes;Histiocytic Syndrome;Familial Hemophagocytic Lymphocytosis;Lymphohistiocytosis;Macrophage Activation Syndrome;Langerhans Cell Histiocytosis (LCH);Hemoglobinopathies;Sickle Cell Disease;Sickle Cell-beta-thalassemia | Biological: CD34 Stem Cell Selection Therapy | Diane George | NULL | Active, not recruiting | N/A | 40 Years | All | 37 | Phase 1/Phase 2 | United States |
| 273 | NCT01821781 (ClinicalTrials.gov) | March 2013 | 19/3/2013 | Immune Disorder HSCT Protocol | A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime | Immune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative Syndrome | Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Washington University School of Medicine | NULL | Recruiting | N/A | 21 Years | All | 20 | Phase 2 | United States |
| 274 | EUCTR2012-000242-35-GB (EUCTR) | 10/01/2013 | 19/07/2012 | Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease | A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients withX-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients | X-linked Chronic Granulomatous Disease MedDRA version: 19.0;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector Product Code: G1XCGD transduced CD34+ cells Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 11 | Phase 1;Phase 2 | France;Germany;Switzerland;United Kingdom | ||
| 275 | NCT01218438 (ClinicalTrials.gov) | January 2013 | 8/10/2010 | Phase 2/3 Study of IGSC, 20% in PIDD | A Clinical Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) for the Evaluation of Efficacy, Safety, Tolerability and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Intravenous (Human), 10% Solution;Drug: Immune Globulin Subcutaneous (Human), 20% Solution | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 86 | Phase 2/Phase 3 | United States;Canada |
| 276 | JPRN-UMIN000009370 | 2012/12/07 | 21/11/2012 | Thalidomide therapy clinical trial for chronic granulomatous disease-associated intractable granulomatous lesion | Chronic granulomatous disease | Oral administration of thalidomide | National Center for Child Heath and Development | NULL | Complete: follow-up complete | 3years-old | Not applicable | Male and Female | 5 | Not selected | Japan | |
| 277 | NCT01380990 (ClinicalTrials.gov) | November 15, 2012 | 23/6/2011 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID) | Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | N/A | 15 Years | All | 36 | Phase 1/Phase 2 | United Kingdom |
| 278 | NCT01581593 (ClinicalTrials.gov) | November 2012 | 16/4/2012 | Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID) | Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID). | Primary Immunodeficiency;Agammaglobulinemia;Hypogammaglobulinemia;Antibody Deficiency | Biological: Kedrion IVIG 10% | Kedrion S.p.A. | NULL | Active, not recruiting | 2 Years | 70 Years | Both | 50 | Phase 3 | United States;Canada |
| 279 | NCT01685827 (ClinicalTrials.gov) | October 2012 | 12/9/2012 | Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2 | Efficacy and Safety of Fexinidazole Compared to Nifurtimox-Eflornithine Combination Therapy (NECT) in Patients With Late-stage Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: Pivotal, Non-inferiority, Multicentre, Randomised, Open-label Study | Human African Trypanosomiasis (HAT);Sleeping Sickness | Drug: Fexinidazole;Drug: Nifurtimox;Drug: Eflornithine | Drugs for Neglected Diseases | NULL | Completed | 15 Years | N/A | All | 394 | Phase 2/Phase 3 | Central African Republic;Congo, The Democratic Republic of the;Congo |
| 280 | NCT01306019 (ClinicalTrials.gov) | September 25, 2012 | 26/2/2011 | Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-linked Severe Combined Immunodeficiency (XSCID) | Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | Male | 30 | Phase 1/Phase 2 | United States |
| 281 | NCT01652092 (ClinicalTrials.gov) | September 4, 2012 | 25/7/2012 | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | SCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell Histiocytosis | Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNA | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 50 Years | All | 30 | N/A | United States |
| 282 | EUCTR2012-000792-16-DE (EUCTR) | 03/09/2012 | 10/05/2012 | Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseases | CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES” | primary immunodeficiency disease (PID) MedDRA version: 14.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 10021428 - Immune system disorders MedDRA version: 14.1;Classification code 10049485;Term: Bruton's agammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Octagam 5% Product Name: Octagam 5% Other descriptive name: IMMUNOGLOBULIN G | OCTAPHARMA AG | NULL | Not Recruiting | Female: yes Male: yes | 23 | Czech Republic;Hungary;Germany | |||
| 283 | EUCTR2010-024253-36-GB (EUCTR) | 03/08/2012 | 09/05/2012 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1aS-ADA lentiviral vector mediated gene modification of autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced patient CD34+ cells INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 284 | NCT01659606 (ClinicalTrials.gov) | July 2012 | 6/8/2012 | Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita | Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease | Dyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic Anemia | Biological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetil | Boston Children's Hospital | Dana-Farber Cancer Institute;Children's Hospital Medical Center, Cincinnati;Children's Hospital Los Angeles;Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium;Baylor College of Medicine;Children's Hospital of Philadelphia;University of Wisconsin, Madison;Karolinska University Hospital;Hackensack Meridian Health;Duke University;Oslo University Hospital;Children's Mercy Hospital Kansas City;Mayo Clinic;University of Chicago;Massachusetts General Hospital | Recruiting | N/A | 65 Years | All | 40 | Phase 2 | United States;Norway;Sweden |
| 285 | NCT02327351 (ClinicalTrials.gov) | July 2012 | 18/12/2014 | TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID | Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder;Hematopoietic Stem Cell Transplantation | Other: Biological: TCR alfa beta T cell depletion | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 1 Month | 18 Years | Both | 60 | Phase 2/Phase 3 | Russian Federation |
| 286 | EUCTR2012-000481-38-GB (EUCTR) | 28/06/2012 | 26/04/2012 | Assessment of the safety of Immunoglobulin and recombinant human hylaluronidase in the treatment of patients with primary immunodeficiency | Tolerability, Safety and Product Administration Evaluation of rHuPH20 Facilitated Subcutaneous Treatment with Immune Globulin (Human), 10% in Subjects with Primary Immunodeficiency Diseases – A Study in Europe - Tolerability and Safety of IG, 10% with rHuPH20 in PIDD | Primary Immunodeficiency Diseases MedDRA version: 14.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Recombinant Human Hyaluronidase (rHuPH20) INN or Proposed INN: HYALURONIDASE Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20) Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: Human normal immunoglobulin INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Recombinant Human Hyaluronidase (rHuPH20) INN or Proposed INN: HYALURONIDASE Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20) | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 40 | Czech Republic;Belgium;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden | |||
| 287 | EUCTR2012-000792-16-HU (EUCTR) | 14/06/2012 | 20/03/2012 | Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseases | CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES” | primary immunodeficiency disease (PID) MedDRA version: 14.1;Level: LLT;Classification code 10049485;Term: Bruton's agammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: OCTAGAM 50 mg/ml oldatos infúzió Product Name: Octagam 5% Other descriptive name: IMMUNOGLOBULIN G | OCTAPHARMA AG | NULL | Not Recruiting | Female: yes Male: yes | 23 | Czech Republic;Hungary;Germany | |||
| 288 | EUCTR2012-000792-16-CZ (EUCTR) | 29/05/2012 | 27/03/2012 | Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseases | CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES” | primary immunodeficiency disease (PID) MedDRA version: 14.1;Level: LLT;Classification code 10049485;Term: Bruton's agammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: OCTAGAM Product Name: Octagam 5% Other descriptive name: IMMUNOGLOBULIN G | OCTAPHARMA AG | NULL | Not Recruiting | Female: yes Male: yes | 23 | Hungary;Czech Republic;Germany | |||
| 289 | NCT04350164 (ClinicalTrials.gov) | April 1, 2012 | 10/4/2020 | Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome. | Retrospective Chart Review of Children With Wiskott-Aldrich Syndrome Who Received Romiplostim in Treatment of Thrombocytopenia. | Wiskott-Aldrich Syndrome | Drug: Romiplostim | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Active, not recruiting | N/A | 18 Years | All | 67 | Russian Federation | |
| 290 | NCT03315078 (ClinicalTrials.gov) | April 2012 | 16/10/2017 | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Combined Immunodeficiency Diseases | Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | All | 13 | Phase 1/Phase 2 | United States |
| 291 | EUCTR2010-019459-23-BE (EUCTR) | 29/03/2012 | 14/02/2012 | CLINICAL STUDY OF THE EFFECTIVENESS AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Phase 2;Phase 3 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | ||
| 292 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 293 | EUCTR2011-001118-32-SE (EUCTR) | 03/02/2012 | 06/12/2011 | Twelve-month study on the immunogenicity (i.e. the ability of the study medication to provoke an immune response), safety and efficacy of the study medication Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia) MedDRA version: 18.0;Level: PT;Classification code 10051645;Term: Idiopathic neutropenia;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 18.0;Classification code 10053176;Term: Cyclic neutropenia;Level: LLT;Classification code 10069819;Term: Congenital neutropenia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Zarzio INN or Proposed INN: FILGRASTIM Trade Name: Zarzio INN or Proposed INN: FILGRASTIM | Sandoz GmbH | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | Germany;Sweden | ||
| 294 | NCT01533961 (ClinicalTrials.gov) | February 2012 | 13/2/2012 | Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the SCYX-7158 | Randomized, Double-blind, Placebo-controlled Sequential Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SCYX-7158 After Single Oral Ascending Doses in Healthy Male Volunteers | Trypanosomiasis;Trypanosomiasis, African;Protozoan Infections;Parasitic Diseases | Drug: SCYX-7158;Drug: Placebo | Drugs for Neglected Diseases | NULL | Completed | 18 Years | 45 Years | Male | 136 | Phase 1 | France |
| 295 | NCT03333200 (ClinicalTrials.gov) | January 11, 2012 | 24/4/2017 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | NULL | Recruiting | N/A | N/A | All | 1500 | United States | |
| 296 | NCT03354533 (ClinicalTrials.gov) | January 1, 2012 | 21/11/2017 | Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II | Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II | Leukocyte Adhesion Deficiency, Type II | Drug: L-fucose | Orpha Labs | NULL | Completed | N/A | 18 Years | All | 4 | Phase 1/Phase 2 | NULL |
| 297 | NCT01485796 (ClinicalTrials.gov) | December 29, 2011 | 2/12/2011 | Tolerability and Safety of IGI, 10% With rHuPH20 in PIDD | Tolerability, Safety and Administration Mode Evaluation of Recombinant Human Hyaluronidase (rHuPH20) Facilitated Subcutaneous Treatment With Immune Globulin Infusion (Human), 10% in Subjects With Primary Immunodeficiency Diseases (PIDD) | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Infusion (Human), 10%;Biological: Recombinant human hyaluronidase | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 54 | Phase 2/Phase 3 | United States |
| 298 | NCT01465958 (ClinicalTrials.gov) | November 2011 | 24/10/2011 | Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency | An Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary Immunodeficiency | Primary Immunodeficiency | Biological: GAMUNEX-C | Grifols Therapeutics Inc. | NULL | Completed | 2 Years | 16 Years | All | 12 | Phase 4 | United States |
| 299 | NCT01461018 (ClinicalTrials.gov) | October 2011 | 24/10/2011 | Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study) | A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency | Biological: Immune globulin subcutaneous (Human) | CSL Behring | NULL | Completed | N/A | 75 Years | Both | 22 | Phase 3 | Japan |
| 300 | NCT01406470 (ClinicalTrials.gov) | September 2011 | 26/7/2011 | Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency | An Open-Label, Single-Arm, Historically Controlled, Prospective, Multicenter Phase III Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Immune Globulin Intravenous (Human) IVIG-SN™ in Subjects With Primary Immunodeficiency | Immunologic Deficiency Syndrome | Drug: Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™ | Green Cross Corporation | Atlantic Research Group | Completed | 2 Years | 70 Years | Both | 45 | Phase 3 | United States;Canada |
| 301 | NCT01483170 (ClinicalTrials.gov) | September 2011 | 29/11/2011 | Multiple Dose Study to Evaluate Security, Tolerance and Pharmacokinetic of Fexinidazole (Drug Candidate for Human African Trypanosomiasis) Administered With a Loading Dose and With Food | Double-blind, Placebo Controlled, Randomized Multiple Ascending Dose Study in Fed Conditions for Ten Days Dosing Regimen With a Loading Dose to Evaluate the Safety, the Tolerability and the Pharmacokinetics of Oral Fexinidazole in 36 Healthy Male Sub-Saharan Volunteers. | Trypanosomiasis, African | Drug: Tablets Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | NULL | Terminated | 18 Years | 45 Years | Male | 30 | Phase 1 | France |
| 302 | EUCTR2010-019459-23-NL (EUCTR) | 24/08/2011 | 27/01/2011 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Phase 2;Phase 3 | Hungary;Belgium;Austria;Germany;Netherlands;United Kingdom;Sweden | ||
| 303 | NCT01410825 (ClinicalTrials.gov) | July 2011 | 4/8/2011 | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Biological: Retrovirus-mediated gene transfer | David Williams | NULL | Active, not recruiting | 3 Months | 35 Years | Male | 5 | Phase 1/Phase 2 | United States |
| 304 | NCT01412385 (ClinicalTrials.gov) | June 20, 2011 | 8/8/2011 | Immune Globulin Subcutaenous (Human), 20% | A Clinical Study of Immune Globulin Subcutaneous (Human) (IGSC), 20% for the Evaluation of Efficacy, Safety, and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: Immune Globulin Subcutaneous (Human), 20%;Biological: Immune Globulin Intravenous (Human), 10%;Biological: Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin) | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 55 | Phase 2/Phase 3 | Austria;Germany;Hungary;Sweden;United Kingdom |
| 305 | EUCTR2011-001118-32-DE (EUCTR) | 25/05/2011 | 27/04/2011 | Twelve-month study on the immunogenicity (i.e. the ability of the study medication to provoke an immune response), safety and efficacy of the study medication Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia | Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia) MedDRA version: 14.1;Level: PT;Classification code 10051645;Term: Idiopathic neutropenia;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 14.1;Level: LLT;Classification code 10069819;Term: Congenital neutropenia;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10053176;Term: Cyclic neutropenia;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Filgrastim Hexal INN or Proposed INN: FILGRASTIM Trade Name: Filgrastim Hexal INN or Proposed INN: FILGRASTIM Product Name: EP2006 (Filgrastim) Product Code: EP2006 INN or Proposed INN: FILGRASTIM | Sandoz GmbH | NULL | Not Recruiting | Female: yes Male: yes | 40 | Germany;Sweden | |||
| 306 | NCT01313507 (ClinicalTrials.gov) | May 2011 | 9/3/2011 | High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam) | Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01) | Primary Immunodeficiency Disease | Biological: NewGam | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 21 | Phase 3 | United States |
| 307 | EUCTR2007-004088-22-DE (EUCTR) | 14/04/2011 | 27/07/2010 | Efficacy and safety of Fibrinogen Concentrate (Human) for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiencyFibrinogen Concentrate (Human) (FCH) - efficacy and safety | Efficacy and safety of Fibrinogen Concentrate (Human) for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiencyFibrinogen Concentrate (Human) (FCH) - efficacy and safety | Congenital fibrinogen deficiency (afibrinogenemia, severe hypofibrinogenemia) MedDRA version: 12.0;Level: LLT;Classification code 10016075;Term: Factor I deficiency | Trade Name: Haemocomplettan(R) P 1g/2g Product Name: Haemocomplettan(R) P INN or Proposed INN: Fibrinogen (coagulation factorI) Other descriptive name: HUMAN FIBRINOGEN | CSL Behring GmbH | NULL | Not Recruiting | Female: yes Male: yes | 23 | Germany;Italy | |||
| 308 | NCT01458171 (ClinicalTrials.gov) | April 2011 | 12/10/2011 | Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency Disorder | Biological: Immune globulin subcutaneous (Human) | CSL Behring | NULL | Completed | N/A | 75 Years | All | 23 | Phase 3 | Japan |
| 309 | EUCTR2010-019459-23-SE (EUCTR) | 23/03/2011 | 25/01/2011 | CLINICAL STUDY OF THE EFFECTIVENESS AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | |||
| 310 | EUCTR2010-023483-41-HU (EUCTR) | 01/03/2011 | 10/11/2010 | A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune system | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) - I10E-0718 | a primary immunodeficiency as defined by the ESID and validated by a reference centre :• X-linked agammaglobulinemia (XLA)• Common variable immunodeficiency (CVID) MedDRA version: 14.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 100000004870 MedDRA version: 14.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;Classification code 10001471;Term: Agammaglobulinemia;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | Not Recruiting | Female: yes Male: yes | 60 | Serbia;France;Czech Republic;Hungary;Poland;Ukraine;Lithuania | |||
| 311 | NCT01289847 (ClinicalTrials.gov) | March 2011 | 27/1/2011 | A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency | A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Hyper-IgM Syndrome;Wiskott-Aldrich Syndrome | Biological: Gammaplex | Bio Products Laboratory | NULL | Completed | 2 Years | 16 Years | All | 25 | Phase 4 | United States;Chile;Israel |
| 312 | EUCTR2010-019459-23-DE (EUCTR) | 18/01/2011 | 28/07/2010 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneos, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | |||
| 313 | NCT01338675 (ClinicalTrials.gov) | January 2011 | 4/3/2011 | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) | Chronic Granulomatous Disease | Drug: Busulfan | Seoul National University Hospital | NULL | Recruiting | N/A | N/A | Both | 5 | Phase 1/Phase 2 | Korea, Republic of | |
| 314 | EUCTR2010-019459-23-GB (EUCTR) | 21/12/2010 | 05/10/2010 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 14.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | |||
| 315 | EUCTR2010-019459-23-HU (EUCTR) | 20/12/2010 | 20/10/2010 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 16.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | |||
| 316 | NCT01220531 (ClinicalTrials.gov) | November 8, 2010 | 22/9/2010 | Thymus Transplantation Safety-Efficacy | Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836 | Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Cultured Thymus Tissue;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH | Available | N/A | N/A | All | United States | ||
| 317 | EUCTR2010-019459-23-AT (EUCTR) | 05/11/2010 | 02/08/2010 | CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | A CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PID | Primary Immunodeficiency Diseases MedDRA version: 14.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: KIOVIG Product Name: KIOVIG Product Code: IGIV, 10% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: SUBCUVIA Product Name: SUBCUVIA Product Code: IGSC, 16% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Immune Globulin Subcutaneous, 20% Product Code: IGSC, 20% INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxter Innovations GmbH | NULL | Not Recruiting | Female: yes Male: yes | 47 | Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden | |||
| 318 | NCT01856582 (ClinicalTrials.gov) | October 2010 | 15/5/2013 | CD34+ Stem Cell Infusion to Augment Graft Function | Post Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure Syndromes | Waning Donor Chimerism;Waning Immune Function;Primary Immunodeficiency Disease(s);Bone Marrow Failure | Biological: CD34+ | Children's Hospital Medical Center, Cincinnati | Hoxworth Blood Center | Terminated | N/A | 35 Years | All | 23 | Phase 2 | United States |
| 319 | NCT01354587 (ClinicalTrials.gov) | October 2010 | 18/3/2011 | Evaluation of Efficacy and Tolerability of Hizentra® | Evaluation of Efficacy and Tolerability of Hizentra® in Subjects Transitioning From Vivaglobin® (16% SCIG Product) to Hizentra® (20% SCIG Product) | Primary Immunodeficiency Disorders | Drug: Hizentra | University of South Florida | NULL | Recruiting | 1 Year | 75 Years | Both | 50 | N/A | United States |
| 320 | EUCTR2010-019249-25-HU (EUCTR) | 22/09/2010 | 06/08/2010 | Eine offene, prospektive Studie zur Untersuchung der Pharmakokinetikund Sicherheit (Teil A) des Immunglobulins vom Menschen zurintravenösen Infusion (IVIG) BT090 sowie der Verträglichkeit undSicherheit von steigenden Infusionsgeschwindigkeiten (Teil B) beiPatienten mit primären Immundefekten (PID) | An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID) | Primary immunodeficiency disease (PID) MedDRA version: 13.1;Level: LLT;Classification code 10010509;Term: Congenital hypogammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Intratect Product Code: BT090 Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 30 | Hungary;Germany | |||
| 321 | NCT01319851 (ClinicalTrials.gov) | September 2010 | 15/9/2010 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Terminated | N/A | 21 Years | All | 3 | N/A | United States |
| 322 | NCT01199705 (ClinicalTrials.gov) | September 2010 | 8/9/2010 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency | Biological: Immune Globulin Subcutaneous (Human) (SCIG) | CSL Behring | NULL | Completed | N/A | 75 Years | All | 25 | Phase 3 | Japan |
| 323 | EUCTR2010-019249-25-DE (EUCTR) | 12/08/2010 | 14/05/2010 | An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID) | An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID) | Primary immunodeficiency disease (PID) MedDRA version: 14.1;Level: LLT;Classification code 10010509;Term: Congenital hypogammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Intratect Product Code: BT090 Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 30 | Hungary;Germany | |||
| 324 | NCT01182675 (ClinicalTrials.gov) | August 2010 | 9/8/2010 | Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim | Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim | Severe Combined Immunodeficiency | Drug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and Alemtuzumab | University of California, San Francisco | NULL | Terminated | N/A | 3 Years | All | 7 | Phase 2 | United States |
| 325 | NCT01175213 (ClinicalTrials.gov) | July 28, 2010 | 3/8/2010 | Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID | Long-Term Tolerability and Safety of Immune Globulin Subcutaneous (IGSC) Solution Administered Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety);Biological: SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety);Biological: IV treatment with IGSC, 10% | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 66 | Phase 3 | United States |
| 326 | NCT01147042 (ClinicalTrials.gov) | May 18, 2010 | 17/6/2010 | Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease | Assessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease | IFN-Gamma Therapy;CGD Gene Mutation;CGD Response to IFNg;CGD - Chronic Granulomatous Disease;Immunodeficiency Disease | Drug: IFN-gamma | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | N/A | N/A | All | 2 | Phase 4 | United States |
| 327 | NCT03534479 (ClinicalTrials.gov) | April 2010 | 10/5/2018 | Human IgGs and Endothelial Function in Vivo in Humans | Effects of Intravenous Human Polyclonal Immunoglobulins G Infusion on Endothelial Function and Insulin Sensitivity in Humans | Common Variable Immunodeficiency | Drug: Polyclonal IgG | Federico II University | NULL | Completed | 18 Years | 70 Years | All | 24 | N/A | Italy |
| 328 | NCT01129544 (ClinicalTrials.gov) | April 2010 | 21/5/2010 | Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector | Multi-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene Transfer | Severe Combined Immunodeficiency | Biological: Gene transfer | David Williams | Boston Children's Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los Angeles | Active, not recruiting | N/A | N/A | Male | 8 | Phase 1/Phase 2 | United States |
| 329 | EUCTR2009-017346-32-IT (EUCTR) | 15/03/2010 | 26/04/2010 | HAEMATOPOIETIC STEM CELL GENE THERAPY | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS | Wiskott-Aldrich Syndrom MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML Product Name: na Product Code: [na] INN or Proposed INN: FLUDARABINA Other descriptive name: FLUDARABINA Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO Product Name: PLERIXAFOR Product Code: [na] INN or Proposed INN: PLERIXAFOR Other descriptive name: Plerixafor Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML Product Name: na Product Code: [na] INN or Proposed INN: LENOGRASTIM Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML Product Name: RITUXIMAB Product Code: [na] INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI Product Name: na Product Code: [na] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 8 | Phase 1;Phase 2 | Italy | ||
| 330 | EUCTR2009-011434-10-DE (EUCTR) | 02/03/2010 | 27/10/2009 | CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency diseases MedDRA version: 12.0;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency MedDRA version: 12.0;Classification code 10049485;Term: Bruton's agammaglobulinemia | Product Name: NewGam Product Code: NewGam INN or Proposed INN: immunoglobulin G Other descriptive name: NewGam | OCTAPHARMA AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Germany | |||
| 331 | NCT01131858 (ClinicalTrials.gov) | March 2010 | 26/5/2010 | Study of Vitamin D3 Substitution to Patients With Primary Immunodeficiency | A Placebo Controlled Double Blinded Study of Vitamin D3 Substitution to Patients With Primary Immunodeficiency | Primary Immune Deficiency Disorder | Drug: Vigantol;Drug: Placebo | Karolinska University Hospital | NULL | Completed | 18 Years | 75 Years | Both | 140 | Phase 1/Phase 2 | Sweden |
| 332 | NCT01075438 (ClinicalTrials.gov) | March 2010 | 24/2/2010 | Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia Patients | Descriptive Immunogenicity of 2 Doses of Pneumococcal 7-valent Conjugate Vaccine (Prevenar®, Wyeth Lederle) Followed by Pneumococcal Polysaccharide Vaccine (Pneumovax® Aventis Pasteur MSD) in Ataxia-telangiectasia Patients | Ataxia Telangiectasia | Biological: Prevenar® (7-valent pneumococcal conjugate vaccine);Biological: Pneumovax® (pneumococcal polysaccharide vaccine) | Sheba Medical Center | NULL | Not yet recruiting | 2 Years | 25 Years | Both | 20 | N/A | Israel |
| 333 | EUCTR2007-002611-27-GB (EUCTR) | 19/02/2010 | 15/03/2010 | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Primary Immundeficiency Diseases (PID) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Trade Name: Octagam 10% Product Name: Octagam 10% | Octapharma AG | NULL | Not Recruiting | Female: yes Male: yes | 45 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Germany;United Kingdom | ||
| 334 | EUCTR2007-002611-27-FR (EUCTR) | 17/02/2010 | 26/11/2009 | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Primary Immunodeficiency Diseases (PID) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Trade Name: Octagam 10% Product Name: Octagam 10% INN or Proposed INN: Human Normal Immunoglobulin | Octapharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | United Kingdom;France | |||
| 335 | EUCTR2007-000684-16-GB (EUCTR) | 21/01/2010 | 17/09/2009 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | X-Linked severe combined Immunodeficiency (SCID-X1) MedDRA version: 9.1;Level: LLT;Classification code 10010099;Term: Combined immunodeficiency | Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector transduced cells Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce | Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 336 | EUCTR2007-004308-11-GB (EUCTR) | 11/01/2010 | 06/07/2009 | Gene therapy for WAS | PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPYFOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages. MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | Phase 1;Phase 2 | United Kingdom | |||
| 337 | NCT01012323 (ClinicalTrials.gov) | January 2010 | 11/11/2009 | A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases | Clinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases | Biological: NewGam | Octapharma | Premier Research Group plc | Completed | 2 Years | 75 Years | All | 51 | Phase 3 | United States |
| 338 | NCT01052623 (ClinicalTrials.gov) | January 2010 | 19/1/2010 | Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT) | Status of the Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis in Relation to Growth Failure, Body Weight and Neuroprotection in Children With Ataxia Telangiectasia | Ataxia Telangiectasia;Growth Failure | Drug: Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate | Johann Wolfgang Goethe University Hospitals | NULL | Recruiting | 3 Years | 18 Years | Both | 24 | Phase 4 | Germany |
| 339 | EUCTR2009-015739-34-DE (EUCTR) | 09/12/2009 | 14/09/2009 | Status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and neuroprotection in children with Ataxia telangiectasia (AT) - Growth hormone in AT (GHAT) | Status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and neuroprotection in children with Ataxia telangiectasia (AT) - Growth hormone in AT (GHAT) | This study will evaluate the status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and composition and neuroprotection in children with Ataxia telangiectasia (AT) | Trade Name: Catapresan 75, Tabletten INN or Proposed INN: Clonidinhydrochlorid Trade Name: L-Arginin-hydrochlorid-einmolar Fresenius INN or Proposed INN: Argininhydrochlorid Trade Name: Progynova 21 mite INN or Proposed INN: Estradiolvalerat Trade Name: NutropinAq 10mg/2ml (30 I.E.) Injektionsllösung INN or Proposed INN: Somatotropin | Goethe-Universität Frankfurt/Main | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Germany | ||||
| 340 | EUCTR2008-004518-28-GB (EUCTR) | 08/12/2009 | 09/11/2009 | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Children with chronic granulomatous disease (CGD). MedDRA version: 9.1;Level: LLT;Classification code 10008906;Term: Chronic granulomatous disease | Trade Name: Noxafil 40 mg/ml oral solution Product Name: noxafil INN or Proposed INN: POSACONAZOLE | Radboud University Nijmegen Medical Centre | NULL | Not Recruiting | Female: yes Male: yes | 20 | United Kingdom;Netherlands | |||
| 341 | NCT01001598 (ClinicalTrials.gov) | November 2009 | 22/10/2009 | Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Phase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Fanconi Anemia;Dyskeratosis Congenita | Drug: danazol | Boston Children's Hospital | NULL | Terminated | 3 Years | N/A | All | 5 | Phase 1/Phase 2 | United States |
| 342 | NCT00982904 (ClinicalTrials.gov) | September 2009 | 22/9/2009 | Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the Fexinidazole | Randomized, Double-blind, Placebo-controlled Study of the Tolerability, and Pharmacokinetics of Fexinidazole After Single and Repeated Oral Ascending Doses, Completed by a Comparative Bioavailability Study of an Oral Suspension Versus a Tablet and an Exploratory Assessment of Food Effect, in Healthy Male Volunteers | Human African Trypanosomiasis | Drug: Fexinidazole/Placebo | Drugs for Neglected Diseases | Sanofi | Completed | 18 Years | 45 Years | Male | 108 | Phase 1 | France |
| 343 | EUCTR2009-012036-32-FR (EUCTR) | 30/07/2009 | 28/05/2009 | SAFETY STUDY OF IGNG,A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATE | SAFETY STUDY OF IGNG,A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATE | PRIMARY IMMUNODEFICIENCY MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE Product Code: IGNG INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 344 | NCT01489618 (ClinicalTrials.gov) | June 2009 | 8/12/2011 | Prime Boost Vaccination Strategy Combining Conjugated Anti- Pneumococcal Vaccine (s0) and Polysaccharide Anti- Pneumococcal Vaccine (s4) Compared to Polysaccharide Anti- Pneumococcal Vaccine Alone (s4) In Patients With Common Variable Immunodeficiency | Randomised, Multicentric, Phase ii Study of the Immunogenicity of a Prime Boost Vaccination Strategy Combining Conjugated Anti- Pneumococcal Vaccine (s0) and Polysaccharide Anti- Pneumococcal Vaccine (s4) Compared to Polysaccharide Anti- Pneumococcal Vaccine Alone (s4) In Patients With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Biological: PPS;Biological: PnCJ PPS | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Terminated | 18 Years | 65 Years | Both | 45 | Phase 2 | France |
| 345 | NCT00909363 (ClinicalTrials.gov) | June 2009 | 27/5/2009 | Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients | Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia. | Wiskott-Aldrich Syndrome;Thrombocytopenia;Bleeding | Drug: Promacta;Diagnostic Test: blood drawing in patients with WAS;Diagnostic Test: blood drawing in healthy controls | Weill Medical College of Cornell University | Novartis Pharmaceuticals | Terminated | 3 Months | 80 Years | Male | 24 | Phase 2 | United States |
| 346 | NCT00906880 (ClinicalTrials.gov) | April 2009 | 19/5/2009 | Clinical Study to Assess the Tolerability, Feasibility and Effectiveness of Nifurtimox and Eflornithine (NECT) for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Phase | Clinical Study to Assess the Clinical Tolerability, Feasibility and Effectiveness Under Field Conditions of the Combination of Nifurtimox and Eflornithine (NECT) for the Treatment of T.b.Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Stage | Human African Trypanosomiasis | Drug: Nifurtimox-Eflronithine Combination Treatment (NECT) | Drugs for Neglected Diseases | Ministry of Public Health, Democratic Republic of the Congo;Swiss Tropical & Public Health Institute | Completed | N/A | N/A | Both | 630 | Phase 4 | Congo |
| 347 | NCT00799071 (ClinicalTrials.gov) | February 2009 | 26/11/2008 | Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD) | Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD) | Chronic Granulomatous Disease | Drug: posaconazole (PSZ) | Radboud University | NULL | Completed | 2 Years | 16 Years | All | 12 | Phase 2 | Netherlands;United Kingdom |
| 348 | EUCTR2007-003235-23-FR (EUCTR) | 09/01/2009 | 23/10/2008 | Essai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICV | Essai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICV | Déficit immunitaire commun variable MedDRA version: 9.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: PNEUMO 23 Trade Name: PREVENAR | Inserm | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | France | |||
| 349 | NCT01289171 (ClinicalTrials.gov) | January 2009 | 12/1/2011 | Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients | Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients: Prospective Treatment Trial With Topical Glycolic Acid and HPV Genotype Characterization | Flat Warts (Diagnosis);HIV Infections | Drug: Glycolic acid | University of Pennsylvania | NeoStrata Company, Inc.;Penn Center for AIDS Research (CFAR) | Completed | 7 Years | N/A | Both | 38 | N/A | Botswana |
| 350 | NCT00811174 (ClinicalTrials.gov) | January 2009 | 17/12/2008 | Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases | Clinical Study to Evaluate the Efficacy, Safety and Kinetics of Octagam 10% for Replacement Therapy in Primary Immunodeficiency Diseases | Immunologic Deficiency Syndromes | Drug: Octagam 10% | Octapharma | NULL | Terminated | 2 Years | 75 Years | All | 5 | Phase 3 | Austria |
| 351 | NCT00814320 (ClinicalTrials.gov) | December 18, 2008 | 23/12/2008 | Gammagard Liquid and rHuPH20 in PID | Efficacy, Tolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human), 10% (GAMMAGARD LIQUID/KIOVIG) Administered Intravenously or Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV) | Baxalta now part of Shire | NULL | Completed | 2 Years | N/A | All | 89 | Phase 3 | United States;Canada |
| 352 | EUCTR2008-000830-30-SE (EUCTR) | 17/12/2008 | 03/11/2008 | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | PID (primary immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: normal human immunoglobulin G | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | United Kingdom;Germany;France;Spain;Sweden | |||
| 353 | EUCTR2008-000830-30-GB (EUCTR) | 10/12/2008 | 20/01/2009 | Extension study designed to test the long-term safety and effectiveness of Immunoglobulin Subcutaneous (Human), IgPro20. | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency (IgPro20 EU Extension Study) | PID (Primary Immune Deficiency) MedDRA version: 13.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Hizentra® Product Code: IgPro20 INN or Proposed INN: Human Normal Immunoglobulin | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Spain;Poland;Romania;Germany;Switzerland;United Kingdom;Sweden | ||
| 354 | EUCTR2008-000830-30-FR (EUCTR) | 24/11/2008 | 21/10/2008 | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | PID (primary immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Normal Immunoglobulin | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 3 | France;Spain;Germany;United Kingdom;Sweden | ||
| 355 | EUCTR2008-004518-28-NL (EUCTR) | 19/11/2008 | 24/11/2008 | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPOD | Children with chronic granulomatous disease (CGD). MedDRA version: 9.1;Level: LLT;Classification code 10008906;Term: Chronic granulomatous disease | Trade Name: Noxafil 40 mg/ml oral solution | Radboud University Nijmegen Medical Centre | NULL | Not Recruiting | Female: yes Male: yes | United Kingdom;Netherlands | ||||
| 356 | EUCTR2008-000830-30-ES (EUCTR) | 12/11/2008 | 01/10/2008 | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of ImmuneGlobulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study)Un estudio de extensión multicéntrico sobre la eficacia, tolerabilidad y seguridad de la inmunoglobulina subcutánea (humana) IgPro20 en pacientes con inmunodeficiencia primaria(Estudio de extensión de la UE sobre IgPro20) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of ImmuneGlobulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study)Un estudio de extensión multicéntrico sobre la eficacia, tolerabilidad y seguridad de la inmunoglobulina subcutánea (humana) IgPro20 en pacientes con inmunodeficiencia primaria(Estudio de extensión de la UE sobre IgPro20) | Primary Immunodeficiency (PID)inmunodeficiencia primaria (IDP) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Normal Immunoglobulin | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | United Kingdom;Germany;France;Spain;Sweden | |||
| 357 | NCT00794508 (ClinicalTrials.gov) | November 2008 | 19/11/2008 | MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID | MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan | Severe Combined Immunodeficiency | Biological: ADA gene transfer | Donald B. Kohn, M.D. | FDA Office of Orphan Products Development;National Institutes of Health (NIH) | Completed | 1 Month | 18 Years | All | 10 | Phase 2 | United States |
| 358 | EUCTR2007-002611-27-DE (EUCTR) | 27/10/2008 | 14/12/2007 | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA | Primary Immundeficiency Diseases (PID) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Trade Name: Octagam 10% Product Name: Octagam 10% | Octapharma AG | NULL | Not Recruiting | Female: yes Male: yes | 45 | France;Germany;United Kingdom | |||
| 359 | EUCTR2006-006745-13-PL (EUCTR) | 16/10/2008 | 11/08/2008 | A multicenter study of the efficacy, tolerability, safety, and pharmacokinetics of Immune Globuline Subcutaneous (Human) IgPro20 in subjects with Primary Immunodeficiency (PID) | A multicenter study of the efficacy, tolerability, safety, and pharmacokinetics of Immune Globuline Subcutaneous (Human) IgPro20 in subjects with Primary Immunodeficiency (PID) | PID (Primary Immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Other descriptive name: IMMUNOGLOBULIN G | CSL Behring | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 3 | France;Spain;Poland;Germany;Italy;United Kingdom;Sweden | ||
| 360 | NCT00774358 (ClinicalTrials.gov) | October 2008 | 16/10/2008 | Interleukin-2 Treatment for Wiskott-Aldrich Syndrome | Reinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 Therapy | Wiskott-Aldrich Syndrome (WAS);X-linked Thrombocytopenia | Drug: Interleukin-2 | Soma Jyonouchi | Texas Children's Hospital | Completed | 24 Months | N/A | All | 9 | Phase 1 | United States |
| 361 | NCT00751621 (ClinicalTrials.gov) | August 2008 | 11/9/2008 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (IgPro20 EU Extension Study) | Primary Immunodeficiency (PID) | Biological: IgPro20 | CSL Behring | NULL | Completed | 2 Years | 65 Years | All | 40 | Phase 3 | France;Germany;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom |
| 362 | EUCTR2008-000830-30-DE (EUCTR) | 10/07/2008 | 09/06/2008 | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency(IgPro20 EU Extension Study) | PID (primary immuno deficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Normal Immunoglobulin | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | United Kingdom;Germany;France;Spain;Sweden | |||
| 363 | EUCTR2008-003368-21-IT (EUCTR) | 04/06/2008 | 06/02/2009 | Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome | Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome | For patients with a stabilized clinical condition, not recovered in care units for acute problems. MedDRA version: 9.1;Level: HLGT;Classification code 10003816 MedDRA version: 9.1;Level: HLT;Classification code 10027657 | Trade Name: ORENCIA INN or Proposed INN: ABATACEPT | ISTITUTO PER L`INFANZIA BURLO GAROFOLO | NULL | Not Recruiting | Female: no Male: yes | Italy | ||||
| 364 | NCT00719680 (ClinicalTrials.gov) | June 2008 | 21/7/2008 | Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency | Biological: IgPro20 | CSL Behring | NULL | Completed | 2 Years | 75 Years | All | 21 | Phase 3 | United States |
| 365 | NCT00634569 (ClinicalTrials.gov) | May 2008 | 5/3/2008 | Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects | Evaluation of the Efficacy and Safety of Flebogamma 5% DIF [Immune Globulin Intravenous (Human)] for Replacement Therapy in Pediatric Subjects With Primary Immunodeficiency Diseases. | Primary Immune Deficiency Disease | Biological: Flebogamma 5% DIF | Instituto Grifols, S.A. | NULL | Completed | 2 Years | 16 Years | All | 24 | Phase 4 | United States |
| 366 | NCT00680446 (ClinicalTrials.gov) | April 2008 | 16/5/2008 | Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary Immunodeficiency | An Open-Label Study of Ig NextGen 16% Administered by Subcutaneous Infusion in Patients With Primary Immunodeficiency (PID). | Primary Immune Deficiency | Drug: Immunoglobulin G (Ig NextGen 16%) | CSL Limited | NULL | Completed | 3 Years | N/A | Both | 41 | Phase 3 | Australia;New Zealand |
| 367 | EUCTR2006-006522-25-GR (EUCTR) | 08/01/2008 | 04/06/2007 | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years MedDRA version: 8.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin INN or Proposed INN: immune globulin subcutaneous (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 4 | Greece;Spain;Belgium;Germany;Italy;United Kingdom | ||
| 368 | NCT00605657 (ClinicalTrials.gov) | January 2008 | 15/1/2008 | Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS) | Pilot (Phase I-II) Study of Valproic Acid (Depakote) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS) | ALPS;Hypersplenism;Lymphadenopathy | Drug: Valproic Acid;Procedure: CT Scan;Procedure: Blood Sample | Koneti Rao | NULL | Completed | 2 Years | 70 Years | All | 6 | Phase 1/Phase 2 | United States |
| 369 | NCT00546871 (ClinicalTrials.gov) | October 3, 2007 | 18/10/2007 | Comparison of Intravenous and Subcutaneous Administration of IGIV, 10% in Primary Immunodeficiency (PID) Subjects | Tolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human) 10% (IGIV, 10%) Administered Intravenously or Subcutaneously in Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Drug: Immune Globulin Intravenous (Human), 10% | Baxalta now part of Shire | NULL | Completed | 24 Months | N/A | All | 49 | Phase 2/Phase 3 | United States |
| 370 | NCT00579137 (ClinicalTrials.gov) | October 2007 | 19/12/2007 | Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders | CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency Disorders | Severe Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick Syndrome | Biological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusion | Baylor College of Medicine | Center for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's Hospital | Terminated | N/A | N/A | All | 3 | Phase 1/Phase 2 | United States |
| 371 | EUCTR2006-006023-39-IT (EUCTR) | 24/09/2007 | 12/06/2007 | Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - ND | Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - ND | PK study in patient with afibrinogemia MedDRA version: 9.1;Level: LLT;Classification code 10016075;Term: Factor I deficiency | Trade Name: Haemocomplettan P | CSL Behring GmbH | NULL | Not Recruiting | Female: yes Male: yes | Italy | ||||
| 372 | EUCTR2006-006745-13-SE (EUCTR) | 19/09/2007 | 23/07/2007 | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | PID (Primary Immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Immunglobulin G (IgG) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 51 | United Kingdom;Germany;France;Spain;Italy;Poland;Sweden | |||
| 373 | EUCTR2006-006522-25-GB (EUCTR) | 10/09/2007 | 28/03/2007 | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years MedDRA version: 8.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin INN or Proposed INN: immune globulin subcutaneous (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Greece;Spain;Belgium;Germany;Italy;United Kingdom | ||
| 374 | NCT00542997 (ClinicalTrials.gov) | September 2007 | 11/10/2007 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy | A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Autosomal Recessive Agammaglobulinemia | Biological: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC) | CSL Behring | NULL | Completed | 2 Years | 65 Years | All | 51 | Phase 3 | France;Germany;Italy;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom |
| 375 | NCT00527878 (ClinicalTrials.gov) | September 2007 | 8/9/2007 | Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome | A Double-Blind, Randomized, Placebo-Controlled Cross-Over Study Assessing the Role of Pathogen-Specific IgE and Histamine Release in the Hyper-IgE Syndrome and the Effect of Ranitidine on Laboratory and Clinical Manifestations | JOB's Syndrome;Hyper-IgE Recurrent Infection Syndrome;Immune Deficiency | Drug: Ranitidine;Drug: Placebo | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | N/A | All | 16 | Phase 2 | United States |
| 376 | EUCTR2006-006745-13-IT (EUCTR) | 30/08/2007 | 01/08/2007 | A Multicenter Study of the Efficacy, Tolerability, Safety and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency - ND | A Multicenter Study of the Efficacy, Tolerability, Safety and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency - ND | PID (Primary Immunodeficiency) MedDRA version: 6.1;Level: HLGT;Classification code 10021460 | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Immunoglobulins, normal human | CSL Behring AG (casa madre) | NULL | Not Recruiting | Female: yes Male: yes | 36 | United Kingdom;Germany;France;Spain;Italy;Poland;Sweden | |||
| 377 | EUCTR2006-006745-13-ES (EUCTR) | 27/08/2007 | 11/06/2007 | Estudio multicéntrico sobre la eficacia, la tolerabilidad, la seguridad y la farmacocinética de la immunoglobulina subcutánea (humana) IgPro20 en sujetos con immunodeficiencia orimaria (PID)A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | Estudio multicéntrico sobre la eficacia, la tolerabilidad, la seguridad y la farmacocinética de la immunoglobulina subcutánea (humana) IgPro20 en sujetos con immunodeficiencia orimaria (PID)A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | immunodeficencia primaria (PID) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Immunglobulin G (IgG) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 3 | France;Poland;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 378 | EUCTR2006-006522-25-IT (EUCTR) | 13/08/2007 | 31/07/2007 | A Multicenter study on the efficacy and safety of Vivaglobin in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) - ND | A Multicenter study on the efficacy and safety of Vivaglobin in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) - ND | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years. MedDRA version: 6.1;Level: PT;Classification code 10057863 | Trade Name: Vivaglobin INN or Proposed INN: immunglobulin (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | United Kingdom;Germany;Belgium;Spain;Italy;Greece | |||
| 379 | NCT00455312 (ClinicalTrials.gov) | August 2007 | 30/3/2007 | Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA | Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia | Dyskeratosis Congenita;Aplastic Anemia | Drug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: Methylprednisolone | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 70 Years | All | 36 | Phase 2/Phase 3 | United States |
| 380 | EUCTR2006-006745-13-GB (EUCTR) | 17/07/2007 | 15/05/2007 | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | PID (Primary Immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Immunglobulin G (IgG) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 51 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 381 | EUCTR2006-006745-13-DE (EUCTR) | 11/07/2007 | 21/03/2007 | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | PID (Primary Immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Immunglobulin G (IgG) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 51 | United Kingdom;Germany;France;Spain;Italy;Poland;Sweden | |||
| 382 | EUCTR2006-006745-13-FR (EUCTR) | 10/07/2007 | 04/06/2007 | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency | PID (Primary Immunodeficiency) MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Immunglobulin G (IgG) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 3 | France;Poland;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 383 | EUCTR2006-006522-25-ES (EUCTR) | 01/07/2007 | 03/03/2010 | Estudio multicéntrico sobre la eficacia y la seguridad de Vivaglobin® en pacientes sin tratamiento previo (PUP) con inmunodeficiencia primaria (PID)(A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID)) | Estudio multicéntrico sobre la eficacia y la seguridad de Vivaglobin® en pacientes sin tratamiento previo (PUP) con inmunodeficiencia primaria (PID)(A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID)) | Pacientes con inmunodeficiencia primaria como inmunodeficiencia variable común (CVID) o agammaglobulinemia vinculada al cromosoma X (XLA) de entre 1 y 70 años de edad.(Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years) MedDRA version: 8.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin INN or Proposed INN: immune globulin subcutaneous (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 4 | Greece;Belgium;Spain;Germany;Italy;United Kingdom | ||
| 384 | EUCTR2006-006522-25-BE (EUCTR) | 27/06/2007 | 17/04/2007 | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years MedDRA version: 8.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin INN or Proposed INN: immune globulin subcutaneous (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | United Kingdom;Germany;Belgium;Spain;Italy;Greece | |||
| 385 | NCT00490100 (ClinicalTrials.gov) | June 2007 | 21/6/2007 | Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1 | Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency (XSCID): A Phase 2 Study Assessing Safety and Clinical Response to Treatment With Insulin-like Growth Factor-1 (IGF-1) | Growth Failure;X-linked Severe Combined Immunodeficiency (XSCID);Growth Hormone Resistence | Drug: Increlex | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | 20 Years | All | 6 | Phase 1/Phase 2 | United States |
| 386 | EUCTR2007-001410-17-FR (EUCTR) | 31/05/2007 | 13/04/2007 | LONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTS | LONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTS | Primary Immunodeficiency MedDRA version: 9.1;Level: LLT;Classification code 10064859;Term: Primary immunodeficiency syndrome | Product Name: Human normal immunoglobulin for intravenous administration Product Code: IGNG INN or Proposed INN: Human normal immunoglobulin for intravenous administration | LFB SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 387 | NCT00640003 (ClinicalTrials.gov) | April 2007 | 14/3/2008 | Baclofen Treatment of Ataxia Telangiectasia | Ataxia Telangiectasia | Drug: Baclofen;Drug: Placebo | Johns Hopkins University | NULL | Completed | 12 Years | N/A | All | 10 | Early Phase 1 | United States | |
| 388 | NCT00391131 (ClinicalTrials.gov) | April 2007 | 20/10/2006 | Subcutaneous Ig NextGen 16% in PID Patients | A Multi-centre, Open-label Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Subcutaneous Infusions of Ig NextGen 16% in Patients With Primary Immunodeficiency (PID). | Primary Immunodeficiency (PID) | Drug: IgNextGen 16% | CSL Limited | NULL | Completed | 3 Years | N/A | Both | 35 | Phase 3 | Australia;New Zealand |
| 389 | NCT00520494 (ClinicalTrials.gov) | March 2007 | 23/8/2007 | Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency | A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID) | Common Variable Immunodeficiency;Agammaglobulinemia | Drug: Vivaglobin | CSL Behring | NULL | Completed | 1 Year | 70 Years | All | 18 | Phase 4 | Canada;Germany;Italy;Spain;Belgium |
| 390 | EUCTR2006-006522-25-DE (EUCTR) | 01/02/2007 | 07/12/2006 | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) | Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years MedDRA version: 8.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Trade Name: Vivaglobin Product Name: Vivaglobin INN or Proposed INN: immune globulin subcutaneous (human) | CSL Behring AG | NULL | Not Recruiting | Female: yes Male: yes | 28 | United Kingdom;Germany;Belgium;Spain;Italy;Greece | |||
| 391 | NCT02512679 (ClinicalTrials.gov) | February 2007 | 21/5/2015 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Protocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological Diseases | Stem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic Diseases | Drug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4 | Children's Hospital Los Angeles | Lucile Packard Children's Hospital | Terminated | 3 Months | N/A | All | 20 | Phase 2 | NULL |
| 392 | NCT00885833 (ClinicalTrials.gov) | February 2007 | 21/4/2009 | Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) | Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Drug: Fludarabine, Busulfan, Thymoglobulin | The Korean Society of Pediatric Hematology Oncology | NULL | Completed | 1 Year | 25 Years | Both | 5 | Phase 1/Phase 2 | Korea, Republic of |
| 393 | NCT02127892 (ClinicalTrials.gov) | January 2, 2007 | 19/10/2012 | SCID Bu/Flu/ATG Study With T Cell Depletion | Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor | Severe Combined Immunodeficiency | Biological: unrelated BM with T cell depletion;Biological: unrelated cord blood;Biological: haplo BM with T cell depletion;Device: unrelated PBSC with T cell depletion | Neena Kapoor, M.D. | NULL | Terminated | N/A | 21 Years | All | 9 | Phase 1/Phase 2 | United States |
| 394 | NCT00778882 (ClinicalTrials.gov) | January 2007 | 21/10/2008 | Gene Therapy for Chronic Granulomatous Disease in Korea | An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients | Chronic Granulomatous Disease | Drug: VM106 | Helixmith Co., Ltd. | NULL | Active, not recruiting | N/A | N/A | Male | 2 | Phase 1/Phase 2 | Korea, Republic of |
| 395 | EUCTR2005-003201-81-DE (EUCTR) | 20/12/2006 | 19/01/2007 | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | Herpes simplex virus type-1 (HSV-1) is the most frequent cause of fatal sporadic encephalitis in humans. Herpes simplex encephalitis (HSE) was one of the first viral infections to be successfully treated with antiviral drugs. Mortality has been significantly reduced since the introduction of acyclovir, a specific inhibitor of HSV replication. Despite appropriate and promptly initiated antiviral therapy the incidence of persistent neurological deficits remain unacceptably high. | Trade Name: Fortecortin Inject 40 mg Amp. Product Name: Fortecortin INN or Proposed INN: dexamethasone 21-dihydrogen phosphate | Universitätsklinikum Heidelberg | NULL | Not Recruiting | Female: yes Male: yes | 450 | Phase 3 | Germany | ||
| 396 | NCT00782106 (ClinicalTrials.gov) | December 4, 2006 | 29/10/2008 | Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV Subcutaneously | Phase 1/2 Determination of the Dose of Recominant Human Hyaluronidase (rHuPH20) Required Enabling Up to 600 mg/kg Bodyweight of IGIV, 10% to be Administered Subcutaneously in a Single Infusion Site in Subjects With Primary Immunodeficiency (PID) | Primary Immunodeficiency Diseases (PID) | Biological: Recombinant human hyaluronidase + immune globulin intravenous | Baxalta now part of Shire | NULL | Completed | 16 Years | N/A | All | 11 | Phase 1/Phase 2 | United States |
| 397 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1/Phase 2 | United States |
| 398 | NCT00419341 (ClinicalTrials.gov) | November 2006 | 22/12/2006 | Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy | A Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency | Biological: Human Normal Immunoglobulin for Subcutaneous Administration | CSL Behring | NULL | Completed | 2 Years | 75 Years | All | 49 | Phase 3 | United States |
| 399 | NCT00389324 (ClinicalTrials.gov) | November 2006 | 17/10/2006 | A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency | An Open-Label Single-Sequence, Crossover Trial to Evaluate the Pharmacokinetics and Safety of Subcutaneous Gamunex® 10% (Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified) in Subjects With Primary Immunodeficiency | Immunologic Deficiency Syndrome | Biological: Immune Globulin Intravenous (Human) | Grifols Therapeutics Inc. | NULL | Completed | 13 Years | 75 Years | All | 35 | Phase 2 | United States;Canada |
| 400 | NCT00394316 (ClinicalTrials.gov) | October 30, 2006 | 31/10/2006 | Gene Therapy for Chronic Granulomatous Disease | Autologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 3 Years | 55 Years | Male | 3 | Early Phase 1 | United States |
| 401 | NCT00395538 (ClinicalTrials.gov) | October 30, 2006 | 2/11/2006 | Effects of PTH Replacement on Bone in Hypoparathyroidism | Effects of PTH Replacement on Bone in Hypoparathyroidism | Hypoparathyroidism;DiGeorge Syndrome | Drug: PTH 1-34 | National Institute of Dental and Craniofacial Research (NIDCR) | NULL | Terminated | 18 Years | 70 Years | All | 46 | Phase 3 | United States;Austria;Italy |
| 402 | NCT00656409 (ClinicalTrials.gov) | June 2006 | 7/4/2008 | Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT) | Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT) | Ataxia Telangiectasia (AT) | Drug: Conjugated pneumococcal vaccine (Prevenar) | Institute of Child Health | Great Ormond Street Hospital for Children NHS Foundation Trust | Completed | 2 Years | N/A | Both | 30 | Phase 3 | United Kingdom |
| 403 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 404 | NCT00325078 (ClinicalTrials.gov) | May 2006 | 11/5/2006 | Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease | Tumor Necrosis Factor Alpha Inhibitor (Lnfliximab, Adalimumab) Treatment for Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease: A Phase I/II Study Assessing Clinical and Immune Responses to Treatment and Genetic Influences | Chronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD) | Drug: Infliximab | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 10 Years | N/A | All | 40 | Phase 1/Phase 2 | United States |
| 405 | NCT00278954 (ClinicalTrials.gov) | January 2006 | 18/1/2006 | Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. | A Phase III, Multicenter, Open-Label Study To Evaluate The Efficacy, Safety, and Pharmacokinetics of Gammaplex® in Primary Immunodeficiency Diseases | Primary Immunodeficiency;Common Variable Hypogammaglobulinemia;X-linked Hypogammaglobulinemia;Hypogammaglobulinemia;Immunodeficiency With Hyper-IgM;Wiskott-Aldrich Syndrome | Biological: Gammaplex (Intravenous immunoglobulin) | Bio Products Laboratory | NULL | Completed | 3 Years | N/A | All | 50 | Phase 3 | United States |
| 406 | NCT00579527 (ClinicalTrials.gov) | December 19, 2005 | 20/12/2007 | Phase I/II Thymus Transplantation With Immunosuppression #950 | Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950 | DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge Syndrome | Biological: Cultured Thymus Tissue for Implantation (CTTI);Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | N/A | All | 14 | Phase 1/Phase 2 | United States |
| 407 | NCT00263237 (ClinicalTrials.gov) | December 2, 2005 | 7/12/2005 | STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable Immunodeficiency | A Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: STA-5326 | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Years | 75 Years | All | 10 | Phase 1 | United States |
| 408 | NCT00260702 (ClinicalTrials.gov) | November 24, 2005 | 1/12/2005 | Omalizumab to Treat Hyper-IgE (Job's) Syndrome | Pilot Study of Omalizumab (Xolair) in Hyper IgE (Job's) Syndrome | Hyper-IgE Syndrome;Job's Syndrome | Drug: Omalizumab (Xolair) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 6 Years | 76 Years | All | 1 | Phase 1 | United States |
| 409 | EUCTR2005-004122-70-GB (EUCTR) | 15/11/2005 | 12/09/2005 | Immunogenicity of conjugate pneumococcal vaccine (Prevenar) in patients with ataxia -telangiectasia (AT) including a randomised study of one versus two doses of vaccine. - Immunogenicity of Prevenar in Ataxia telangiectasia | Immunogenicity of conjugate pneumococcal vaccine (Prevenar) in patients with ataxia -telangiectasia (AT) including a randomised study of one versus two doses of vaccine. - Immunogenicity of Prevenar in Ataxia telangiectasia | Ataxia Telangiectasia (AT) is a genetic condition in which there is a deficiency of the immune system with excessive susceptibility to pneumonia and other infections. | Trade Name: Prevenar | Great Ormond Street Hospital | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | United Kingdom | ||
| 410 | NCT00322556 (ClinicalTrials.gov) | November 2005 | 5/5/2006 | Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID) | A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | NULL | Completed | 4 Years | 71 Years | All | 55 | Phase 3 | United States |
| 411 | NCT00119431 (ClinicalTrials.gov) | September 2005 | 4/7/2005 | Kinetics, Efficacy and Safety of C1-Esteraseremmer-N | Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema | Hereditary Angioedema Type I;Angioneurotic Edema | Drug: C1 inhibitor concentrate | Sanquin | NULL | Completed | 18 Years | N/A | Both | 12 | Phase 2 | Netherlands |
| 412 | EUCTR2004-004465-15-HU (EUCTR) | 22/07/2005 | 11/05/2005 | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | primary immunodeficiency syndrome as congenital agammaglobulinaemia or hypogammaglobulinaemia, common variable immunodeficiency, severe combined immunodeficiencies, Wiskott Aldrich syndrome MedDRA version: 7.0;Level: HLT;Classification code 10036700 | Trade Name: Intratect Product Name: Intratect Product Code: BT681 Other descriptive name: human normal immunoglobulin (IVIg) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Germany | |||
| 413 | NCT00160355 (ClinicalTrials.gov) | May 2005 | 8/9/2005 | Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome | Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study | Wiskott-Aldrich Syndrome | Procedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, Thiotepa | St. Jude Children's Research Hospital | NULL | Completed | N/A | 18 Years | Male | 4 | Phase 1 | United States |
| 414 | EUCTR2004-004465-15-DE (EUCTR) | 05/04/2005 | 04/02/2005 | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID) | primary immunodeficiency syndrome as congenital agammaglobulinaemia or hypogammaglobulinaemia, common variable immunodeficiency, severe combined immunodeficiencies, Wiskott Aldrich syndrome MedDRA version: 7.0;Level: HLT;Classification code 10036700 | Product Name: Intratect Product Code: BT681 Other descriptive name: human normal immunoglobulin (IVIg) | Biotest AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Germany | |||
| 415 | NCT00295971 (ClinicalTrials.gov) | April 2005 | 23/2/2006 | Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease | Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases | Congenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiation | University of California, San Francisco | National Cancer Institute (NCI) | Completed | 1 Year | 17 Years | Both | 21 | Phase 1 | United States |
| 416 | NCT00566488 (ClinicalTrials.gov) | January 2005 | 30/11/2007 | Parathyroid and Thymus Transplantation in DiGeorge #931 | Parathyroid and Thymus Transplantation in DiGeorge Syndrome, #931 | DiGeorge Syndrome;Hypoparathyroidism;Complete DiGeorge Syndrome | Biological: Thymus/Parathyroid Transplantation | M. Louise Markert | Food and Drug Administration (FDA);National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Completed | N/A | 24 Months | All | 25 | Phase 1 | United States |
| 417 | NCT00301834 (ClinicalTrials.gov) | January 2005 | 9/3/2006 | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders | Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Completed | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 418 | NCT00123916 (ClinicalTrials.gov) | November 2004 | 21/7/2005 | BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease | Benznidazole Evaluation for Interrupting Trypanosomiasis - The BENEFIT Trial | Chagas Disease;Trypanosomiasis;Heart Disease | Drug: Benznidazole;Drug: Placebo | Population Health Research Institute | Canadian Institutes of Health Research (CIHR);World Health Organization;Instituto Dante Pazzanese de Cardiologia;University of Sao Paulo | Completed | 18 Years | 75 Years | All | 2854 | Phase 3 | Argentina;Bolivia;Brazil;Colombia;El Salvador;Canada |
| 419 | NCT00576836 (ClinicalTrials.gov) | September 2, 2004 | 17/12/2007 | Thymus Transplantation Dose in DiGeorge #932 | Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1 | DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Cultured Thymus Tissue Implantation (CTTI);Other: Cultured Thymus Tissue Implantation with Parathyroid Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Completed | N/A | N/A | All | 7 | Phase 2 | United States |
| 420 | NCT00168025 (ClinicalTrials.gov) | September 2004 | 12/9/2005 | Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID) | A Multicenter Study on the Efficacy, Safety and Pharmacokinetics of IgPro10 in Patients With Primary Immunodeficiency (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | NULL | Completed | 3 Years | 70 Years | Both | 89 | Phase 3 | NULL |
| 421 | NCT00146627 (ClinicalTrials.gov) | September 2004 | 6/9/2005 | Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis | Clinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic Phase | Trypanosomiasis, African | Drug: Eflornithine;Drug: Nifurtimox | Drugs for Neglected Diseases | Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization;Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization | Completed | 15 Years | 70 Years | Both | 280 | Phase 3 | Congo, The Democratic Republic of the;Congo;The Democratic Republic of the Congo;Uganda |
| 422 | NCT00168012 (ClinicalTrials.gov) | September 2004 | 12/9/2005 | Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID) | An Open Study to Evaluate the Safety and Efficacy of IVIG-F10 in Patients With Primary Immunodeficiency Diseases (PID) | Agammaglobulinemia;IgG Deficiency;Common Variable Immunodeficiency | Drug: Immunoglobulins Intravenous (Human) | CSL Behring | NULL | Completed | 3 Years | 70 Years | Both | 42 | Phase 3 | NULL |
| 423 | NCT00228852 (ClinicalTrials.gov) | April 2004 | 27/9/2005 | IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | Phase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | T-Cell Immune Deficiency Diseases;Severe Combined Immunodeficiency | Drug: Busulfan, Fludarabine and ATG | Emory University | NULL | Completed | N/A | N/A | Both | Phase 1/Phase 2 | United States | |
| 424 | NCT00578643 (ClinicalTrials.gov) | March 2004 | 19/12/2007 | Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell Infusion | Baylor College of Medicine | NULL | Completed | N/A | N/A | All | 15 | Phase 2 | United States |
| 425 | NCT00152100 (ClinicalTrials.gov) | February 2004 | 7/9/2005 | Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome | Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome | Severe Combined Immunodeficiency | Procedure: Stem cell transplant;Drug: Filgrastim, Alemtuzumab;Device: Miltenyi CliniMACS | St. Jude Children's Research Hospital | NULL | Completed | N/A | 2 Years | Both | 4 | Phase 1 | United States |
| 426 | NCT00564759 (ClinicalTrials.gov) | January 2004 | 26/11/2007 | Gene Therapy for Chronic Granulomatous Disease | Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic | Drug: retroviral SF71-gp91phox transduced CD34+ cells | Johann Wolfgang Goethe University Hospitals | German Federal Ministry of Education and Research | Active, not recruiting | 18 Years | N/A | Male | 2 | Phase 1/Phase 2 | Germany |
| 427 | NCT01279720 (ClinicalTrials.gov) | October 2003 | 18/1/2011 | Gene Therapy ADA Deficiency | Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency | Adenosine Deaminase Deficiency | Biological: Intravenous infusion of transduced cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Completed | N/A | 18 Years | Both | 8 | Phase 1/Phase 2 | United Kingdom |
| 428 | NCT00065390 (ClinicalTrials.gov) | July 2003 | 21/7/2003 | Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome | Pilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS) | Autoimmune Disease;Lymphatic Disease;Lymphoproliferative Disorder | Drug: Pyrimethamine | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 8 | Phase 1 | United States |
| 429 | NCT00803933 (ClinicalTrials.gov) | February 2003 | 4/12/2008 | Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Phase II b Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | African Trypanosomiasis | Drug: DB289;Drug: Pentamidine | Immtech Pharmaceuticals, Inc | Bill and Melinda Gates Foundation | Completed | 15 Years | 50 Years | Both | 111 | Phase 2 | Congo |
| 430 | NCT00489658 (ClinicalTrials.gov) | October 2002 | 20/6/2007 | Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, Uganda | Efficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, Uganda | Trypanosomiasis, African | Drug: Eflornithine plus Nifurtimox combination therapy | Epicentre | Medecins Sans Frontieres | Terminated | N/A | N/A | Both | 31 | Phase 2/Phase 3 | Uganda |
| 431 | NCT00187057 (ClinicalTrials.gov) | September 2002 | 12/9/2005 | Study for Treatment of Cancer in Children With Ataxia-telangiectasia | Pilot Study I for Treatment of Cancer in Children With Ataxia-Telangiectasia | Ataxia-Telangiectasia | Drug: vinblastine, vincristine, prednisone, daunorubicin;Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase;Drug: etoposide, cytarabine, mercaptopurine;Drug: dexamethasone, procarbazine;Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy | St. Jude Children's Research Hospital | Children's Hospital of Philadelphia;National Cancer Institute (NCI) | Completed | N/A | 10 Years | Both | 6 | N/A | United States |
| 432 | NCT00220766 (ClinicalTrials.gov) | August 2002 | 13/9/2005 | Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients | IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients | Immunologic Deficiency Syndrome;Agammaglobulinemia;Severe Combined Immunodeficiency;Wiskott-Aldrich Syndrome;Common Variable Immunodeficiency | Drug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified;Drug: Dextrose, 5% in Water | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | 75 Years | Both | 100 | Phase 3 | United States;Canada |
| 433 | NCT00579709 (ClinicalTrials.gov) | July 2002 | 20/12/2007 | Thymus Transplantation With Immunosuppression | Thymus Transplantation With Immunosuppression, #884 | DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Thymus Tissue for Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Completed | N/A | N/A | All | 15 | Phase 1 | United States |
| 434 | NCT00157079 (ClinicalTrials.gov) | June 25, 2002 | 8/9/2005 | Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders | A Clinical Investigation to Assess the Safety and Efficacy of Immune Globulin Intravenous (Human), 10% in Subjects With Primary Immunodeficiency Disorders | Primary Immunodeficiency Diseases (PID);Immune Thrombocytopenic Purpura (ITP);Kawasaki Syndrome | Biological: Immune Globulin Intravenous (Human), 10% | Baxalta now part of Shire | NULL | Completed | 24 Months | N/A | All | 61 | Phase 3 | United States |
| 435 | NCT00161993 (ClinicalTrials.gov) | June 13, 2002 | 8/9/2005 | Safety, Pharmacokinetic and Efficacy Study of a 10% Triple Virally Reduced Intravenous Immune Globulin Solution in Patients With Primary Immunodeficiency (Hypo- or Agammaglobulinemia) | Prospective Open-Label Study of Pharmacokinetics, Efficacy and Safety of Immune Globulin Intravenous (Human), 10% TVR Solution in Patients With Hypo- or Agammaglobulinemia | Primary Immunodeficiency Diseases (PID);Agammaglobulinemia;Hypogammaglobulinemia | Drug: Immune Globulin Intravenous (Human), 10% TVR (Triple Virally Reduced) Solution;Drug: Gammagard S/D (Solvent/Detergent) | Baxalta now part of Shire | NULL | Completed | 18 Years | N/A | All | 24 | Phase 2 | Finland;Sweden |
| 436 | NCT00176852 (ClinicalTrials.gov) | June 2002 | 12/9/2005 | Stem Cell Transplant for Hemoglobinopathy | Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism | Sickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond Syndrome | Drug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusion | Masonic Cancer Center, University of Minnesota | National Marrow Donor Program | Completed | N/A | 50 Years | All | 22 | Phase 2/Phase 3 | United States |
| 437 | NCT01019876 (ClinicalTrials.gov) | June 2002 | 23/11/2009 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | NULL | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States |
| 438 | NCT00661401 (ClinicalTrials.gov) | January 2002 | 14/4/2008 | Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin | Serum IgG Antibody to Streptococcus Pneumoniae, Haemophilus Influenzae Type b and Tetanus Toxoid in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin Infusions | Common Variable Immunodeficiency;Agammaglobulinemia | Biological: gammaglobulin | Federal University of São Paulo | CSL Behring | Completed | 2 Years | 75 Years | Both | 5 | N/A | Brazil |
| 439 | NCT00028236 (ClinicalTrials.gov) | December 10, 2001 | 17/12/2001 | Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) | Ex Vivo Retroviral Gene Transfer For Treatment of X-Linked Severe Combined Immunodeficiency (XSCID) | Severe Combined Immunodeficiency | Drug: Gene-Transduced Autologous CD34+ Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Months | 20 Years | All | 3 | Phase 1 | United States |
| 440 | NCT00024934 (ClinicalTrials.gov) | October 2001 | 9/10/2001 | B-Lymphocyte Stimulator (BLyS) To Treat Selective IgA Deficiency | A Phase I Dose Escalation Study of B-Lymphocyte Stimulator (BLyS) Administered Subcutaneously in Patients With Selective IgA Deficiency | IgA Deficiency | Drug: B-Lymphocyte Stimulator (BLyS) | National Cancer Institute (NCI) | NULL | Completed | N/A | N/A | Both | 20 | Phase 1 | United States |
| 441 | NCT00802594 (ClinicalTrials.gov) | August 2001 | 3/12/2008 | A Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Phase II A Trial of DB289 for the Treatment of Stage I African Trypanosomiasis | Trypanosomiasis, African | Drug: DB289 | Immtech Pharmaceuticals, Inc | Bill and Melinda Gates Foundation | Completed | 16 Years | N/A | Both | 30 | Phase 2 | Angola;Congo |
| 442 | NCT00023192 (ClinicalTrials.gov) | August 2001 | 29/8/2001 | Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care | Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care | Chronic Granulomatous Disease | Drug: T-Cell Depleted & CD34+Select/w/StemCell Enriched Product | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 60 | Phase 3 | United States |
| 443 | NCT00018018 (ClinicalTrials.gov) | June 20, 2001 | 27/6/2001 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency | Treatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA Gene | Severe Combined Immunodeficiency Syndrome | Drug: CD34+ cells transduced with ADA retrovir | National Human Genome Research Institute (NHGRI) | NULL | Completed | 1 Month | N/A | All | 8 | Phase 1 | United States |
| 444 | NCT00330148 (ClinicalTrials.gov) | March 2001 | 24/5/2006 | Randomized Clinical Trial of Three Drug Combinations for Late-Stage Gambiense Human African Trypanosomiasis | Clinical Trial Comparing the Therapeutic Combinations Melarsoprol-Nifurtimox, Melarsoprol-Eflornithine and Eflornithine-Nifurtimox in the Treatment of Gambiense Human African Trypanosomiasis in the Meningo-Encephalitic Phase | Trypanosomiasis, African | Drug: melarsoprol 1.8 mg/kg/d, 10d + nifurtimox 15/20 mg/kg/d, 10d;Drug: melarsoprol 1.8 mg/kg/d, 10d + eflornithine 400 mg/kg/d, 7d;Drug: nifurtimox 15/20 mg/kg/d 10d + eflornithine 400 mg/kg/d 7d | Epicentre | Medecins Sans Frontieres;Embassy of France in Uganda;National Sleeping Sickness Control Program, Uganda | Terminated | N/A | N/A | Both | 435 | Phase 3 | Uganda |
| 445 | NCT00013689 (ClinicalTrials.gov) | March 2001 | 28/3/2001 | Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome | Pilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS) | Autoimmune Disease;Lymphoproliferative Disorder | Drug: Fansidar (pyrimethamine and sulfadoxine) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 8 | Phase 1 | United States |
| 446 | NCT00031486 (ClinicalTrials.gov) | September 2000 | 6/3/2002 | Long Term Treatment of Herpes Simplex Encephalitis (HSE) With Valacyclovir | A Phase III Double-Blind, Placebo-Controlled Trial of Long Term Therapy of Herpes Simplex Encephalitis (HSE): An Evaluation of Valacyclovir (CASG-204) | Encephalitis | Drug: Valacyclovir;Drug: Placebo | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 12 Years | N/A | All | 91 | Phase 3 | United States;Canada;Sweden;United Kingdom |
| 447 | NCT00305708 (ClinicalTrials.gov) | August 2000 | 21/3/2006 | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission | Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy | University of California, San Francisco | National Cancer Institute (NCI) | Completed | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| 448 | NCT00176878 (ClinicalTrials.gov) | June 2000 | 12/9/2005 | Stem Cell Transplant for Bone Marrow Failure Syndromes | Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders | Diamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond Syndrome | Procedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulin | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 35 Years | All | 10 | Phase 2/Phase 3 | United States |
| 449 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 450 | NCT00006054 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | Immunologic Deficiency Syndromes;Chediak-Higashi Syndrome;Common Variable Immunodeficiency;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Hyper IgM Syndrome;Severe Combined Immunodeficiency;Leukocyte Adhesion Deficiency Syndrome;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow Transplantation | Fairview University Medical Center | NULL | Terminated | N/A | 35 Years | Both | N/A | United States | ||
| 451 | NCT00001145 (ClinicalTrials.gov) | October 1999 | 3/11/1999 | Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome | Study of Immune Responses and Safety of Recombinant CD40 Ligand in Patients With X-Linked Hyper IgM Syndrome | Immunoproliferative Disorder | Drug: Bacteriophage;Drug: rhuCD40L;Drug: KLH | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 5 | Phase 2 | United States |
| 452 | NCT00001905 (ClinicalTrials.gov) | April 1999 | 3/11/1999 | Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I | Interferon Gamma Administration in Leukocyte Adhesion Deficiency Type I | Leukocyte Adhesion Deficiency Syndrome | Drug: Interferon gamma | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 5 | Phase 2 | United States |
| 453 | NCT00004695 (ClinicalTrials.gov) | September 1997 | 24/2/2000 | Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: PEG-interleukin-2 | Mount Sinai School of Medicine | NULL | Completed | 2 Years | N/A | Both | 48 | N/A | NULL | |
| 454 | NCT00008450 (ClinicalTrials.gov) | August 11, 1997 | 6/1/2001 | Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant | Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil | Adenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined Immunodeficiency | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | N/A | N/A | All | 6 | Phase 1 | United States |
| 455 | NCT00001476 (ClinicalTrials.gov) | June 1, 1995 | 3/11/1999 | Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up | Gene Therapy Approach for Chronic Granulomatous Disease | Chronic Granulomatous Disease;Communicable Disease | Drug: Gene Therapy Method for CGD;Device: Isolex 300i Magnetic Cell Selector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 5 Years | N/A | All | 14 | Phase 1 | United States |
| 456 | NCT00004787 (ClinicalTrials.gov) | December 1994 | 24/2/2000 | Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes | Shwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;Thrombocytopenia | Drug: filgrastim | National Center for Research Resources (NCRR) | James Whitcomb Riley Hospital for Children | Completed | N/A | N/A | Both | 20 | Phase 2 | NULL | |
| 457 | NCT00001317 (ClinicalTrials.gov) | May 1992 | 3/11/1999 | A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood | A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood | Chronic Granulomatous Disease | Drug: interferon-gamma | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 100 | Phase 4 | United States |
| 458 | NCT00576407 (ClinicalTrials.gov) | October 1991 | 17/12/2007 | Thymus Transplantation in DiGeorge Syndrome #668 | Phase II Study of Thymus Transplantation in Complete DiGeorge Syndrome #668 | DiGeorge Syndrome;Complete Typical DiGeorge Anomaly | Biological: Cultured Thymus Tissue for Implantation (CTTI) | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Completed | N/A | N/A | All | 26 | Phase 2 | United States |
| 459 | NCT00001280 (ClinicalTrials.gov) | January 1991 | 3/11/1999 | Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease | Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease | Mycoses | Drug: itraconazole | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | N/A | N/A | Both | 100 | Phase 2 | United States |
| 460 | NCT00001255 (ClinicalTrials.gov) | September 1990 | 3/11/1999 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study | Treatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History Study | Severe Combined Immunodeficiency | Drug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced Lymphocytes | National Human Genome Research Institute (NHGRI) | NULL | Completed | N/A | N/A | Both | 10 | N/A | United States |
| 461 | EUCTR2011-005015-82-Outside-EU/EEA (EUCTR) | 10/01/2012 | Study to evaluate the safety of human immune globulin in patients withprimary immunodeficiency diseases. | Clinical study to evaluate the safety and tolerability of immunoglobulin intravenous (human) 10% (NewGam) administered at high infusion rates to patients with primary immunodeficiency diseases (extension of study NGAM 01) | primary immunodeficiency diseases MedDRA version: 14.1;Level: LLT;Classification code 10049485;Term: Bruton's agammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: NewGAM Product Code: NewGam Other descriptive name: IMMUNOGLOBULIN G | Octapharma AG | NULL | NA | Female: yes Male: yes | 20 | United States | ||||
| 462 | EUCTR2016-000468-41-DE (EUCTR) | 20/01/2021 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d- activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI | APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Leniolisib Product Code: CDZ173 INN or Proposed INN: leniolisib | Pharming Technologies B.V. | NULL | NA | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | |||
| 463 | EUCTR2018-000338-36-PL (EUCTR) | 06/11/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | NA | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Belgium;Poland;Australia;Tunisia;Norway;Germany;United Kingdom;Italy;India | |||
| 464 | EUCTR2020-003445-11-CZ (EUCTR) | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Belgium;Poland;Germany;Netherlands | |||
| 465 | EUCTR2020-004734-37-SK (EUCTR) | 02/06/2021 | Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases | Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases | Primary Immunodeficiency Diseases MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Newnorm INN or Proposed INN: Newnorm Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | NA | Female: yes Male: yes | 50 | Phase 1;Phase 3 | United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany | |||
| 466 | EUCTR2018-003199-10-DE (EUCTR) | 04/10/2018 | OPEN-LABEL STUDY: THERAPEUTIC USE OF TADEKINIG ALFA IN NLRC4 MUTATION AND XIAP DEFICIENCY | Open-label extension study with Tadekinig alfa (r-hIL-18BP) to monitor safety and tolerability in patients with IL-18 driven monogenic autoinflammatory conditions: NLRC4 mutation and XIAP deficiency | NLRC4 mutation XIAP deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Tadekinig alfa Product Code: r-hIL-18BP INN or Proposed INN: Tadekinig alfa Other descriptive name: TADEKINIG ALFA | AB2 Bio Ltd. | NULL | NA | Female: yes Male: yes | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Canada;Germany | |||
| 467 | EUCTR2016-003438-26-SK (EUCTR) | 01/02/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | |||
| 468 | EUCTR2020-004734-37-PL (EUCTR) | 16/06/2021 | Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases | Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases | Primary Immunodeficiency Diseases MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Newnorm INN or Proposed INN: Newnorm Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | NA | Female: yes Male: yes | 50 | Phase 1;Phase 3 | United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany | |||
| 469 | EUCTR2016-003799-33-Outside-EU/EEA (EUCTR) | 30/01/2017 | Safety and tolerability of higher infusion parameters of IgPro20 (Hizentra) in subjects with primary immunodeficiency (PID) | An open-label multicenter study to evaluate the safety and tolerability of higher infusion parameters of immune globulin subcutaneous (human), 20% liquid (Hizentra®) in subjects with primary immunodeficiency | Primary immunodeficiency MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra Product Name: Hizentra; human normal immunoglobulin (subcutaneous) Product Code: IgPro20 INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring LLC | NULL | NA | Female: yes Male: yes | 51 | Phase 4 | United States;Canada | |||
| 470 | EUCTR2009-011152-22-FR (EUCTR) | 09/12/2010 | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the lentiviral vector w1.6_hWASP_WPRE (VSVg) in up to 5 patients with WAS. | Product Name: Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy Product Code: GTG003.08 | GENETHON | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | France | |||
| 471 | EUCTR2014-003772-23-Outside-EU/EEA (EUCTR) | 14/04/2015 | Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID) | A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID) | Primary Immune Deficiency (Common Variable Immunodeficiency and X-linked agammaglobulinemia) MedDRA version: 17.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Privigen® Product Name: Privigen® INN or Proposed INN: Human Normal Immunoglobulin G (IgG > 98% purity) Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring AG | NULL | NA | Female: yes Male: yes | 55 | United States | ||||
| 472 | EUCTR2010-023483-41-Outside-EU/EEA (EUCTR) | 11/04/2014 | A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune system | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | a primary immunodeficiency as defined by the ESID and validated by a reference centre :• X-linked agammaglobulinemia (XLA)• Common variable immunodeficiency (CVID) MedDRA version: 16.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 100000004870 MedDRA version: 16.1;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;Classification code 10001471;Term: Agammaglobulinemia;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | NA | Female: yes Male: yes | 60 | Poland;Serbia;Czech Republic;France;Hungary;Lithuania;Ukraine | ||||
| 473 | EUCTR2014-003609-14-Outside-EU/EEA (EUCTR) | 05/01/2015 | Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring | NULL | NA | Female: yes Male: yes | 23 | Japan | ||||
| 474 | EUCTR2014-003608-61-Outside-EU/EEA (EUCTR) | 05/01/2015 | Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study) | A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency | Primary Immune Deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring | NULL | NA | Female: yes Male: yes | 25 | Japan | ||||
| 475 | EUCTR2014-003605-15-Outside-EU/EEA (EUCTR) | 26/02/2015 | Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID) | A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency (PID) MedDRA version: 17.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring AG | NULL | NA | Female: yes Male: yes | 21 | United States | ||||
| 476 | EUCTR2016-001631-12-Outside-EU/EEA (EUCTR) | 30/01/2017 | The pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiency | Prospective open-label single-arm study of the pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiency | Primary immunodeficiency MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Privigen Product Name: Privigen; immunoglobulin intravenous (human) Product Code: IgPro10 INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring KK | NULL | NA | Female: yes Male: yes | 10 | Phase 3 | Japan | |||
| 477 | EUCTR2015-005241-31-PL (EUCTR) | 01/07/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;India;Poland;Belgium;Australia;Germany;Norway;Tunisia | |||
| 478 | EUCTR2014-003607-30-Outside-EU/EEA (EUCTR) | 26/02/2015 | Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy | A Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunodeficiency (PID) | Primary Immune Deficiency MedDRA version: 17.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® Product Name: IgPro20 Product Code: IgPro20 INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | CSL Behring AG | NULL | NA | Female: yes Male: yes | 49 | Phase 3 | United States | |||
| 479 | EUCTR2009-011434-10-Outside-EU/EEA (EUCTR) | 27/01/2012 | Study to evaluate the efficacy, metabolism and safety of human immune globulin in patients with primary immunodeficiency diseases. | CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency diseases MedDRA version: 14.1;Level: LLT;Classification code 10049485;Term: Bruton's agammaglobulinemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10010112;Term: Common variable immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: NewGam Product Code: NewGam Other descriptive name: Immunoglobulin G | OCTAPHARMA AG | NULL | NA | Female: yes Male: yes | 51 | United States | ||||
| 480 | EUCTR2011-005679-18-Outside-EU/EEA (EUCTR) | 07/12/2011 | A research study to determine if Gammaplex®, is safe, tolerable, and effective when given to children and adolescents who have Primary Immunodeficiency Diseases (PID) | A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents - n/a | Primary immunodeficiency diseases MedDRA version: 14.0;Level: SOC;Classification code 10005329;Term: Blood and lymphatic system disorders;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Gammaplex Product Name: Gammaplex Product Code: n/a INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Bio Products Laboratory Limited | NULL | NA | Female: yes Male: yes | 25 | Phase 4 | Chile;United States | |||
| 481 | EUCTR2014-003409-13-Outside-EU/EEA (EUCTR) | 05/01/2015 | Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects with Primary Immunodeficiency (Japan Study) | A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects with Primary Immunodeficiency | Primary Immune Deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Hizentra® INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: Human Normal Immunoglobulin | CSL Behring | NULL | NA | Female: yes Male: yes | 22 | Japan | ||||
| 482 | EUCTR2015-003290-15-PL (EUCTR) | 15/12/2016 | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency | A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503 | Primary Immunodeficiency (PI) diseases MedDRA version: 20.1;Level: LLT;Classification code 10045792;Term: Unspecified disorder of immune mechanism;System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) Product Code: IGSC 20% INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Germany;United Kingdom |