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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCT2031210309
20/12/202213/09/2021RALPIDPhase 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 KohsukeNULLRecruiting>= 2month oldNot applicableBoth10Phase 2Japan
2NCT05104723
(ClinicalTrials.gov)
March 24, 20222/11/2021Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory ComplicationsA Phase 1/2 Open-label Study to Evaluate the Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory ComplicationsChronic Granulomatous Disease;Inflammatory Gastrointestinal Disease;Inflammatory Skin Disease;Inflammatory Lung DiseaseDrug: XELJANZ (tofacitinib)National Institute of Allergy and Infectious Diseases (NIAID)NULLEnrolling by invitation18 YearsN/AAll20Phase 1/Phase 2United States
3NCT04370795
(ClinicalTrials.gov)
March 24, 202230/4/2020Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusMatched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusSevere 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)NULLEnrolling by invitation3 Years40 YearsAll30Phase 1/Phase 2United States
4NCT05189925
(ClinicalTrials.gov)
March 24, 202212/1/2022NADPH 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;InfectionBiological: gp91 GransNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting18 Years75 YearsMale25Phase 1United States
5EUCTR2021-000227-13-BG
(EUCTR)
07/03/202202/12/2021A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom
6NCT05259917
(ClinicalTrials.gov)
March 1, 20224/2/2022A 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 IIHereditary AngioedemaDrug: Placebo;Drug: KVD900 600 mg;Drug: KVD900 300 mgKalVista Pharmaceuticals, Ltd.NULLNot yet recruiting12 YearsN/AAll114Phase 3NULL
7NCT05070455
(ClinicalTrials.gov)
March 1, 202223/9/2021An 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 DeficiencyBiological: Asceniv™ADMA Biologics, Inc.NULLNot yet recruiting2 Years11 YearsAll12Phase 4NULL
8NCT04965597
(ClinicalTrials.gov)
March 202221/6/2021Treosulfan-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 DiseasesBone 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 SyndromeDrug: 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 AssessmentFred Hutchinson Cancer Research CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States
9NCT05047185
(ClinicalTrials.gov)
March 20227/9/2021Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type IIA 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 DeficiencyDrug: PHA-022121;Drug: PlaceboPharvaris Netherlands B.V.NULLRecruiting18 Years75 YearsAll30Phase 2United States;Canada;United Kingdom
10EUCTR2021-003327-15-NL
(EUCTR)
21/02/202219/01/2022Thymosin-a1 in immunodeficiency associated mood disordersAn 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 MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Netherlands
11EUCTR2021-000227-13-PL
(EUCTR)
02/02/202218/11/2021A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom
12NCT04513002
(ClinicalTrials.gov)
February 202226/7/2020Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of AnaplerosisA 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 DeprivationAtaxia TelangiectasiaDietary Supplement: TriheptanoinThe University of QueenslandNational Health and Medical Research Council, AustraliaNot yet recruitingN/AN/AAll30Phase 2Australia
13JPRN-jRCT2031210457
24/01/202227/11/2021A 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 AtsushiNULLRecruiting>= 2age oldNot applicableBoth15Phase 3Japan
14NCT05150340
(ClinicalTrials.gov)
January 24, 202224/11/2021A 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-771TakedaNULLRecruiting2 YearsN/AAll15Phase 3Japan
15NCT05256017
(ClinicalTrials.gov)
December 30, 202117/1/2022Safety and Tolerability Study of Acoziborole in g-HAT Seropositive SubjectsSafety and Tolerability Study of Acoziborole in g-HAT Seropositive Non-parasitologically Confirmed Subjects: a Multicentre Randomised Double-blind Placebo-controlled StudyTrypanosomiasis, African;Trypanosoma Brucei Gambiense; Infection;Sleeping SicknessDrug: Acoziborole;Drug: PlaceboDrugs for Neglected DiseasesNULLRecruiting15 YearsN/AAll1200Phase 2/Phase 3Congo, The Democratic Republic of the;Guinea
16EUCTR2021-005001-26-NL
(EUCTR)
24/12/202108/12/2021The effect of high dose versus low dose antibody supplementation on airway disease in primary antibody defciencyInfluencing 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 immunoglobulinUMC UtrechtNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 4Netherlands
17EUCTR2020-004734-37-DE
(EUCTR)
14/12/202131/05/2021Prospective, 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.HNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 1;Phase 3United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany
18NCT05078879
(ClinicalTrials.gov)
November 16, 202114/10/2021Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 DeficiencyA Phase 1 Study of Empagliflozin as Treatment for Severe Congenital Neutropenia Due to G6PC3 DeficiencyCrohn's Disease;Glycogen Metabolism;Inflammatory Bowel Disease (IBD)Drug: EmpagliflozinNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting18 YearsN/AAll10Phase 1United States
19EUCTR2021-000136-59-BG
(EUCTR)
12/11/202105/10/2021A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 2United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand
20EUCTR2021-000136-59-IT
(EUCTR)
21/10/202127/07/2021A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 2United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand
21EUCTR2021-000136-59-CZ
(EUCTR)
13/10/202125/08/2021A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 2United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand
22NCT05055258
(ClinicalTrials.gov)
September 27, 202114/9/2021A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or IIRandomized, 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 IIAngioedema, Hereditary, Types I and IIDrug: KVD824;Drug: Placebo to KVD824KalVista Pharmaceuticals, Ltd.NULLRecruiting18 YearsN/AAll48Phase 2United States;Australia;Canada;Czechia;France;Germany;Hungary;Italy;New Zealand;Puerto Rico;United Kingdom
23EUCTR2021-000136-59-HU
(EUCTR)
24/09/202116/07/2021A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 2United States;Czechia;North Macedonia;Italy;United Kingdom;France;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand
24EUCTR2021-000136-59-DE
(EUCTR)
20/09/202122/07/2021A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 2United States;Czechia;North Macedonia;Italy;United Kingdom;France;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand
25NCT04638517
(ClinicalTrials.gov)
September 7, 20214/11/2020The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary FibrosisThe TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary FibrosisPulmonary Fibrosis;Telomere Shortening;Telomere Disease;Dyskeratosis CongenitaDrug: Danazol;Drug: PlaceboThe University of QueenslandNULLRecruiting5 YearsN/AAll50Phase 2Australia
26NCT03597594
(ClinicalTrials.gov)
September 2, 202119/6/2018Haplocompatible 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 ImmunodeficiencyDevice: CliniMACS;Other: Donor Lymphocyte Infusion;Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: ThiotepaSt. Jude Children's Research HospitalNULLRecruiting2 MonthsN/AAll42Phase 1/Phase 2United States
27NCT04640142
(ClinicalTrials.gov)
August 4, 202117/11/2020Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency DiseasesProspective, 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 DiseasesPrimary Immune DeficiencyBiological: NewnormOctapharmaNULLRecruiting2 Years75 YearsAll50Phase 3United States;Germany;Poland;Slovakia;Ukraine
28NCT04986735
(ClinicalTrials.gov)
August 1, 202127/7/2021Prospective Cohort Study of Children With GSD1b Receiving EmpagliflozinEvaluation of Safety and Efficacy of Empagliflozin for Neutropenia and Neutrophil Dysfunction in Children With Glycogen Storage Disease Type 1b (GSD1b)Glycogen Storage Disease Type IBDrug: EmpagliflozinHong Kong Children's HospitalNULLNot yet recruiting6 Months18 YearsAll11NULL
29NCT02860559
(ClinicalTrials.gov)
August 202126/7/2016Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined ImmunodeficiencySafety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined ImmunodeficiencySevere Combined ImmunodeficiencyBiological: TBX-1400Taiga Biotechnologies, Inc.NULLNot yet recruiting1 Month4 YearsAll8Phase 1Israel
30NCT04554914
(ClinicalTrials.gov)
July 14, 202114/9/2020A Study to Evaluate Tabelecleucel in Participants With Epstein-barr Virus-associated DiseasesAn Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects With Epstein-Barr Virus-associated DiseasesEpstein-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, HemophagocyticBiological: TabelecleucelAtara BiotherapeuticsNULLRecruitingN/AN/AAll228Phase 2United States;Austria;Belgium;France;Italy;Spain;United Kingdom
31NCT04925375
(ClinicalTrials.gov)
July 14, 202111/5/2021Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung DiseaseAbatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung DiseaseInterstitial Lung Disease;Common Variable ImmunodeficiencyDrug: Abatacept;Other: PlaceboChildren's Hospital Medical Center, CincinnatiBristol-Myers SquibbRecruiting4 YearsN/AAll60Phase 2United States
32NCT04887311
(ClinicalTrials.gov)
July 20215/5/2021MBM-01 (Tempol) for the Treatment of Ataxia TelangiectasiaAn Open Label Study to Assess the Safety and Efficacy of MBM-01 for the Treatment of Ataxia TelangiectasiaAtaxia Telangiectasia Louis-Bar;Ataxia Telangiectasia in Children;Ataxia TelangiectasiaDrug: MBM-01Matrix Biomed, Inc.The University of Texas Health Science Center, HoustonNot yet recruiting4 YearsN/AAll20Phase 2United States
33NCT04902807
(ClinicalTrials.gov)
June 202121/5/2021Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and InflammationConception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and InflammationAutoimmune 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;IBDBiological: Collection of samplesInstitut National de la Santé Et de la Recherche Médicale, FranceNULLNot yet recruiting1 Year18 YearsAll500NULL
34EUCTR2018-000338-36-IT
(EUCTR)
12/05/202104/06/2021Open-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
155Phase 3United States;Spain;Poland;Belgium;Australia;Norway;Germany;Tunisia;United Kingdom;Italy;India
35EUCTR2020-001496-32-PT
(EUCTR)
11/05/202111/08/2020Study, 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.ANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;Hungary;Slovakia;Russian Federation;Italy
36NCT04842643
(ClinicalTrials.gov)
April 27, 202111/4/2021A 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 DiseaseBiological: IGSC 20% infusionTakedaNULLRecruiting2 YearsN/AAll10Phase 3Japan
37EUCTR2020-003445-11-BG
(EUCTR)
14/04/202102/03/2021A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
72Phase 2United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands
38EUCTR2020-003445-11-NL
(EUCTR)
14/04/202101/12/2020A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
72Phase 2United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany
39NCT04944979
(ClinicalTrials.gov)
March 31, 202111/6/2021Clinical 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 DiseaseBiological: Kedrion IVIG 10%Kedrion S.p.A.NULLRecruiting2 Years16 YearsAll30Phase 3Hungary;Italy;Slovakia
40EUCTR2020-003445-11-DE
(EUCTR)
26/02/202111/02/2021A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
72Phase 2United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands
41EUCTR2020-003445-11-PL
(EUCTR)
23/02/202103/12/2020A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
72Phase 2United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands
42EUCTR2021-000515-24-NL
(EUCTR)
15/02/202105/02/2021Vaccination Against Covid in Primary Immune DeficienciesVaccination 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 ModernaErasmus University Medical CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
700Phase 4Netherlands
43ChiCTR2100043049
2021-02-042021-02-04The application of T/B cell antigen-treated shear ring (Trecs/Krecs) quantitative PCR detection in neonatal screening for primary immunodeficiency diseaseThe application of T/B cell antigen-treated shear ring (Trecs/Krecs) quantitative PCR detection in neonatal screening for primary immunodeficiency disease Severe combined immunodeficiencyGold 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 HospitalNULLRecruitingBothTarget condition:20000;Difficult condition:0China
44NCT04618211
(ClinicalTrials.gov)
February 3, 202126/10/2020Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary AngioedemaA 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 IIHereditary 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 DeficiencyDrug: PHA-022121;Drug: PlaceboPharvaris Netherlands B.V.NULLRecruiting18 Years75 YearsAll72Phase 2United States;Bulgaria;Canada;Czechia;France;Germany;Hungary;Israel;Italy;Netherlands;Poland;Spain;United Kingdom
45EUCTR2020-003445-11-HU
(EUCTR)
27/01/202130/11/2020A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
54Phase 2France;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
46EUCTR2016-000468-41-FR
(EUCTR)
12/01/202102/12/2020Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
42Phase 2;Phase 3United States;Belarus;France;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy
47NCT04566692
(ClinicalTrials.gov)
January 5, 202122/9/2020A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary ImmunodeficiencyA 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 ImmunodeficiencyPrimary ImmunodeficiencyBiological: IGSC 20%Grifols Therapeutics LLCNULLActive, not recruiting6 Years75 YearsAll33Phase 4United States
48EUCTR2019-001153-10-GB
(EUCTR)
21/12/202029/06/2020A 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of
49EUCTR2020-001496-32-IT
(EUCTR)
21/12/202021/10/2020Study, 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.ANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;Hungary;Slovakia;Italy
50EUCTR2020-003445-11-FR
(EUCTR)
18/12/202022/10/2020A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
54Phase 2France;Czech Republic;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
51EUCTR2018-000338-36-NO
(EUCTR)
06/11/202004/02/2020Not ApplicableOpen-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
155Phase 3United States;Spain;Poland;Belgium;Australia;Tunisia;Germany;Norway;United Kingdom;Italy;India
52NCT04691622
(ClinicalTrials.gov)
October 26, 202028/12/2020Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised HostsAdoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised HostsViral Infection;Hematopoietic Stem Cell Transplantation (HSCT);Primary Immunodeficiency Disorders (PID)Biological: Norovirus -specific T-cell (NST) therapyChildren's National Research InstituteNULLRecruiting3 Months80 YearsAll48Phase 1United States
53EUCTR2019-001153-10-DE
(EUCTR)
20/10/202026/03/2020A 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
28Phase 3Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Korea, Republic of;United States;Spain;Turkey;Austria;Israel
54NCT04154488
(ClinicalTrials.gov)
October 16, 20204/11/2019A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia DisordersA Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia DisordersNeutropeniaDrug: MavorixaforX4 PharmaceuticalsNULLRecruiting12 YearsN/AAll25Phase 1United States
55EUCTR2019-001153-10-AT
(EUCTR)
16/10/202024/03/2020A 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of
56NCT04561115
(ClinicalTrials.gov)
October 6, 202010/9/2020A 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 ImmunodeficiencyA 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 ImmunodeficiencyPrimary ImmunodeficiencyBiological: Gamunex-C;Biological: IVIG-PEGGrifols Therapeutics LLCNULLActive, not recruiting18 Years75 YearsAll36Phase 3United States
57EUCTR2020-001496-32-SK
(EUCTR)
01/10/202002/07/2020Study, 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.ANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;Hungary;Slovakia;Russian Federation;Italy
58EUCTR2019-002343-14-NL
(EUCTR)
25/09/202026/07/2019GENE THERAPY IN CHILDREN WITH RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCYPHASE 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 CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 1;Phase 2Netherlands
59NCT04339777
(ClinicalTrials.gov)
September 22, 20208/4/2020Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency DiseasesA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency DiseasesLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency DisordersDrug: 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)NULLSuspended4 Years69 YearsAll10Phase 2United States
60EUCTR2020-001496-32-HU
(EUCTR)
09/09/202002/07/2020Study, 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.ANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;Hungary;Slovakia;Russian Federation;Italy
61EUCTR2019-001153-10-NL
(EUCTR)
09/09/202006/04/2020A 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Korea, Republic of
62ChiCTR2000032139
2020-09-012020-04-20Newborn screening and precision medical system construction for primary immunodeficiency diseaseNewborn screening and precision medical system construction for primary immunodeficiency disease primary immunodeficiencyGold 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 UniversityNULLRecruiting01BothTarget condition:50000;Difficult condition:0China
63NCT04528355
(ClinicalTrials.gov)
August 20, 202018/8/2020Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICA 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 ConditionsDrug: data collectionPaul SzabolcsNULLRecruiting2 Months60 YearsAll50United States
64NCT04346108
(ClinicalTrials.gov)
August 11, 202010/4/2020A 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 ShireNULLCompleted2 YearsN/AAll17Phase 3Japan
65NCT04414046
(ClinicalTrials.gov)
July 22, 202024/5/2020TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in ChildrenStudy 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 ChildrenPrimary Immune Deficiency Disorders;Metabolic DiseaseBiological: Haploidentical Hematopoietic Cell TransplantationJohns Hopkins All Children's HospitalNULLRecruitingN/A21 YearsAll17Phase 2United States
66EUCTR2020-000517-33-GB
(EUCTR)
14/07/202021/02/2020Gene 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 GeneGene 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 1;Phase 2United States;Spain;United Kingdom
67EUCTR2018-004407-39-DE
(EUCTR)
26/06/202005/02/2020A 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Spain;Germany;United Kingdom
68EUCTR2018-000338-36-GB
(EUCTR)
27/05/202020/01/2020Not ApplicableOpen-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
51Phase 3United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India
69NCT04400045
(ClinicalTrials.gov)
May 22, 202019/5/2020Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage SyndromeClinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage SyndromeNijmegen Breakage SyndromeDrug: TreosulfanFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting3 Months21 YearsAll10Phase 2Russian Federation
70NCT03879876
(ClinicalTrials.gov)
May 13, 202015/3/2019Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID PatientsA 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 PatientsPediatric Patients;Any Type of Severe Combined Immunodeficiency (SCID);Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)Drug: Human T Lymphoid Progenitor (HTLP) injectionAssistance Publique - Hôpitaux de ParisNULLRecruiting2 Years18 YearsAll12Phase 1/Phase 2France
71EUCTR2019-001153-10-PL
(EUCTR)
27/04/202009/03/2020A 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
28Phase 3United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Poland;Australia;Denmark;Germany;Netherlands;Korea, Republic of
72EUCTR2019-001153-10-ES
(EUCTR)
02/03/202002/12/2019A 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 IncorporatedNULLAuthorised-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): noPortugal;United States;Spain;Turkey;Austria;Israel;Italy;United Kingdom;France;Hungary;European Union;Canada;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;Korea, Republic of
73EUCTR2019-001153-10-IT
(EUCTR)
18/02/202017/06/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Spain;Turkey;Austria;Israel;Russian Federation;United Kingdom;Italy;France;Hungary;Canada;Korea, Republic of;Germany;Netherlands;Denmark;Australia;Poland
74EUCTR2019-001693-28-DE
(EUCTR)
06/02/202002/09/2019Single-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 olderProspective, 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.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Belarus;Ukraine;Russian Federation;Germany
75EUCTR2019-001153-10-FR
(EUCTR)
05/02/202016/10/2019A 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Portugal;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Hungary;European Union;Canada;Belgium;Poland;Romania;Australia;Denmark;Germany;Netherlands;Korea, Republic of
76NCT04275479
(ClinicalTrials.gov)
January 10, 202010/2/2020Diabetes/ Endocrine Surveillance in SDSEndocrine Diabetes Screening in Patients With Shwachman-Diamond Syndrome DIABETES/ ENDOCRINE SURVEILLANCE IN SDSShwachman-Diamond SyndromeDiagnostic 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 QuestionnairesWashington University School of MedicineShwachman Diamond Syndrome Foundation;Barnes-Jewish HospitalRecruiting3 YearsN/AAll60United States
77EUCTR2019-001153-10-HU
(EUCTR)
08/01/202008/11/2019A 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
28Phase 3United States;Portugal;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Hungary;European Union;Canada;Poland;Belgium;Romania;Australia;Denmark;Germany;Netherlands;Korea, Republic of
78NCT03759678
(ClinicalTrials.gov)
January 8, 202027/11/2018N-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 StudyAtaxia Telangiectasia;Louis Bar SyndromeDrug: IB1001IntraBio IncNULLRecruiting6 YearsN/AAll39Phase 2United States;Germany;Spain;United Kingdom
79EUCTR2019-001153-10-DK
(EUCTR)
16/12/201921/10/2019A 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 3United States;Spain;Austria;Israel;Russian Federation;Italy;United Kingdom;France;Hungary;Poland;Denmark;Australia;Germany;Netherlands;Korea, Republic of
80NCT03814798
(ClinicalTrials.gov)
December 201922/1/2019Study Evaluating IGSC 20% Flexible Dosing in Treatment-Experienced and Treatment-Naive Subjects With Primary ImmunodeficiencyA 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 PIPrimary ImmunodeficiencyBiological: 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/kgGrifols Therapeutics LLCNULLWithdrawn2 Years75 YearsAll0Phase 3United States
81NCT04371939
(ClinicalTrials.gov)
November 5, 201929/4/2020Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich SyndromeRandomized, 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 SyndromeWiskott-Aldrich SyndromeDrug: Romiplostim;Drug: EltrombopagFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruitingN/A18 YearsAll30Phase 2Russian Federation
82NCT03910452
(ClinicalTrials.gov)
October 28, 20199/4/2019Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cellNational Institute of Allergy and Infectious Diseases (NIAID)NULLSuspended4 Years65 YearsAll4Early Phase 1United States
83NCT03995108
(ClinicalTrials.gov)
October 17, 201919/6/2019Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) SyndromeA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label ExtensionWHIM SyndromeDrug: Mavorixafor;Drug: PlaceboX4 PharmaceuticalsNULLActive, not recruiting12 YearsN/AAll28Phase 3United States;Australia;Austria;Denmark;France;Hungary;Israel;Italy;Korea, Republic of;Netherlands;Russian Federation;Spain;Turkey
84NCT03939533
(ClinicalTrials.gov)
October 17, 20193/5/2019Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency DiseasesClinical 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 DiseasesPrimary Immune Deficiency DisorderDrug: CUTAQUIGOctapharmaNULLCompleted2 Years75 YearsAll64Phase 3United States
85EUCTR2018-003297-27-DE
(EUCTR)
15/10/201928/03/2019Therapeutic use of Tadekinig alfa in NLRC4 mutation and XIAP deficiencyMulticenter, 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 3United States;Canada;Germany
86NCT04140539
(ClinicalTrials.gov)
October 15, 201924/10/2019A Clinical Study to Enable Process Validation of Commercial Grade OTL-101A 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 DeficiencyBiological: OTL-101Orchard TherapeuticsUniversity of California, Los AngelesTerminatedN/A17 YearsAll3Phase 2/Phase 3United States
87EUCTR2018-004489-32-AT
(EUCTR)
02/10/201907/03/2019A 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom
88NCT03974178
(ClinicalTrials.gov)
September 29, 201923/5/2019Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei RhodesienseEfficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense: a Multicentre, Open-label Clinical TrialTrypanosoma Brucei Rhodesiense; InfectionDrug: FexinidazoleDrugs for Neglected DiseasesEuropean and Developing Countries Clinical Trials Partnership (EDCTP)Recruiting6 YearsN/AAll50Phase 2/Phase 3Malawi;Uganda
89NCT04049084
(ClinicalTrials.gov)
September 26, 20196/8/2019An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDAn 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 TherapeuticsUniversity of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation TrustEnrolling by invitationN/AN/AAll70United States;United Kingdom
90EUCTR2018-004407-39-GB
(EUCTR)
18/09/201906/08/2019A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-TelangiectasiaEffects 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.NULLAuthorised-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): noUnited States;Germany;United Kingdom
91NCT03812263
(ClinicalTrials.gov)
August 30, 201918/1/2019A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-IGene 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 GeneLeukocyte Adhesion Defect - Type IBiological: RP-L201Rocket Pharmaceuticals Inc.California Institute for Regenerative Medicine (CIRM)Active, not recruiting3 MonthsN/AAll9Phase 1/Phase 2United States;Spain;United Kingdom
92EUCTR2018-004489-32-PL
(EUCTR)
29/08/201929/05/2019A 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom
93EUCTR2016-003438-26-HU
(EUCTR)
28/08/201920/06/2019Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Czech Republic;Hungary;Slovakia;Greece;Denmark;United Kingdom;Sweden
94NCT03984890
(ClinicalTrials.gov)
August 1, 20195/12/2018Vitamin D3 For CGD Patients With BCGosis/ItisEffect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/ItisVitamin D3;Chronic-granulomatous Disease;BCGDrug: Vitamin D3;Drug: Traditional treatment of CGD and TBChildren's Hospital of Fudan UniversityNULLRecruitingN/A18 YearsAll50Phase 2/Phase 3China
95EUCTR2018-004489-32-IT
(EUCTR)
23/07/201905/01/2021Uno 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 LtdNULLNot RecruitingFemale: yes
Male: yes
60Phase 2France;United States;Czechia;Hungary;Poland;Austria;Netherlands;Germany;United Kingdom;Italy
96NCT04208412
(ClinicalTrials.gov)
July 2, 201920/12/2019A 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 IIA 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 IIHereditary AngioedemaDrug: KVD900;Drug: PlaceboKalVista Pharmaceuticals, Ltd.NULLCompleted18 YearsN/AAll68Phase 2United States;Austria;Czechia;Germany;Hungary;Italy;Netherlands;North Macedonia;Poland;United Kingdom
97NCT03878069
(ClinicalTrials.gov)
June 25, 201912/3/2019Registry Study of Revcovi Treatment in Patients With ADA-SCIDSingle Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement TherapyAdenosine Deaminase Deficiency;Severe Combined ImmunodeficiencyBiological: elapegademase-lvlrChiesi Farmaceutici S.p.A.NULLRecruitingN/A65 YearsAll20United States
98EUCTR2018-004489-32-DE
(EUCTR)
24/06/201912/02/2019A 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Hungary;Poland;Austria;North Macedonia;Netherlands;Germany;Italy;United Kingdom
99EUCTR2018-004489-32-NL
(EUCTR)
19/06/201925/03/2019A 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Hungary;Poland;Austria;North Macedonia;Germany;Netherlands;Italy;United Kingdom
100NCT04870866
(ClinicalTrials.gov)
June 5, 201923/4/2021NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia TelangiectasiaNAD Supplementation to Prevent Progressive Neurological Disease in Ataxia TelangiectasiaAtaxia TelangiectasiaDrug: Nicotinamide ribonucleosideUniversity Hospital, AkershusThe 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 BergenActive, not recruiting3 YearsN/AAll13Phase 2Norway
101EUCTR2018-004489-32-HU
(EUCTR)
21/05/201925/03/2019A 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 2Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom
102NCT03961009
(ClinicalTrials.gov)
April 30, 201921/5/2019Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID PatientsA Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety and Pharmacokinetics of Kedrion Intravenous Immunoglobulin (IVIg) 10% in Primary Immunodeficiency Disease (PID) PatientsPrimary Immunodeficiency DiseaseBiological: Kedrion IVIG 10%Kedrion S.p.A.NULLCompleted2 Years70 YearsAll47Phase 3United States;Canada
103NCT03825783
(ClinicalTrials.gov)
April 15, 201925/1/2019A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-IGene 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 IBiological: RP-L201Rocket Pharmaceuticals Inc.NULLRecruiting3 MonthsN/AAll2Phase 1Spain
104NCT03547830
(ClinicalTrials.gov)
April 13, 201924/5/2018Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD PatientsA Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Plerixafor;Drug: GcsfFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting1 Month24 YearsAll17Phase 2Russian Federation
105EUCTR2018-004191-35-BE
(EUCTR)
12/04/201905/03/2019Use 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-LucNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 4Belgium
106NCT03962114
(ClinicalTrials.gov)
March 18, 201911/4/2019Effects of Vitamin B3 in Patients With Ataxia TelangiectasiaEffects of Nicotinamide Riboside (Vitamin B3) in Patients With Ataxia Telangiectasia.Ataxia Telangiectasia;ATM Gene MutationDietary Supplement: Vitamin B3Radboud UniversityA-T childrens project;Twan foundationEnrolling by invitation2 YearsN/AAll24Phase 2Netherlands
107EUCTR2018-004489-32-GB
(EUCTR)
13/03/201927/12/2018A 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 LtdNULLAuthorised-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): noUnited States;Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom
108EUCTR2016-000468-41-GB
(EUCTR)
25/02/201912/03/2018Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2;Phase 3United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Italy;United Kingdom
109EUCTR2018-003842-18-IT
(EUCTR)
08/01/201919/11/2018Gene 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
6Phase 3Italy
110NCT03601286
(ClinicalTrials.gov)
December 21, 201822/2/2018Lentiviral Gene Therapy for X-linked Severe Combined ImmunodeficiencyPhase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted BusulfanSevere Combined Immunodeficiency, X-LinkedDrug: Lentiviral vector transduced CD34+ cellsGreat Ormond Street Hospital for Children NHS Foundation TrustNULLRecruiting8 Weeks5 YearsMale5Phase 1United Kingdom
111EUCTR2018-002680-26-ES
(EUCTR)
27/11/201802/08/2018Gene 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 GeneGene 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.NULLNot RecruitingFemale: yes
Male: yes
2Phase 1Spain
112EUCTR2016-000468-41-IE
(EUCTR)
25/10/201827/02/2018Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
42Phase 2;Phase 3France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy
113EUCTR2018-001029-14-FR
(EUCTR)
25/10/201802/07/2018HTLPA 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)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 1;Phase 2France
114NCT03619551
(ClinicalTrials.gov)
October 22, 201816/7/2018Conditioning SCID Infants Diagnosed EarlyA 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)SCIDDrug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletionMichael Pulsipher, MDNULLRecruitingN/A2 YearsAll64Phase 2United States
115EUCTR2018-000673-68-GB
(EUCTR)
09/10/201827/07/2018 A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined ImmunodeficiencyPhase 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 TrustNULLAuthorised-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): noUnited Kingdom
116NCT03716700
(ClinicalTrials.gov)
September 24, 201831/8/2018Real-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: CUVITRUBaxalta now part of ShireBaxalta Innovations GmbH, now part of ShireCompleted3 YearsN/AAll126Canada
117EUCTR2018-000338-36-DE
(EUCTR)
20/09/201821/06/2018Not ApplicableOpen-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
150Phase 3United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India
118NCT03677557
(ClinicalTrials.gov)
September 19, 20186/7/2018Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) TreatmentSafety, 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 ImmunodeficiencyDrug: 16,5% CutaquigOttawa Hospital Research InstituteOctapharmaRecruiting18 YearsN/AAll30Phase 4Canada
119NCT03663933
(ClinicalTrials.gov)
September 4, 20187/9/2018Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or DysregulationPhase II Trial of Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or DysregulationLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Primary T-cell Immunodeficiency Disorders;Immune System Diseases;Common Variable ImmunodeficiencyDrug: Immunosuppression Only Conditioning;Drug: Reduced Intensity Conditioning;Drug: GVHD Prophylaxis;Procedure: Allogeneic HSCNational Cancer Institute (NCI)NULLRecruiting4 YearsN/AAll177Phase 2United States
120EUCTR2018-000338-36-BE
(EUCTR)
23/08/201828/06/2018Not ApplicableOpen-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.NULLAuthorised-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): noUnited States;Poland;Spain;Belgium;Australia;Israel;Germany;India
121EUCTR2018-001489-41-SE
(EUCTR)
02/07/201808/05/2018A 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 UniversitetssjukhusetNULLAuthorised-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): noUnited States;Sweden
122NCT03366142
(ClinicalTrials.gov)
July 2, 20187/12/2017Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory PathologyUse of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory PathologyLAD1Drug: UstekinumabNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting12 Years65 YearsAll20Phase 1/Phase 2United States
123NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone 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;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchRecruiting3 Months39 YearsAll40Phase 1/Phase 2United States
124NCT03576469
(ClinicalTrials.gov)
June 13, 201815/6/2018A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG InfusionsA Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG InfusionsCVI - Common Variable ImmunodeficiencyBiological: C1-esterase inhibitor [recombinant] (C1-INH-R)IMMUNOe Research CentersNULLCompleted18 YearsN/AAll20Phase 4United States
125EUCTR2016-000468-41-IT
(EUCTR)
01/06/201802/09/2021Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2;Phase 3France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy
126NCT03538899
(ClinicalTrials.gov)
May 31, 20183/5/2018Autologous Gene Therapy for Artemis-Deficient SCIDA 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 CellsSevere Combined ImmunodeficiencyDrug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: BusulfanUniversity of California, San FranciscoNULLRecruiting2 MonthsN/AAll25Phase 1/Phase 2United States
127EUCTR2018-000338-36-ES
(EUCTR)
31/05/201805/04/2018Not ApplicableOpen-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
61Phase 3United States;Poland;Belgium;Spain;Australia;Israel;Norway;Germany;Italy;United Kingdom;India
128JPRN-jRCTs031180398
30/05/201820/03/2019A phase II study of RIC-SCT for CGDReduced 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 HirotoshiNULLRecruitingNot applicable< 25age oldBoth22Phase 2None (Japan only);Japan
129NCT03548818
(ClinicalTrials.gov)
May 16, 201820/3/2018Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous DiseaseRole of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Interferon Gamma-1BUniversity of Colorado, DenverNULLCompleted5 Years60 YearsAll9United States
130NCT03232203
(ClinicalTrials.gov)
April 12, 201825/7/2017Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ TreatmentSevere Combined Immunodeficiency Due to ADA DeficiencyDrug: STRIMVELISOrchard TherapeuticsNULLCompleted18 YearsN/AAll16Italy
131NCT03512314
(ClinicalTrials.gov)
January 24, 20189/4/2018Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label ExtensionOpen-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 DeficiencyXIAP Deficiency;NLRC4-MASDrug: Tadekinig alfaAB2 Bio Ltd.NULLRecruitingN/AN/AAll10Phase 3United States;Canada;Germany
132NCT03311503
(ClinicalTrials.gov)
January 19, 201812/10/2017Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningPhase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningSevere Combined Immunodeficiency, X Linked;Gene TherapyBiological: autologous CD34+ cell transduced with G2SCID vectorDavid WilliamsNULLRecruitingN/A5 YearsMale10Phase 1/Phase 2United States;United Kingdom
133JPRN-UMIN000030806
2018/01/1715/01/2018A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich SyndromeA 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 syndromeWASP 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 DevelopmentNULLComplete: follow-up continuingNot applicableNot applicableMale3Phase 1,2Japan
134NCT03765632
(ClinicalTrials.gov)
January 3, 20188/8/2018Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDEfficacy 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 DeficiencySevere Combined Immunodeficiency Due to ADA DeficiencyGenetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsActive, not recruitingN/A17 YearsAll13Phase 1/Phase 2United Kingdom
135JPRN-UMIN000030647
2017/12/3031/12/2017Reduced 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 diseaseConditioning 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 DevelopmentNULLRecruitingNot applicable25years-oldMale and Female22Phase 2Japan
136NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated 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 SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLRecruiting1 Year75 YearsAll58Phase 2United States
137NCT03330795
(ClinicalTrials.gov)
December 1, 201730/10/2017Bilateral Orthotopic Lung Transplant - Bone Marrow TransplantBilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003)Primary Immunodeficiency;PIDBiological: CD3/CD19 neg allogeneic BMTNational Institute of Allergy and Infectious Diseases (NIAID)University of Pittsburgh;PPDActive, not recruiting10 Years45 YearsAll5Phase 1/Phase 2United States
138EUCTR2016-003438-26-GR
(EUCTR)
13/11/201711/10/2017Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
139EUCTR2017-001731-39-IT
(EUCTR)
12/10/201702/07/2020Retroviral insertion site methodology studyMethodology 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) LtdNULLNot RecruitingFemale: yes
Male: yes
15Phase 4Turkey;Switzerland;Italy
140NCT03828279
(ClinicalTrials.gov)
October 1, 201723/1/2019Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and IIThe Global Registry on Hereditary Angioedema Type I and IIHereditary Angioedema Type I and IIDiagnostic Test: functional and antigenic C1 inhibitorHAE Global Registry FoundationNULLRecruitingN/AN/AAll220Italy
141JPRN-jRCT2031200236
28/09/201710/12/2020Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitisDouble-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 ToshinaoKawai Toshinao;Japan Agency for Medical Research and DevelopmentRecruiting>= 1age oldNot applicableBoth8Phase 2-3Japan
142JPRN-UMIN000029324
2017/09/2801/10/2017Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitisDouble-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis - Clinical trial of thalidomide for CGD colitis Chronic granulomatous disease associated colitisOral 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 DevelopmentNULLRecruiting1years-oldNot applicableMale and Female8Not selectedJapan
143NCT03277313
(ClinicalTrials.gov)
September 25, 20177/9/2017Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD SubjectsEfficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: HYQVIA;Biological: GAMMAGARD LIQUIDBaxalta now part of ShireBaxalta Innovations GmbH, now part of ShireActive, not recruiting2 Years15 YearsAll44Phase 3United States
144EUCTR2017-001275-23-GB
(EUCTR)
21/09/201703/07/2017A clinical trial to study the effects of genetically modified patients' CD34+ cellsEfficacy 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 TrustNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2United Kingdom
145EUCTR2015-003290-15-HU
(EUCTR)
01/09/201712/05/2017A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot 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): noFrance;Czech Republic;Hungary;Poland;Spain;Australia;Germany;United Kingdom;Sweden
146NCT03080480
(ClinicalTrials.gov)
September 1, 201727/2/2017Pioglitazone Therapy for Chronic Granulomatous DiseaseEfficacy and Safety of Pioglitazone Therapy for Chronic Granulomatous Disease Patients With Severe Infection.Chronic Granulomatous DiseaseDrug: PioglitazoneChildren's Hospital of Fudan UniversityNULLTerminated1 Month18 YearsAll3Phase 1/Phase 2China
147NCT03113760
(ClinicalTrials.gov)
July 21, 201728/2/2017Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP DeficiencyMulticenter, 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 DeficiencyNLRC4-MAS;XIAP DeficiencyDrug: Tadekinig alfa;Other: 0.9% sodium chlorideAB2 Bio Ltd.NULLRecruitingN/AN/AAll10Phase 3United States;Canada;Germany
148NCT03217617
(ClinicalTrials.gov)
July 15, 20173/7/2017Gene 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 LinkedBiological: TYF-IL-2Rg gene-modified autologous stem cellsShenzhen Geno-Immune Medical InstituteNULLRecruiting1 Month10 YearsMale10Phase 1/Phase 2China
149NCT03116347
(ClinicalTrials.gov)
June 13, 201712/4/2017Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD SubjectsPost-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: HYQVIA;Biological: KIOVIG;Biological: CuvitruBaxalta now part of ShireBaxalta Innovations GmbH, now part of ShireCompleted2 Years17 YearsAll42Phase 4Czechia;Denmark;France;Greece;Slovakia;Sweden;United Kingdom
150EUCTR2016-003438-26-CZ
(EUCTR)
18/05/201727/01/2017Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
151EUCTR2016-003438-26-DK
(EUCTR)
08/05/201719/01/2017Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
152EUCTR2016-003438-26-FR
(EUCTR)
18/04/201721/03/2017Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
153EUCTR2016-003438-26-SE
(EUCTR)
04/04/201708/11/2016Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
154NCT03988426
(ClinicalTrials.gov)
March 7, 201713/6/2019Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency DiseasesClinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases.Primary Immune Deficiency DisorderBiological: OctanormOctapharmaNULLCompleted18 Years70 YearsAll25Phase 3Russian Federation
155NCT02770807
(ClinicalTrials.gov)
March 2, 20172/5/2016EDS in Ataxia Telangiectasia PatientsMulti-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With Ataxia TelangiectasiaNervous System Disease;Genetic SyndromeDrug: EDS-EP dose range of ~5-10 mg DSP/infusion;Drug: EDS-EP dose range of ~14-22 mg DSP/infusion;Drug: PlaceboErydelNULLCompleted6 YearsN/AAll175Phase 3United States;Australia;Belgium;Germany;India;Israel;Italy;Norway;Poland;Spain;Tunisia;United Kingdom
156NCT03112655
(ClinicalTrials.gov)
February 24, 201731/3/2017Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: Early Test-of-cureDiagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: WP4 Early Test-of-cureAfrican Trypanosomiasis;African; Trypanosomiasis, West;Sleeping Sickness; West African;Trypanosoma Brucei Gambiense; InfectionDiagnostic Test: RNA and neopterin detectionInstitut de Recherche pour le DeveloppementInstitute of Tropical Medicine, Belgium;Institut National de Recherche Biomédicale. Kinshasa, République Démocratique du Congo;Ministry of Public Health, Democratic Republic of the CongoCompleted15 YearsN/AAll88N/ACongo, The Democratic Republic of the
157EUCTR2016-003438-26-GB
(EUCTR)
14/02/201701/11/2016Study 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 4France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
158EUCTR2015-003652-52-GB
(EUCTR)
02/02/201708/08/2016An 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 AGNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Spain;Russian Federation;Germany;United Kingdom
159NCT03033745
(ClinicalTrials.gov)
February 1, 201723/1/2017Safety 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 ImmunodeficiencyPrimary ImmunodeficiencyDrug: IgPro20CSL BehringNULLCompleted2 YearsN/AAll49Phase 4United States;Canada
160EUCTR2013-003877-87-SK
(EUCTR)
13/01/201717/03/2016Clinical 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.HNULLNot RecruitingFemale: yes
Male: yes
64Phase 1;Phase 3United States;Hungary;Czech Republic;Slovakia;Canada;Poland;Russian Federation
161NCT03733249
(ClinicalTrials.gov)
January 20172/11/2018Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyFollow-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 DisordersAcute 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 CellDrug: Rimiducid;Biological: rivogenlecleucelBellicum PharmaceuticalsNULLEnrolling by invitation1 Month18 YearsAll193Phase 1/Phase 2Italy;Saudi Arabia;United Kingdom
162NCT03054181
(ClinicalTrials.gov)
December 22, 201613/2/2017Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO)Facilitated Immunoglobulin Administration Registry and Outcomes StudyPrimary Immunodeficiency;Secondary Immune DeficiencyBiological: HyQviaGWT-TUD GmbHNULLCompletedN/AN/AAll156France;Germany;Italy
163NCT02999984
(ClinicalTrials.gov)
December 16, 201619/12/2016Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDEfficacy 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 DeficiencySevere Combined Immunodeficiency Due to ADA DeficiencyGenetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERTOrchard TherapeuticsCalifornia Institute for Regenerative Medicine (CIRM);University of California, Los AngelesCompletedN/A17 YearsAll10Phase 1/Phase 2United States
164NCT03037359
(ClinicalTrials.gov)
December 201620/1/2017A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin ProductsA 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 InfusionPrimary Immune Deficiency DisorderBiological: Bivigam;Biological: OtherADMA Biologics, Inc.NULLRecruitingN/AN/AAll200United States
165NCT03005327
(ClinicalTrials.gov)
December 201620/12/2016A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) SyndromeA Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM SyndromeWHIM SyndromeDrug: X4P-001X4 PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll15Phase 2United States;Australia
166EUCTR2015-005241-31-NO
(EUCTR)
21/11/201602/06/2016n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
175Phase 3United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;Norway;United Kingdom;Italy;India
167NCT03025789
(ClinicalTrials.gov)
November 17, 201611/11/2016Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any StageAn Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole in Patients With Human African Trypanosomiasis Due to T.b. Gambiense at Any StageTrypanosomiasis, African;Sleeping Sickness;Trypanosomiasis; GambianDrug: FexinidazoleDrugs for Neglected DiseasesSanofiCompleted6 YearsN/AAll174Phase 3Congo, The Democratic Republic of the;Guinea
168NCT02996448
(ClinicalTrials.gov)
November 17, 201616/12/2016Safety, Tolerability, and Immunogenicity of One Dose of NDV 3A Vaccine in People With STAT3-Mutated Hyper-IgE SyndromeA Phase 2a Study to Evaluate the Safety, Tolerability, and Immunogenicity of One Dose of NDV-3A Vaccine in Patients With STAT3-Mutated Hyper-IgE SyndromeAutosomal-dominant Hyper-IgE SyndromeDrug: NDV-3ANational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated18 Years55 YearsAll3Phase 2United States
169NCT02881437
(ClinicalTrials.gov)
November 11, 201617/8/2016IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQviaAssessment of the IgG Trough Level in Subjects With Primary Immunodeficiency Switching From Standard Subcutaneous Immunoglobulin (SCIG) to Every Other Week HyQviaPrimary ImmunodeficiencyDrug: IgHy10University Hospital, LilleShireCompleted18 YearsN/AAll22Phase 4France
170EUCTR2016-000468-41-NL
(EUCTR)
03/11/201619/09/2016Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2;Phase 3France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy
171EUCTR2015-005241-31-IT
(EUCTR)
02/11/201628/02/2018n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
180Phase 3Tunisia;United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;Belgium;Poland;Australia;Germany;Norway
172EUCTR2015-005241-31-BE
(EUCTR)
21/10/201601/06/2016n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
180Phase 3United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;United Kingdom;Italy;India;Norway
173NCT03087955
(ClinicalTrials.gov)
October 11, 20167/3/2017Prospective Study on Efficacy and Safety of Acoziborole (SCYX-7158) in Patients Infected by Human African Trypanosomiasis Due to T.b. GambienseEfficacy and Safety Study of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective StudyTrypanosomiasis, African;Gambiense Trypanosomiasis;Sleeping SicknessDrug: Acoziborole (SCYX-7158)Drugs for Neglected DiseasesNULLCompleted15 YearsN/AAll260Phase 2/Phase 3Congo, The Democratic Republic of the;Guinea
174NCT02810444
(ClinicalTrials.gov)
October 4, 201615/6/2016Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PIDAn 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 DiseaseBiological: IgG Next Generation (BT595)BiotestSyneos HealthCompleted2 Years75 YearsAll81Phase 3United States;Germany;Hungary;Russian Federation;Spain
175EUCTR2015-003652-52-DE
(EUCTR)
29/09/201618/05/2016An 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 AGNULLNot RecruitingFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;Germany;United Kingdom
176NCT02733679
(ClinicalTrials.gov)
September 29, 201631/3/2016Response of Individuals With Ataxia-Telangiectasia to Metformin and PioglitazoneResponse of Individuals With Ataxia-Telangiectasia to Metformin and PioglitazoneAtaxia-TelangiectasiaDrug: Metformin;Drug: PioglitazoneNHS TaysideUniversity of DundeeCompleted18 Years30 YearsAll27Phase 4United Kingdom
177EUCTR2015-003652-52-ES
(EUCTR)
19/09/201615/07/2016An 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 AGNULLNot 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): noUnited States;Hungary;Spain;Russian Federation;Germany;United Kingdom
178EUCTR2015-005241-31-DE
(EUCTR)
14/09/201627/05/2016n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
175Phase 3United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Norway;Germany;United Kingdom;Italy;India
179NCT01512888
(ClinicalTrials.gov)
August 17, 201613/1/2012Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed InfantsA 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 CellsSevere Combined Immunodeficiency Disease, X-linkedGenetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacsSt. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM)RecruitingN/A24 MonthsMale28Phase 1/Phase 2United States
180EUCTR2016-000468-41-CZ
(EUCTR)
19/07/201625/05/2016Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2;Phase 3France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy
181EUCTR2015-003290-15-DE
(EUCTR)
12/07/201616/03/2016A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot 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): noFrance;Hungary;Czech Republic;Poland;Spain;Australia;Germany;United Kingdom;Sweden
182EUCTR2016-001480-36-FR
(EUCTR)
11/07/201622/11/2016Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQviaAssessment 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 LilleNULLNot 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): noFrance
183EUCTR2015-003369-27-DE
(EUCTR)
01/07/201618/05/2016A 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 FreiburgNULLNot RecruitingFemale: yes
Male: yes
6Phase 2Germany
184NCT02609932
(ClinicalTrials.gov)
July 201611/11/2015Effect of IFN-? on Innate Immune CellsEffect of Interferon-gamma 1-b on Innate Immune CellsChronic Granulomatous DiseaseDrug: Administration of drug (Interferon-gamma 1-b) subcutaneouslyUniversity of Colorado, DenverNULLCompleted18 Years60 YearsAll20Phase 1United States
185NCT02822495
(ClinicalTrials.gov)
July 201630/6/2016Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or MalignanciesExpanded Access Protocol for Providing Tabelecleucel to Patients With Epstein-Barr Virus-Associated Viremia or Malignancies for Whom There Are No Appropriate Alternative TherapiesEpstein-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 ComplicationsBiological: tabelecleucelAtara BiotherapeuticsNULLAvailableN/AN/AAllUnited States
186EUCTR2015-003290-15-FR
(EUCTR)
29/06/201628/09/2018A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot 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): noFrance;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom
187EUCTR2015-003290-15-CZ
(EUCTR)
22/06/201601/02/2016A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot 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): noFrance;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom
188NCT02737384
(ClinicalTrials.gov)
June 14, 20168/4/2016Hematopoietic 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 GraftCombined ImmunodeficienciesBiological: Depletion in CD45RA graft donorAssistance Publique - Hôpitaux de ParisNULLTerminated12 Months18 YearsAll4Phase 2France
189JPRN-UMIN000022688
2016/06/1013/06/2016A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous diseaseA 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 diseaseConditioning 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 DevelopmentNULLComplete: follow-up completeNot applicable25years-oldMale and Female9Not selectedJapan
190NCT03019809
(ClinicalTrials.gov)
June 201611/1/2017A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS PatientsA 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 FailureBiological: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT.Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLUnknown status1 Month19 YearsAll30Phase 2Russian Federation
191NCT02806986
(ClinicalTrials.gov)
June 20163/6/2016Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary ImmunodeficiencyA Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: IGSC 20%Grifols Therapeutics LLCNULLCompleted2 Years75 YearsAll61Phase 3Australia;Czechia;France;Germany;Hungary;Poland;Spain;Sweden;United Kingdom;Czech Republic
192EUCTR2015-005241-31-ES
(EUCTR)
31/05/201609/06/2016n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
180Phase 3United States;Spain;Costa Rica;Turkey;Israel;United Kingdom;Italy;Belgium;Poland;Australia;Tunisia;Germany;Norway
193EUCTR2015-003290-15-GB
(EUCTR)
12/05/201613/01/2016A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Hungary;Czech Republic;Spain;Poland;Australia;Germany;United Kingdom;Sweden
194EUCTR2015-002491-24-DE
(EUCTR)
11/05/201630/03/2016Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related diseaseSafety 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 DirektorNULLNot RecruitingFemale: yes
Male: yes
10Phase 2Germany
195NCT02789397
(ClinicalTrials.gov)
May 2, 201616/5/2016Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease in Patients With Common Variable ImmunodeficiencyClinical 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 DiseaseDrug: Rituximab (RTX) and Azathioprine (AZA);Drug: PlacebosMedical College of WisconsinNULLWithdrawn18 YearsN/AAll0Phase 2NULL
196EUCTR2015-003290-15-ES
(EUCTR)
12/04/201622/02/2016A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot 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): noFrance;Hungary;Czech Republic;Poland;Spain;Germany;United Kingdom
197NCT03907241
(ClinicalTrials.gov)
March 1, 20167/12/2018CLINICAL 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 TRIALTitle 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 TRIALPrimary ImmunodeficiencyDrug: Octanorm 16.5%OctapharmaNULLCompleted2 Years75 YearsAll27Phase 3United States;Canada
198NCT02627300
(ClinicalTrials.gov)
March 20163/12/2015Study of Octanorm Subcutaneous IG in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 TrialClinical 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 TrialPrimary Immunodeficiency DiseaseDrug: Octanorm 16.5%OctapharmaNULLCompletedN/AN/AAll21Phase 3United States;Canada
199NCT02269163
(ClinicalTrials.gov)
January 26, 201616/10/2014Study 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 DiseasesPrimary ImmunodeficiencyBiological: Immune Globulin Intravenous;Biological: Prometic's Immune Globulin Intravenous 10%Prometic Biotherapeutics, Inc.Atlantic Research GroupCompleted2 Years80 YearsAll82Phase 3United States
200NCT02604810
(ClinicalTrials.gov)
January 20166/11/2015Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary ImmunodeficiencyAn Open-label, Multi-center Study to Evaluate the Safety and Pharmacokinetics of IGSC 20% Administered for 6 Months in Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: IGIV-C 10%;Biological: IGSC 20%Grifols Therapeutics LLCNULLCompleted2 Years75 YearsAll53Phase 3United States;Canada
201NCT02123615
(ClinicalTrials.gov)
January 201611/4/2014ASIS for GAMMAGARD in Primary ImmunodeficiencyASIS for GAMMAGARD in Primary ImmunodeficiencyPrimary ImmunodeficiencyDrug: 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 36ASIS CorporationNULLNot yet recruiting21 Years65 YearsBoth60Phase 1/Phase 2United States
202NCT02629120
(ClinicalTrials.gov)
December 17, 201510/12/2015High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseHigh Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseChronic Granulomatous Disease TransplantDrug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting4 Years65 YearsAll50Phase 1/Phase 2United States
203JPRN-UMIN000019532
2015/12/0101/12/2015Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseasesClinical 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 disorderfludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4Graduate Schoool,Dept. of Community Pediatrics,Perinatal and Maternal Medicine,Tokyo Medical and Dental UniversityNULLRecruitingNot applicableNot applicableMale and Female5Phase 2Japan
204NCT02579967
(ClinicalTrials.gov)
November 19, 201516/10/2015Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary ImmunodeficienciesPilot Trial of Allogeneic Blood or Marrow Transplantation for Primary ImmunodeficienciesPrimary T-cell Immunodeficiency Disorders;Common Variable Immunodeficiency;Immune System Diseases;Autoimmune Lymphoproliferative;Lymphoproliferative DisordersDrug: Immunosuppression Only Conditioning -Closed with amendment L;Drug: Reduced Intensity Conditioning;Drug: Myeloablative Conditioning-Closed with amendment L;Drug: GVHD Prophylaxis;Procedure: Allo BMTNational Cancer Institute (NCI)NULLRecruiting4 Years75 YearsAll224Phase 2United States
205NCT02593188
(ClinicalTrials.gov)
November 12, 201529/10/2015Non-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: HYQVIABaxalta now part of ShireNULLActive, not recruiting16 YearsN/AAll264United States
206NCT03055247
(ClinicalTrials.gov)
November 201521/7/2016Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGDA 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-CGDChronic Granulomatous Disease X-linked (X-CGD)Drug: Ibuprofen;Drug: Myelostim;Drug: MozobilIRCCS San RaffaeleFondazione TelethonRecruiting18 Years45 YearsMale3Phase 2Italy
207NCT02234934
(ClinicalTrials.gov)
October 29, 20154/9/2014Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous DiseaseA 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 DiseaseGranulomatous Disease, Chronic, X-linkedBiological: Lentiviral G1XCGD Gene TherapyUniversity of California, Los AngelesBoston Children's Hospital;National Heart, Lung, and Blood Institute (NHLBI);Genethon;California Institute for Regenerative Medicine (CIRM)Recruiting23 MonthsN/AMale16Phase 1/Phase 2United States
208NCT02199496
(ClinicalTrials.gov)
October 19, 201523/7/2014Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyAn Open-Label Phase I/II Pilot Study to Assess the Safety/Tolerability and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyGastrointestinal Inflammation Associated With CVID;CVID EnteropathyBiological: Stelara (ustekinumab)National Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted18 Years75 YearsAll5Phase 1/Phase 2United States
209EUCTR2015-002356-27-IT
(EUCTR)
16/10/201529/07/2015Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGDA 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 RaffaeleNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
3Phase 2Italy
210NCT02787486
(ClinicalTrials.gov)
October 201526/5/2016Expanded Noninvasive Genomic Medical Assessment: The Enigma StudyA 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 WomenDown Syndrome;Edwards Syndrome;Patau Syndrome;Klinefelter Syndrome;Turner Syndrome;DiGeorge Syndrome;Chromosome Deletion;AneuploidyOther: Blood sampling for Laboratory Developed Test (LDT) analysisProgenity, Inc.NULLCompleted18 Years54 YearsFemale760United States
211NCT04136028
(ClinicalTrials.gov)
September 25, 201521/10/2019IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous DiseaseA Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: KineretFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLCompletedN/A18 YearsAll13Early Phase 1Russian Federation
212NCT02435173
(ClinicalTrials.gov)
August 24, 201524/2/2015Study of Efficacy of CDZ173 in Patients With APDS/PASLIAn 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/PASLICommon Variable Immunodeficiency (CVID), APDS / PASLIDrug: CDZ173;Other: PlaceboNovartis PharmaceuticalsNULLCompleted12 Years75 YearsAll37Phase 2/Phase 3United States;Belarus;Czechia;Germany;Ireland;Italy;Netherlands;Russian Federation;United Kingdom;Czech Republic;France
213NCT01884311
(ClinicalTrials.gov)
August 20, 201514/6/2013Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency DiseasesA Phase III, Multicenter, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Subgam-VF in Primary Immunodeficiency DiseasesPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyperimmunoglobulin M SyndromeBiological: SubgamBio Products LaboratoryNULLCompleted2 Years75 YearsAll38Phase 3United States
214NCT02369978
(ClinicalTrials.gov)
August 201517/2/2015CHICAMOCHA 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 DiseaseDrug: Nifurtimox;Drug: Benznidazole;Drug: PlaceboUniversidad Autónoma de BucaramangaFundación Cardioinfantil Instituto de Cardiología;Instituto Nacional de Salud (Colombia);Instituto Nacional de Parasitologia Dr. Mario Fatala ChabenRecruiting20 Years55 YearsAll500Phase 2/Phase 3Colombia
215NCT02503293
(ClinicalTrials.gov)
July 29, 20151/7/2015A 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 PushA 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 PushPrimary ImmunodeficiencyDevice: Chrono Super PID then Generic Syringe-Gammanorm;Device: Generic Syringe then Chrono Super PID-GammanormOctapharmaNULLCompleted18 YearsN/AAll30Phase 4Australia;Germany;Italy;United Kingdom
216EUCTR2013-005508-33-IT
(EUCTR)
13/07/201521/05/2015Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical 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 GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2Italy;Germany;Austria;Poland;Czech Republic
217EUCTR2014-003746-27-GB
(EUCTR)
16/04/201514/01/2015A 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 syringeA 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/mLOctapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, AustriaNULLNot RecruitingFemale: yes
Male: yes
40Phase 3;Phase 4Australia;Germany;United Kingdom
218EUCTR2014-003746-27-DE
(EUCTR)
15/04/201501/12/2014A 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 syringeA 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/mLOctapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, AustriaNULLNot RecruitingFemale: yes
Male: yes
40Phase 3;Phase 4Australia;Germany;Italy;United Kingdom
219NCT02490956
(ClinicalTrials.gov)
April 201530/3/2015Diagnostic Immunization With Rabies Vaccine in Patients With PIDDiagnostic Immunization With Rabies Vaccine in Patients With Primary Immunodeficiency DisordersPrimary ImmunodeficiencyBiological: Verorab® (PVRV; Purified Vero Cell Vaccine)Chulalongkorn UniversityNULLRecruiting12 Months60 YearsBoth40Phase 4Thailand
220NCT02349906
(ClinicalTrials.gov)
April 201526/1/2015Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant DiseasesClinical 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 DiseasesPrimary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure SyndromesDrug: Treosulfan;Drug: Busilvexmedac GmbHCelerion;Venn Life Sciences;Syneos HealthActive, not recruitingN/A17 YearsAll106Phase 2Czechia;Germany;Italy;Poland;Austria;Czech Republic
221EUCTR2013-003877-87-PL
(EUCTR)
11/03/201504/12/2014Clinical 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.HNULLNot RecruitingFemale: yes
Male: yes
64Phase 3United States;Hungary;Czech Republic;Slovakia;Canada;Poland
222NCT03198195
(ClinicalTrials.gov)
March 10, 201522/6/2017Post-transplant Cyclophosphamide in Wiskott-Aldrich SyndromePost-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeProcedure: cyclophosphamideCapital Research Institute of PediatricsNULLEnrolling by invitation5 Months10 YearsAll5N/ANULL
223NCT02571062
(ClinicalTrials.gov)
March 201524/6/2015Bioequivalence Study - Reference Clinical Fexinidazole Tablet Versus Proposed Market FormulationA 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 ConditionTrypanosomiasis, AfricanDrug: FexinidazoleDrugs for Neglected DiseasesNULLCompleted18 Years45 YearsMale30Phase 1France
224NCT02972281
(ClinicalTrials.gov)
March 201521/11/2016Systematic Search for Primary Immunodeficiency in Adults With InfectionsSystematic Search for Primary Immunodeficiency in Adults With Unexplained Recurrent and/or Severe Infections With Encapsulated BacteriaComplement Deficiency;Antibody Deficiency;Chronic Sinus Infection;Meningitis, Bacterial;Pneumonia, Bacterial;Otitis Media;Streptococcal Infection;Neisseria Infections;Haemophilus Influenza;Pneumococcal InfectionsBiological: Immunological diagnosis testsUniversity Hospital, LilleImagine Institute;Octapharma;CSL Behring;Laboratoire français de Fractionnement et de Biotechnologies;Air Liquide SA;The Binding Site LtdTerminated18 Years65 YearsAll120N/AFrance
225NCT02909244
(ClinicalTrials.gov)
February 20155/9/2016Study of Gut Microbiota in Primary Immune Deficiency, Possibly Associated With Inflammatory Bowel DiseaseStudy 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 DeficienciesBiological: Biological samplingImagine InstituteSaint Antoine University Hospital;Assistance Publique - Hôpitaux de ParisCompletedN/AN/AAll51N/AFrance
226NCT02162420
(ClinicalTrials.gov)
January 201510/6/2014Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A70 YearsAll50N/AUnited States
227NCT02244450
(ClinicalTrials.gov)
December 20142/9/2014Generalized Neonatal Screening of Severe Combined ImmunodeficienciesEvaluation of the Clinical Utility and Cost Effectiveness Ratio of Generalized Neonatal Screening for Severe Combined Immunodeficiencies (SCID) by Quantification of TRECs on Guthrie CardsSevere Combined Immunodeficiency, AtypicalBiological: SCID screeningNantes University HospitalNULLCompletedN/A18 MonthsAll190539N/AFrance
228EUCTR2013-005508-33-PL
(EUCTR)
17/11/201408/08/2014Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical 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 GmbHNULLAuthorised-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): noCzech Republic;Poland;Austria;Germany;Italy
229EUCTR2013-005508-33-CZ
(EUCTR)
12/11/201429/07/2014Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical 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 mbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
230NCT02282904
(ClinicalTrials.gov)
October 23, 20144/11/2014Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated2 Years65 YearsAll7Phase 1/Phase 2United States
231NCT02231879
(ClinicalTrials.gov)
October 14, 20143/9/2014Plerixafor Versus G-CSF in the Treatment of People With WHIM SyndromeA Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome.Myelokathexis;Infections;Neutropenia;Warts;HypogammaglobulinemiaDrug: Plerixafor;Drug: G-CSFNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted10 Years75 YearsAll20Phase 2/Phase 3United States
232EUCTR2013-002290-21-HU
(EUCTR)
13/10/201414/07/2014Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency DiseasesA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
48Phase 3United States;Hungary;United Kingdom
233EUCTR2013-005508-33-DE
(EUCTR)
29/09/201406/06/2014Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical 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 mbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
234NCT02179359
(ClinicalTrials.gov)
September 2, 201427/6/2014Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesMT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent DisordersSickle 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 DisordersDrug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative RegimenMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll25N/AUnited States
235EUCTR2013-005508-33-AT
(EUCTR)
08/08/201408/07/2014Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical 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 mbHNULLNot RecruitingFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
236NCT02177760
(ClinicalTrials.gov)
July 201419/6/2014Sirolimus Prophylaxis for aGVHD in TME SCIDSirolimus 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 TransplantDrug: SirolimusUniversity of California, San FranciscoNULLWithdrawnN/A1 YearBoth0Phase 2United States
237NCT02498782
(ClinicalTrials.gov)
July 201420/8/2014Study to Evaluate Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chagas DiseasePhase 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, ChagasDrug: Fexinidazole;Drug: PlaceboDrugs for Neglected DiseasesNULLRecruiting18 Years50 YearsBoth140Phase 2Bolivia
238EUCTR2014-000274-20-GB
(EUCTR)
14/05/201412/03/2014Long-term follow-up of the WAS gene therapy studyLONG 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
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
10Phase 2United Kingdom
239NCT02184689
(ClinicalTrials.gov)
May 3, 201419/6/2014Efficacy 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 StudyEfficacy 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 StudyHuman African Trypanosomiasis (HAT)Drug: fexinidazoleDrugs for Neglected DiseasesNULLCompleted6 Years14 YearsAll125Phase 2/Phase 3Congo, The Democratic Republic of the
240EUCTR2013-003257-20-GB
(EUCTR)
02/05/201412/03/2014TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATIONEvaluation 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 TRUSTNULLNot 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): yesUnited Kingdom
241EUCTR2013-002290-21-GB
(EUCTR)
30/04/201421/10/2013Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency DiseasesA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
48Phase 3United States;Hungary;Israel;United Kingdom
242NCT02169557
(ClinicalTrials.gov)
April 30, 201415/5/2014Efficacy 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 StudyEfficacy 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 StudyHuman African Trypanosomiasis (HAT)Drug: FexinidazoleDrugs for Neglected DiseasesNULLCompleted15 YearsN/AAll230Phase 2/Phase 3Congo, The Democratic Republic of the
243NCT02180763
(ClinicalTrials.gov)
April 201423/6/2014Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or PumpsA 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: GammanormOctapharmaNULLCompleted18 YearsN/AAll31Phase 4France
244NCT02510404
(ClinicalTrials.gov)
April 201413/7/2015Multivirus-specific Cytotoxic T Lymphocytes (mCTL)Treatment of EBV, CMV, and Adenovirus Infections in Primary Immunodeficiency Disorders With Viral-specific Cytotoxic T-LymphocytesRefractory Viral InfectionsBiological: mCTLsCatherine BollardChildren's National Research InstituteCompletedN/A45 YearsAll1Phase 1United States
245NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase 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 DisordersAcute 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 CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNULLActive, not recruiting1 Month18 YearsAll193Phase 2Italy;United Kingdom;Germany;Spain;United States
246EUCTR2013-003877-87-HU
(EUCTR)
14/03/201407/01/2014Clinical 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.HNULLNot RecruitingFemale: yes
Male: yes
64Phase 3United States;Czech Republic;Hungary;Slovakia;Canada;Poland
247EUCTR2013-003877-87-CZ
(EUCTR)
12/03/201419/12/2013Clinical 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.HNULLNot RecruitingFemale: yes
Male: yes
64Phase 1;Phase 3United States;Hungary;Czech Republic;Slovakia;Canada;Poland
248JPRN-UMIN000013102
2014/03/1001/03/2014Efficacy of Zoledronate for RAS associated ALPS like disease (RALD) RAS associated ALPS like disease (RALD)intravenous drip infusion of ZoledronateDepartment of Pediatrics, Tokyo Medical and Dental UniversityNULLComplete: follow-up completeNot applicableNot applicableMale and Female5Not selectedJapan
249NCT01888484
(ClinicalTrials.gov)
March 201421/6/2013Study of Octanorm Subcutaneous IG in Patients With PIDClinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency DiseasesPrimary Immune Deficiency DisorderBiological: octanorm 16.5%OctapharmaNULLCompleted2 Years75 YearsAll75Phase 3United States;Canada;Czechia;Hungary;Poland;Russian Federation;Slovakia;Czech Republic
250NCT01962415
(ClinicalTrials.gov)
February 4, 201410/10/2013Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTA 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 TransplantationPrimary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory ConditionsDrug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: ThiotepaPaul SzabolcsNULLRecruiting2 Months55 YearsAll100Phase 2United States
251NCT01814800
(ClinicalTrials.gov)
February 20146/3/2013Pharmacokinetics, 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 DisorderBiological: RI-002ADMA Biologics, Inc.NULLCompleted2 Years75 YearsAll59Phase 3United States
252NCT01963143
(ClinicalTrials.gov)
February 201413/9/2013Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency DiseasesA 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 DiseasesPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyper-IgM SyndromeBiological: Gammaplex (5%);Biological: Gammaplex 10Bio Products LaboratoryNULLCompleted2 Years55 YearsAll48Phase 3United States;Hungary;United Kingdom
253NCT01420627
(ClinicalTrials.gov)
January 24, 201418/8/2011EZN-2279 in Patients With ADA-SCIDA 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 ImmunodeficiencyADA-SCID;Adenosine Deaminase Deficiency;Severe Combined ImmunodeficiencyBiological: EZN-2279;Biological: AdagenLeadiant Biosciences, Inc.NULLCompletedN/AN/AAll7Phase 3United States
254EUCTR2013-000620-34-GB
(EUCTR)
10/01/201409/08/2013A 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) syndromesA 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 BiotechnologiesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
55Phase 3France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom
255NCT01917708
(ClinicalTrials.gov)
January 201424/7/2013Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler 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 DiseaseDrug: AbataceptEmory UniversityNULLCompletedN/A21 YearsAll10Phase 1United States
256EUCTR2012-000242-35-DE
(EUCTR)
23/12/201329/04/2013Gene 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
GENETHONNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 1/2France;Germany;United Kingdom;Switzerland
257NCT01985373
(ClinicalTrials.gov)
December 201331/10/2013Pharmacokinetics and Safety of IVIG Nanogam 100 mg/mlPharmacokinetics and Safety of the Intravenous Human Immunoglobulin Product Nanogam 100 mg/mlPrimary ImmunodeficiencyDrug: Intravenous immunoglobulin infusionSanquinNULLCompleted18 YearsN/ABoth23Phase 3Netherlands
258NCT01998633
(ClinicalTrials.gov)
December 201329/10/2013Reduced 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;IPEXBiological: Hematopoietic Stem Cell TransplantMedical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor ProgramCompleted4 Months45 YearsAll47Phase 2United States;Canada
259EUCTR2013-000961-36-DE
(EUCTR)
15/11/201305/08/2013See the full titleTolerability 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 setKedrion SpANULLNot RecruitingFemale: yes
Male: yes
35Phase 3Germany;Italy
260NCT02054832
(ClinicalTrials.gov)
November 201331/1/2014Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked CornstarchA Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked CornstarchGlycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0Dietary Supplement: GlycosadeJohn MitchellNULLCompleted2 Years50 YearsBoth11N/ACanada
261EUCTR2013-000620-34-DE
(EUCTR)
07/10/201305/08/2013A 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) syndromesA 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 BiotechnologiesNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom
262NCT01946906
(ClinicalTrials.gov)
October 201312/9/2013The Rifaximin Study in CVIDEffects 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 TrialCommon Variable Immunodeficiency (CVID)Drug: RifaximinOslo University HospitalNULLCompleted18 Years74 YearsBoth40Phase 4Norway
263EUCTR2013-000620-34-HU
(EUCTR)
19/09/201301/08/2013A 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) syndromesA 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 BiotechnologiesNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;Hungary;Poland;Ukraine;Germany;Italy;United Kingdom
264EUCTR2013-000883-27-NO
(EUCTR)
16/08/201320/06/2013The 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: XifaxanOslo University hospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Norway
265EUCTR2013-000620-34-IT
(EUCTR)
05/08/201330/07/2013A 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) syndromesA 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 BiotechnologiesNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;Hungary;Poland;Ukraine;Germany;United Kingdom;Italy
266NCT01852071
(ClinicalTrials.gov)
August 2, 20137/5/2013Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA GeneAutologous 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-SCIDGenetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERTOrchard TherapeuticsNational Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los AngelesCompleted1 Month17 YearsAll20Phase 1/Phase 2United States
267EUCTR2012-005727-32-NL
(EUCTR)
25/07/201323/05/2013The pharmacokinetics (the behaviour in the body) and safety of the intravenous immunoglobulin product Nanogam 100 mg/mlPharmacokinetics 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 FoundationNULLNot RecruitingFemale: yes
Male: yes
Netherlands
268EUCTR2011-004154-25-IT
(EUCTR)
25/06/201320/02/2013Clinical study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiencyA 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 AGNULLNot RecruitingFemale: yes
Male: yes
20Phase 1;Phase 2Egypt;Lebanon;Germany;Italy
269NCT01852370
(ClinicalTrials.gov)
June 20, 201326/4/2013Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric DonorsBilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric DonorsSevere 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 cellsPaul SzabolcsNULLEnrolling by invitation5 Years45 YearsAll16Phase 1/Phase 2United States
270EUCTR2010-023483-41-FR
(EUCTR)
24/04/201304/01/2011A 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 BIOTECHNOLOGIESNULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Hungary
271EUCTR2012-001725-26-DE
(EUCTR)
12/03/201318/12/2012A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vectorA 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-UniversityNULLNot RecruitingFemale: yes
Male: yes
5Phase 1;Phase 2Germany
272NCT01966367
(ClinicalTrials.gov)
March 201317/10/2013CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationCD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant DiseaseBone 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-thalassemiaBiological: CD34 Stem Cell Selection TherapyDiane GeorgeNULLActive, not recruitingN/A40 YearsAll37Phase 1/Phase 2United States
273NCT01821781
(ClinicalTrials.gov)
March 201319/3/2013Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune 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 SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineNULLRecruitingN/A21 YearsAll20Phase 2United States
274EUCTR2012-000242-35-GB
(EUCTR)
10/01/201319/07/2012Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous DiseaseA 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
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
11Phase 1;Phase 2France;Germany;Switzerland;United Kingdom
275NCT01218438
(ClinicalTrials.gov)
January 20138/10/2010Phase 2/3 Study of IGSC, 20% in PIDDA 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% SolutionBaxalta now part of ShireNULLCompleted2 YearsN/AAll86Phase 2/Phase 3United States;Canada
276JPRN-UMIN000009370
2012/12/0721/11/2012Thalidomide therapy clinical trial for chronic granulomatous disease-associated intractable granulomatous lesion Chronic granulomatous diseaseOral administration of thalidomideNational Center for Child Heath and DevelopmentNULLComplete: follow-up complete3years-oldNot applicableMale and Female5Not selectedJapan
277NCT01380990
(ClinicalTrials.gov)
November 15, 201223/6/2011Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyPhase 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 IndividualsAdenosine 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-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsCompletedN/A15 YearsAll36Phase 1/Phase 2United Kingdom
278NCT01581593
(ClinicalTrials.gov)
November 201216/4/2012Efficacy 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 DeficiencyBiological: Kedrion IVIG 10%Kedrion S.p.A.NULLActive, not recruiting2 Years70 YearsBoth50Phase 3United States;Canada
279NCT01685827
(ClinicalTrials.gov)
October 201212/9/2012Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2Efficacy 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 StudyHuman African Trypanosomiasis (HAT);Sleeping SicknessDrug: Fexinidazole;Drug: Nifurtimox;Drug: EflornithineDrugs for Neglected DiseasesNULLCompleted15 YearsN/AAll394Phase 2/Phase 3Central African Republic;Congo, The Democratic Republic of the;Congo
280NCT01306019
(ClinicalTrials.gov)
September 25, 201226/2/2011Lentiviral 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 ImmunodeficiencyX-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 vectorNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting2 Years40 YearsMale30Phase 1/Phase 2United States
281NCT01652092
(ClinicalTrials.gov)
September 4, 201225/7/2012Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;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 HistiocytosisDrug: 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: MESNAMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A50 YearsAll30N/AUnited States
282EUCTR2012-000792-16-DE
(EUCTR)
03/09/201210/05/2012Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseasesCLINICAL 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 AGNULLNot RecruitingFemale: yes
Male: yes
23Czech Republic;Hungary;Germany
283EUCTR2010-024253-36-GB
(EUCTR)
03/08/201209/05/2012A clinical trial to study the effects of genetically modified patients' CD34+ cellsPhase 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 TrustNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2United Kingdom
284NCT01659606
(ClinicalTrials.gov)
July 20126/8/2012Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaRadiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere DiseaseDyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic AnemiaBiological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetilBoston Children's HospitalDana-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 HospitalRecruitingN/A65 YearsAll40Phase 2United States;Norway;Sweden
285NCT02327351
(ClinicalTrials.gov)
July 201218/12/2014TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PIDPhase 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 DiseasesPrimary Immune Deficiency Disorder;Hematopoietic Stem Cell TransplantationOther: Biological: TCR alfa beta T cell depletionFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting1 Month18 YearsBoth60Phase 2/Phase 3Russian Federation
286EUCTR2012-000481-38-GB
(EUCTR)
28/06/201226/04/2012Assessment of the safety of Immunoglobulin and recombinant human hylaluronidase in the treatment of patients with primary immunodeficiencyTolerability, 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
40Czech Republic;Belgium;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden
287EUCTR2012-000792-16-HU
(EUCTR)
14/06/201220/03/2012Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseasesCLINICAL 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 AGNULLNot RecruitingFemale: yes
Male: yes
23Czech Republic;Hungary;Germany
288EUCTR2012-000792-16-CZ
(EUCTR)
29/05/201227/03/2012Study to evaluate the efficacy, metabolism and safety of human immuneglobulin in patients with primary immunodeficiency diseasesCLINICAL 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 AGNULLNot RecruitingFemale: yes
Male: yes
23Hungary;Czech Republic;Germany
289NCT04350164
(ClinicalTrials.gov)
April 1, 201210/4/2020Romiplostim 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 SyndromeDrug: RomiplostimFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLActive, not recruitingN/A18 YearsAll67Russian Federation
290NCT03315078
(ClinicalTrials.gov)
April 201216/10/2017Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyLentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyX-Linked Combined Immunodeficiency DiseasesBiological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting2 Years40 YearsAll13Phase 1/Phase 2United States
291EUCTR2010-019459-23-BE
(EUCTR)
29/03/201214/02/2012CLINICAL STUDY OF THE EFFECTIVENESS AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Phase 2;Phase 3Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
292NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated 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 SyndromeDrug: 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 transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
293EUCTR2011-001118-32-SE
(EUCTR)
03/02/201206/12/2011Twelve-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 neutropeniaTwelve-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 GmbHNULLNot RecruitingFemale: yes
Male: yes
40Phase 4Germany;Sweden
294NCT01533961
(ClinicalTrials.gov)
February 201213/2/2012Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the SCYX-7158Randomized, 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 VolunteersTrypanosomiasis;Trypanosomiasis, African;Protozoan Infections;Parasitic DiseasesDrug: SCYX-7158;Drug: PlaceboDrugs for Neglected DiseasesNULLCompleted18 Years45 YearsMale136Phase 1France
295NCT03333200
(ClinicalTrials.gov)
January 11, 201224/4/2017Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;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 DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghNULLRecruitingN/AN/AAll1500United States
296NCT03354533
(ClinicalTrials.gov)
January 1, 201221/11/2017Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type IIStudy of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type IILeukocyte Adhesion Deficiency, Type IIDrug: L-fucoseOrpha LabsNULLCompletedN/A18 YearsAll4Phase 1/Phase 2NULL
297NCT01485796
(ClinicalTrials.gov)
December 29, 20112/12/2011Tolerability and Safety of IGI, 10% With rHuPH20 in PIDDTolerability, 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 hyaluronidaseBaxalta now part of ShireNULLCompleted2 YearsN/AAll54Phase 2/Phase 3United States
298NCT01465958
(ClinicalTrials.gov)
November 201124/10/2011Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary ImmunodeficiencyAn Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: GAMUNEX-CGrifols Therapeutics Inc.NULLCompleted2 Years16 YearsAll12Phase 4United States
299NCT01461018
(ClinicalTrials.gov)
October 201124/10/2011Multicenter 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 ImmunodeficiencyPrimary Immune DeficiencyBiological: Immune globulin subcutaneous (Human)CSL BehringNULLCompletedN/A75 YearsBoth22Phase 3Japan
300NCT01406470
(ClinicalTrials.gov)
September 201126/7/2011Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary ImmunodeficiencyAn 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 ImmunodeficiencyImmunologic Deficiency SyndromeDrug: Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™Green Cross CorporationAtlantic Research GroupCompleted2 Years70 YearsBoth45Phase 3United States;Canada
301NCT01483170
(ClinicalTrials.gov)
September 201129/11/2011Multiple Dose Study to Evaluate Security, Tolerance and Pharmacokinetic of Fexinidazole (Drug Candidate for Human African Trypanosomiasis) Administered With a Loading Dose and With FoodDouble-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, AfricanDrug: Tablets Fexinidazole;Drug: PlaceboDrugs for Neglected DiseasesNULLTerminated18 Years45 YearsMale30Phase 1France
302EUCTR2010-019459-23-NL
(EUCTR)
24/08/201127/01/2011CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Phase 2;Phase 3Hungary;Belgium;Austria;Germany;Netherlands;United Kingdom;Sweden
303NCT01410825
(ClinicalTrials.gov)
July 20114/8/2011Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromePilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeBiological: Retrovirus-mediated gene transferDavid WilliamsNULLActive, not recruiting3 Months35 YearsMale5Phase 1/Phase 2United States
304NCT01412385
(ClinicalTrials.gov)
June 20, 20118/8/2011Immune 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 DiseasesPrimary 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 ShireNULLCompleted2 YearsN/AAll55Phase 2/Phase 3Austria;Germany;Hungary;Sweden;United Kingdom
305EUCTR2011-001118-32-DE
(EUCTR)
25/05/201127/04/2011Twelve-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 neutropeniaTwelve-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 GmbHNULLNot RecruitingFemale: yes
Male: yes
40Germany;Sweden
306NCT01313507
(ClinicalTrials.gov)
May 20119/3/2011High 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 DiseaseBiological: NewGamOctapharmaNULLCompleted2 Years75 YearsAll21Phase 3United States
307EUCTR2007-004088-22-DE
(EUCTR)
14/04/201127/07/2010Efficacy and safety of Fibrinogen Concentrate (Human) for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiencyFibrinogen Concentrate (Human) (FCH) - efficacy and safetyEfficacy 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
23Germany;Italy
308NCT01458171
(ClinicalTrials.gov)
April 201112/10/2011Follow-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 ImmunodeficiencyPrimary Immune Deficiency DisorderBiological: Immune globulin subcutaneous (Human)CSL BehringNULLCompletedN/A75 YearsAll23Phase 3Japan
309EUCTR2010-019459-23-SE
(EUCTR)
23/03/201125/01/2011CLINICAL STUDY OF THE EFFECTIVENESS AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
310EUCTR2010-023483-41-HU
(EUCTR)
01/03/201110/11/2010A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune systemA 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 BIOTECHNOLOGIESNULLNot RecruitingFemale: yes
Male: yes
60Serbia;France;Czech Republic;Hungary;Poland;Ukraine;Lithuania
311NCT01289847
(ClinicalTrials.gov)
March 201127/1/2011A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary ImmunodeficiencyA Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and AdolescentsPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Hyper-IgM Syndrome;Wiskott-Aldrich SyndromeBiological: GammaplexBio Products LaboratoryNULLCompleted2 Years16 YearsAll25Phase 4United States;Chile;Israel
312EUCTR2010-019459-23-DE
(EUCTR)
18/01/201128/07/2010CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
313NCT01338675
(ClinicalTrials.gov)
January 20114/3/2011Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)Chronic Granulomatous DiseaseDrug: BusulfanSeoul National University HospitalNULLRecruitingN/AN/ABoth5Phase 1/Phase 2Korea, Republic of
314EUCTR2010-019459-23-GB
(EUCTR)
21/12/201005/10/2010CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
315EUCTR2010-019459-23-HU
(EUCTR)
20/12/201020/10/2010CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUSIMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARYIMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
316NCT01220531
(ClinicalTrials.gov)
November 8, 201022/9/2010Thymus Transplantation Safety-EfficacySafety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Cultured Thymus Tissue;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Mycophenolate mofetilM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbHAvailableN/AN/AAllUnited States
317EUCTR2010-019459-23-AT
(EUCTR)
05/11/201002/08/2010CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
47Hungary;Belgium;Austria;Netherlands;Germany;United Kingdom;Sweden
318NCT01856582
(ClinicalTrials.gov)
October 201015/5/2013CD34+ Stem Cell Infusion to Augment Graft FunctionPost Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure SyndromesWaning Donor Chimerism;Waning Immune Function;Primary Immunodeficiency Disease(s);Bone Marrow FailureBiological: CD34+Children's Hospital Medical Center, CincinnatiHoxworth Blood CenterTerminatedN/A35 YearsAll23Phase 2United States
319NCT01354587
(ClinicalTrials.gov)
October 201018/3/2011Evaluation 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 DisordersDrug: HizentraUniversity of South FloridaNULLRecruiting1 Year75 YearsBoth50N/AUnited States
320EUCTR2010-019249-25-HU
(EUCTR)
22/09/201006/08/2010Eine 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 AGNULLNot RecruitingFemale: yes
Male: yes
30Hungary;Germany
321NCT01199705
(ClinicalTrials.gov)
September 20108/9/2010Study 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 ImmunodeficiencyPrimary Immune DeficiencyBiological: Immune Globulin Subcutaneous (Human) (SCIG)CSL BehringNULLCompletedN/A75 YearsAll25Phase 3Japan
322NCT01319851
(ClinicalTrials.gov)
September 201015/9/2010Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;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 AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaTerminatedN/A21 YearsAll3N/AUnited States
323EUCTR2010-019249-25-DE
(EUCTR)
12/08/201014/05/2010An 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 AGNULLNot RecruitingFemale: yes
Male: yes
30Hungary;Germany
324NCT01182675
(ClinicalTrials.gov)
August 20109/8/2010Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & FilgrastimHematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & FilgrastimSevere Combined ImmunodeficiencyDrug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and AlemtuzumabUniversity of California, San FranciscoNULLTerminatedN/A3 YearsAll7Phase 2United States
325NCT01175213
(ClinicalTrials.gov)
July 28, 20103/8/2010Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PIDLong-Term Tolerability and Safety of Immune Globulin Subcutaneous (IGSC) Solution Administered Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency DiseasesPrimary 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 ShireNULLCompleted2 YearsN/AAll66Phase 3United States
326NCT01147042
(ClinicalTrials.gov)
May 18, 201017/6/2010Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous DiseaseAssessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous DiseaseIFN-Gamma Therapy;CGD Gene Mutation;CGD Response to IFNg;CGD - Chronic Granulomatous Disease;Immunodeficiency DiseaseDrug: IFN-gammaNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminatedN/AN/AAll2Phase 4United States
327NCT01129544
(ClinicalTrials.gov)
April 201021/5/2010Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral VectorMulti-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene TransferSevere Combined ImmunodeficiencyBiological: Gene transferDavid WilliamsBoston Children's Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los AngelesActive, not recruitingN/AN/AMale8Phase 1/Phase 2United States
328NCT03534479
(ClinicalTrials.gov)
April 201010/5/2018Human IgGs and Endothelial Function in Vivo in HumansEffects of Intravenous Human Polyclonal Immunoglobulins G Infusion on Endothelial Function and Insulin Sensitivity in HumansCommon Variable ImmunodeficiencyDrug: Polyclonal IgGFederico II UniversityNULLCompleted18 Years70 YearsAll24N/AItaly
329EUCTR2009-017346-32-IT
(EUCTR)
15/03/201026/04/2010HAEMATOPOIETIC STEM CELL GENE THERAPYA 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) LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
8Phase 1;Phase 2Italy
330EUCTR2009-011434-10-DE
(EUCTR)
02/03/201027/10/2009CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESCLINICAL 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 AGNULLNot RecruitingFemale: yes
Male: yes
50Germany
331NCT01131858
(ClinicalTrials.gov)
March 201026/5/2010Study of Vitamin D3 Substitution to Patients With Primary ImmunodeficiencyA Placebo Controlled Double Blinded Study of Vitamin D3 Substitution to Patients With Primary ImmunodeficiencyPrimary Immune Deficiency DisorderDrug: Vigantol;Drug: PlaceboKarolinska University HospitalNULLCompleted18 Years75 YearsBoth140Phase 1/Phase 2Sweden
332NCT01075438
(ClinicalTrials.gov)
March 201024/2/2010Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia PatientsDescriptive 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 PatientsAtaxia TelangiectasiaBiological: Prevenar® (7-valent pneumococcal conjugate vaccine);Biological: Pneumovax® (pneumococcal polysaccharide vaccine)Sheba Medical CenterNULLNot yet recruiting2 Years25 YearsBoth20N/AIsrael
333EUCTR2007-002611-27-GB
(EUCTR)
19/02/201015/03/2010Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NAClinical 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 AGNULLNot 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): noFrance;Germany;United Kingdom
334EUCTR2007-002611-27-FR
(EUCTR)
17/02/201026/11/2009Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NAClinical 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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45United Kingdom;France
335EUCTR2007-000684-16-GB
(EUCTR)
21/01/201017/09/2009Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1Gene 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 HealthNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2United Kingdom
336EUCTR2007-004308-11-GB
(EUCTR)
11/01/201006/07/2009Gene therapy for WASPHASE 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
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
Phase 1;Phase 2United Kingdom
337NCT01012323
(ClinicalTrials.gov)
January 201011/11/2009A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency DiseasesClinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency DiseasesPrimary Immunodeficiency DiseasesBiological: NewGamOctapharmaPremier Research Group plcCompleted2 Years75 YearsAll51Phase 3United States
338NCT01052623
(ClinicalTrials.gov)
January 201019/1/2010Status 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 TelangiectasiaAtaxia Telangiectasia;Growth FailureDrug: Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerateJohann Wolfgang Goethe University HospitalsNULLRecruiting3 Years18 YearsBoth24Phase 4Germany
339EUCTR2009-015739-34-DE
(EUCTR)
09/12/200914/09/2009Status 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/MainNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Germany
340EUCTR2008-004518-28-GB
(EUCTR)
08/12/200909/11/2009Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPODInvestigation 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 CentreNULLNot RecruitingFemale: yes
Male: yes
20United Kingdom;Netherlands
341NCT01001598
(ClinicalTrials.gov)
November 200922/10/2009Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaPhase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaFanconi Anemia;Dyskeratosis CongenitaDrug: danazolBoston Children's HospitalNULLTerminated3 YearsN/AAll5Phase 1/Phase 2United States
342NCT00982904
(ClinicalTrials.gov)
September 200922/9/2009Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the FexinidazoleRandomized, 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 VolunteersHuman African TrypanosomiasisDrug: Fexinidazole/PlaceboDrugs for Neglected DiseasesSanofiCompleted18 Years45 YearsMale108Phase 1France
343EUCTR2009-012036-32-FR
(EUCTR)
30/07/200928/05/2009SAFETY 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 RATESAFETY 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 BIOTECHNOLOGIESNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
France
344NCT00909363
(ClinicalTrials.gov)
June 200927/5/2009Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) PatientsEffects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.Wiskott-Aldrich Syndrome;Thrombocytopenia;BleedingDrug: Promacta;Diagnostic Test: blood drawing in patients with WAS;Diagnostic Test: blood drawing in healthy controlsWeill Medical College of Cornell UniversityNovartis PharmaceuticalsTerminated3 Months80 YearsMale24Phase 2United States
345NCT01489618
(ClinicalTrials.gov)
June 20098/12/2011Prime 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 ImmunodeficiencyRandomised, 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 ImmunodeficiencyCommon Variable ImmunodeficiencyBiological: PPS;Biological: PnCJ PPSInstitut National de la Santé Et de la Recherche Médicale, FranceNULLTerminated18 Years65 YearsBoth45Phase 2France
346NCT00906880
(ClinicalTrials.gov)
April 200919/5/2009Clinical 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 PhaseClinical 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 StageHuman African TrypanosomiasisDrug: Nifurtimox-Eflronithine Combination Treatment (NECT)Drugs for Neglected DiseasesMinistry of Public Health, Democratic Republic of the Congo;Swiss Tropical & Public Health InstituteCompletedN/AN/ABoth630Phase 4Congo
347NCT00799071
(ClinicalTrials.gov)
February 200926/11/2008Pharmacokinetics 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 DiseaseDrug: posaconazole (PSZ)Radboud UniversityNULLCompleted2 Years16 YearsAll12Phase 2Netherlands;United Kingdom
348EUCTR2007-003235-23-FR
(EUCTR)
09/01/200923/10/2008Essai 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 DICVEssai 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
InsermNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2France
349NCT00811174
(ClinicalTrials.gov)
January 200917/12/2008Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency DiseasesClinical Study to Evaluate the Efficacy, Safety and Kinetics of Octagam 10% for Replacement Therapy in Primary Immunodeficiency DiseasesImmunologic Deficiency SyndromesDrug: Octagam 10%OctapharmaNULLTerminated2 Years75 YearsAll5Phase 3Austria
350NCT01289171
(ClinicalTrials.gov)
January 200912/1/2011Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric PatientsAcquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients: Prospective Treatment Trial With Topical Glycolic Acid and HPV Genotype CharacterizationFlat Warts (Diagnosis);HIV InfectionsDrug: Glycolic acidUniversity of PennsylvaniaNeoStrata Company, Inc.;Penn Center for AIDS Research (CFAR)Completed7 YearsN/ABoth38N/ABotswana
351NCT00814320
(ClinicalTrials.gov)
December 18, 200823/12/2008Gammagard Liquid and rHuPH20 in PIDEfficacy, 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 DiseasesPrimary Immunodeficiency Diseases (PID)Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)Baxalta now part of ShireNULLCompleted2 YearsN/AAll89Phase 3United States;Canada
352EUCTR2008-000830-30-SE
(EUCTR)
17/12/200803/11/2008A 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 AGNULLNot RecruitingFemale: yes
Male: yes
36United Kingdom;Germany;France;Spain;Sweden
353EUCTR2008-000830-30-GB
(EUCTR)
10/12/200820/01/2009Extension 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 AGNULLNot 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): noFrance;Spain;Poland;Romania;Germany;Switzerland;United Kingdom;Sweden
354EUCTR2008-000830-30-FR
(EUCTR)
24/11/200821/10/2008A 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 AGNULLNot RecruitingFemale: yes
Male: yes
36Phase 3France;Spain;Germany;United Kingdom;Sweden
355EUCTR2008-004518-28-NL
(EUCTR)
19/11/200824/11/2008Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPODInvestigation 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 solutionRadboud University Nijmegen Medical CentreNULLNot RecruitingFemale: yes
Male: yes
United Kingdom;Netherlands
356EUCTR2008-000830-30-ES
(EUCTR)
12/11/200801/10/2008A 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 AGNULLNot RecruitingFemale: yes
Male: yes
36United Kingdom;Germany;France;Spain;Sweden
357NCT00794508
(ClinicalTrials.gov)
November 200819/11/2008MND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDMND-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 BusulfanSevere Combined ImmunodeficiencyBiological: ADA gene transferDonald B. Kohn, M.D.FDA Office of Orphan Products Development;National Institutes of Health (NIH)Completed1 Month18 YearsAll10Phase 2United States
358EUCTR2007-002611-27-DE
(EUCTR)
27/10/200814/12/2007Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NAClinical 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 AGNULLNot RecruitingFemale: yes
Male: yes
45France;Germany;United Kingdom
359EUCTR2006-006745-13-PL
(EUCTR)
16/10/200811/08/2008A 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 BehringNULLNot RecruitingFemale: yes
Male: yes
43Phase 3France;Spain;Poland;Germany;Italy;United Kingdom;Sweden
360NCT00774358
(ClinicalTrials.gov)
October 200816/10/2008Interleukin-2 Treatment for Wiskott-Aldrich SyndromeReinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 TherapyWiskott-Aldrich Syndrome (WAS);X-linked ThrombocytopeniaDrug: Interleukin-2Soma JyonouchiTexas Children's HospitalCompleted24 MonthsN/AAll9Phase 1United States
361NCT00751621
(ClinicalTrials.gov)
August 200811/9/2008Study 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: IgPro20CSL BehringNULLCompleted2 Years65 YearsAll40Phase 3France;Germany;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom
362EUCTR2008-000830-30-DE
(EUCTR)
10/07/200809/06/2008A 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 AGNULLNot RecruitingFemale: yes
Male: yes
36United Kingdom;Germany;France;Spain;Sweden
363EUCTR2008-003368-21-IT
(EUCTR)
04/06/200806/02/2009Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndromePilot 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 GAROFOLONULLNot RecruitingFemale: no
Male: yes
Italy
364NCT00719680
(ClinicalTrials.gov)
June 200821/7/2008Extension 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 DeficiencyBiological: IgPro20CSL BehringNULLCompleted2 Years75 YearsAll21Phase 3United States
365NCT00634569
(ClinicalTrials.gov)
May 20085/3/2008Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric SubjectsEvaluation 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 DiseaseBiological: Flebogamma 5% DIFInstituto Grifols, S.A.NULLCompleted2 Years16 YearsAll24Phase 4United States
366NCT00680446
(ClinicalTrials.gov)
April 200816/5/2008Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary ImmunodeficiencyAn Open-Label Study of Ig NextGen 16% Administered by Subcutaneous Infusion in Patients With Primary Immunodeficiency (PID).Primary Immune DeficiencyDrug: Immunoglobulin G (Ig NextGen 16%)CSL LimitedNULLCompleted3 YearsN/ABoth41Phase 3Australia;New Zealand
367EUCTR2006-006522-25-GR
(EUCTR)
08/01/200804/06/2007A 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 AGNULLNot RecruitingFemale: yes
Male: yes
28Phase 4Greece;Spain;Belgium;Germany;Italy;United Kingdom
368NCT00605657
(ClinicalTrials.gov)
January 200815/1/2008Valproic 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;LymphadenopathyDrug: Valproic Acid;Procedure: CT Scan;Procedure: Blood SampleKoneti RaoNULLCompleted2 Years70 YearsAll6Phase 1/Phase 2United States
369NCT00546871
(ClinicalTrials.gov)
October 3, 200718/10/2007Comparison of Intravenous and Subcutaneous Administration of IGIV, 10% in Primary Immunodeficiency (PID) SubjectsTolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human) 10% (IGIV, 10%) Administered Intravenously or Subcutaneously in Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Drug: Immune Globulin Intravenous (Human), 10%Baxalta now part of ShireNULLCompleted24 MonthsN/AAll49Phase 2/Phase 3United States
370NCT00579137
(ClinicalTrials.gov)
October 200719/12/2007Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency DisordersCD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency DisordersSevere Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick SyndromeBiological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusionBaylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's HospitalTerminatedN/AN/AAll3Phase 1/Phase 2United States
371EUCTR2006-006023-39-IT
(EUCTR)
24/09/200712/06/2007Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - NDPharmacokinetics 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 PCSL Behring GmbHNULLNot RecruitingFemale: yes
Male: yes
Italy
372EUCTR2006-006745-13-SE
(EUCTR)
19/09/200723/07/2007A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyA 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 AGNULLNot RecruitingFemale: yes
Male: yes
51United Kingdom;Germany;France;Spain;Italy;Poland;Sweden
373EUCTR2006-006522-25-GB
(EUCTR)
10/09/200728/03/2007A 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 AGNULLNot 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): yesGreece;Spain;Belgium;Germany;Italy;United Kingdom
374NCT00527878
(ClinicalTrials.gov)
September 20078/9/2007Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) SyndromeA 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 ManifestationsJOB's Syndrome;Hyper-IgE Recurrent Infection Syndrome;Immune DeficiencyDrug: Ranitidine;Drug: PlaceboNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated2 YearsN/AAll16Phase 2United States
375NCT00542997
(ClinicalTrials.gov)
September 200711/10/2007Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement TherapyA Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary ImmunodeficiencyCommon Variable Immunodeficiency;X-linked Agammaglobulinemia;Autosomal Recessive AgammaglobulinemiaBiological: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)CSL BehringNULLCompleted2 Years65 YearsAll51Phase 3France;Germany;Italy;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom
376EUCTR2006-006745-13-IT
(EUCTR)
30/08/200701/08/2007A Multicenter Study of the Efficacy, Tolerability, Safety and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency - NDA 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)NULLNot RecruitingFemale: yes
Male: yes
36United Kingdom;Germany;France;Spain;Italy;Poland;Sweden
377EUCTR2006-006745-13-ES
(EUCTR)
27/08/200711/06/2007Estudio 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 ImmunodeficiencyEstudio 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 AGNULLNot RecruitingFemale: yes
Male: yes
36Phase 3France;Poland;Spain;Germany;Italy;United Kingdom;Sweden
378EUCTR2006-006522-25-IT
(EUCTR)
13/08/200731/07/2007A Multicenter study on the efficacy and safety of Vivaglobin in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID) - NDA 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 AGNULLNot RecruitingFemale: yes
Male: yes
28United Kingdom;Germany;Belgium;Spain;Italy;Greece
379NCT00455312
(ClinicalTrials.gov)
August 200730/3/2007Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: MethylprednisoloneMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll36Phase 2/Phase 3United States
380EUCTR2006-006745-13-GB
(EUCTR)
17/07/200715/05/2007A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyA 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 AGNULLNot 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): noFrance;Poland;Spain;Germany;Italy;United Kingdom;Sweden
381EUCTR2006-006745-13-DE
(EUCTR)
11/07/200721/03/2007A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyA 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 AGNULLNot RecruitingFemale: yes
Male: yes
51United Kingdom;Germany;France;Spain;Italy;Poland;Sweden
382EUCTR2006-006745-13-FR
(EUCTR)
10/07/200704/06/2007A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyA 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 AGNULLNot RecruitingFemale: yes
Male: yes
36Phase 3France;Poland;Spain;Germany;Italy;United Kingdom;Sweden
383EUCTR2006-006522-25-ES
(EUCTR)
01/07/200703/03/2010Estudio 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 AGNULLNot RecruitingFemale: yes
Male: yes
28Phase 4Greece;Belgium;Spain;Germany;Italy;United Kingdom
384EUCTR2006-006522-25-BE
(EUCTR)
27/06/200717/04/2007A 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 AGNULLNot RecruitingFemale: yes
Male: yes
28United Kingdom;Germany;Belgium;Spain;Italy;Greece
385NCT00490100
(ClinicalTrials.gov)
June 200721/6/2007Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1Treatment 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 ResistenceDrug: IncrelexNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated2 Years20 YearsAll6Phase 1/Phase 2United States
386EUCTR2007-001410-17-FR
(EUCTR)
31/05/200713/04/2007LONG-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 PATIENTSLONG-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 SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
France
387NCT00640003
(ClinicalTrials.gov)
April 200714/3/2008Baclofen Treatment of Ataxia TelangiectasiaAtaxia TelangiectasiaDrug: Baclofen;Drug: PlaceboJohns Hopkins UniversityNULLCompleted12 YearsN/AAll10Early Phase 1United States
388NCT00391131
(ClinicalTrials.gov)
April 200720/10/2006Subcutaneous Ig NextGen 16% in PID PatientsA 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 LimitedNULLCompleted3 YearsN/ABoth35Phase 3Australia;New Zealand
389NCT00520494
(ClinicalTrials.gov)
March 200723/8/2007Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary ImmunodeficiencyA Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)Common Variable Immunodeficiency;AgammaglobulinemiaDrug: VivaglobinCSL BehringNULLCompleted1 Year70 YearsAll18Phase 4Canada;Germany;Italy;Spain;Belgium
390EUCTR2006-006522-25-DE
(EUCTR)
01/02/200707/12/2006A 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 AGNULLNot RecruitingFemale: yes
Male: yes
28United Kingdom;Germany;Belgium;Spain;Italy;Greece
391NCT02512679
(ClinicalTrials.gov)
February 200721/5/2015Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsProtocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological DiseasesStem 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 DiseasesDrug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4Children's Hospital Los AngelesLucile Packard Children's HospitalTerminated3 MonthsN/AAll20Phase 2NULL
392NCT00885833
(ClinicalTrials.gov)
February 200721/4/2009Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeDrug: Fludarabine, Busulfan, ThymoglobulinThe Korean Society of Pediatric Hematology OncologyNULLCompleted1 Year25 YearsBoth5Phase 1/Phase 2Korea, Republic of
393NCT02127892
(ClinicalTrials.gov)
January 2, 200719/10/2012SCID Bu/Flu/ATG Study With T Cell DepletionPhase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling DonorSevere Combined ImmunodeficiencyBiological: unrelated BM with T cell depletion;Biological: unrelated cord blood;Biological: haplo BM with T cell depletion;Device: unrelated PBSC with T cell depletionNeena Kapoor, M.D.NULLTerminatedN/A21 YearsAll9Phase 1/Phase 2United States
394NCT00778882
(ClinicalTrials.gov)
January 200721/10/2008Gene Therapy for Chronic Granulomatous Disease in KoreaAn 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 PatientsChronic Granulomatous DiseaseDrug: VM106Helixmith Co., Ltd.NULLActive, not recruitingN/AN/AMale2Phase 1/Phase 2Korea, Republic of
395EUCTR2005-003201-81-DE
(EUCTR)
20/12/200619/01/2007GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHEGACHE: 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 HeidelbergNULLNot RecruitingFemale: yes
Male: yes
450Phase 3Germany
396NCT00782106
(ClinicalTrials.gov)
December 4, 200629/10/2008Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV SubcutaneouslyPhase 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 intravenousBaxalta now part of ShireNULLCompleted16 YearsN/AAll11Phase 1/Phase 2United States
397NCT00392951
(ClinicalTrials.gov)
December 200624/10/2006Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNULLCompleted1 Year30 YearsAll30Phase 1/Phase 2United States
398NCT00389324
(ClinicalTrials.gov)
November 200617/10/2006A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary ImmunodeficiencyAn 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 ImmunodeficiencyImmunologic Deficiency SyndromeBiological: Immune Globulin Intravenous (Human)Grifols Therapeutics Inc.NULLCompleted13 Years75 YearsAll35Phase 2United States;Canada
399NCT00419341
(ClinicalTrials.gov)
November 200622/12/2006Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement TherapyA 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 DeficiencyBiological: Human Normal Immunoglobulin for Subcutaneous AdministrationCSL BehringNULLCompleted2 Years75 YearsAll49Phase 3United States
400NCT00395538
(ClinicalTrials.gov)
October 30, 20062/11/2006Effects of PTH Replacement on Bone in HypoparathyroidismEffects of PTH Replacement on Bone in HypoparathyroidismHypoparathyroidism;DiGeorge SyndromeDrug: PTH 1-34National Institute of Dental and Craniofacial Research (NIDCR)NULLTerminated18 Years70 YearsAll46Phase 3United States;Austria;Italy
401NCT00394316
(ClinicalTrials.gov)
October 30, 200631/10/2006Gene Therapy for Chronic Granulomatous DiseaseAutologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem CellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated3 Years55 YearsMale3Early Phase 1United States
402NCT00656409
(ClinicalTrials.gov)
June 20067/4/2008Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)Ataxia Telangiectasia (AT)Drug: Conjugated pneumococcal vaccine (Prevenar)Institute of Child HealthGreat Ormond Street Hospital for Children NHS Foundation TrustCompleted2 YearsN/ABoth30Phase 3United Kingdom
403NCT00358657
(ClinicalTrials.gov)
May 24, 200628/7/2006Fludarabine 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 DisordersHLA-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 CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: 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 IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
404NCT00325078
(ClinicalTrials.gov)
May 200611/5/2006Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous DiseaseTumor 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 InfluencesChronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD)Drug: InfliximabNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated10 YearsN/AAll40Phase 1/Phase 2United States
405NCT00278954
(ClinicalTrials.gov)
January 200618/1/2006Efficacy, 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 DiseasesPrimary Immunodeficiency;Common Variable Hypogammaglobulinemia;X-linked Hypogammaglobulinemia;Hypogammaglobulinemia;Immunodeficiency With Hyper-IgM;Wiskott-Aldrich SyndromeBiological: Gammaplex (Intravenous immunoglobulin)Bio Products LaboratoryNULLCompleted3 YearsN/AAll50Phase 3United States
406NCT00579527
(ClinicalTrials.gov)
December 19, 200520/12/2007Phase I/II Thymus Transplantation With Immunosuppression #950Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge SyndromeBiological: 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 mofetilM. Louise MarkertNational 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)CompletedN/AN/AAll14Phase 1/Phase 2United States
407NCT00263237
(ClinicalTrials.gov)
December 2, 20057/12/2005STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable ImmunodeficiencyA Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyCommon Variable ImmunodeficiencyDrug: STA-5326National Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted18 Years75 YearsAll10Phase 1United States
408NCT00260702
(ClinicalTrials.gov)
November 24, 20051/12/2005Omalizumab to Treat Hyper-IgE (Job's) SyndromePilot Study of Omalizumab (Xolair) in Hyper IgE (Job's) SyndromeHyper-IgE Syndrome;Job's SyndromeDrug: Omalizumab (Xolair)National Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted6 Years76 YearsAll1Phase 1United States
409EUCTR2005-004122-70-GB
(EUCTR)
15/11/200512/09/2005Immunogenicity 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 telangiectasiaImmunogenicity 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: PrevenarGreat Ormond Street HospitalNULLNot 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): yesUnited Kingdom
410NCT00322556
(ClinicalTrials.gov)
November 20055/5/2006Safety 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 ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNULLCompleted4 Years71 YearsAll55Phase 3United States
411NCT00119431
(ClinicalTrials.gov)
September 20054/7/2005Kinetics, Efficacy and Safety of C1-Esteraseremmer-NPharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) AngioedemaHereditary Angioedema Type I;Angioneurotic EdemaDrug: C1 inhibitor concentrateSanquinNULLCompleted18 YearsN/ABoth12Phase 2Netherlands
412EUCTR2004-004465-15-HU
(EUCTR)
22/07/200511/05/2005A 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 AGNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Germany
413NCT00160355
(ClinicalTrials.gov)
May 20058/9/2005Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeHaploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot StudyWiskott-Aldrich SyndromeProcedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, ThiotepaSt. Jude Children's Research HospitalNULLCompletedN/A18 YearsMale4Phase 1United States
414EUCTR2004-004465-15-DE
(EUCTR)
05/04/200504/02/2005A 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 AGNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Germany
415NCT00295971
(ClinicalTrials.gov)
April 200523/2/2006Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency DiseaseStem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency DiseasesCongenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: 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 irradiationUniversity of California, San FranciscoNational Cancer Institute (NCI)Completed1 Year17 YearsBoth21Phase 1United States
416NCT00566488
(ClinicalTrials.gov)
January 200530/11/2007Parathyroid and Thymus Transplantation in DiGeorge #931Parathyroid and Thymus Transplantation in DiGeorge Syndrome, #931DiGeorge Syndrome;Hypoparathyroidism;Complete DiGeorge SyndromeBiological: Thymus/Parathyroid TransplantationM. Louise MarkertFood 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 GmbHCompletedN/A24 MonthsAll25Phase 1United States
417NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation 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)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: 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 transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A21 YearsAll35Phase 2United States
418NCT00123916
(ClinicalTrials.gov)
November 200421/7/2005BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' DiseaseBenznidazole Evaluation for Interrupting Trypanosomiasis - The BENEFIT TrialChagas Disease;Trypanosomiasis;Heart DiseaseDrug: Benznidazole;Drug: PlaceboPopulation Health Research InstituteCanadian Institutes of Health Research (CIHR);World Health Organization;Instituto Dante Pazzanese de Cardiologia;University of Sao PauloCompleted18 Years75 YearsAll2854Phase 3Argentina;Bolivia;Brazil;Colombia;El Salvador;Canada
419NCT00576836
(ClinicalTrials.gov)
September 2, 200417/12/2007Thymus Transplantation Dose in DiGeorge #932Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Cultured Thymus Tissue Implantation (CTTI);Other: Cultured Thymus Tissue Implantation with Parathyroid TransplantationM. Louise MarkertNational 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 GmbHCompletedN/AN/AAll7Phase 2United States
420NCT00146627
(ClinicalTrials.gov)
September 20046/9/2005Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African TrypanosomiasisClinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic PhaseTrypanosomiasis, AfricanDrug: Eflornithine;Drug: NifurtimoxDrugs for Neglected DiseasesMedecins 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 OrganizationCompleted15 Years70 YearsBoth280Phase 3Congo, The Democratic Republic of the;Congo;The Democratic Republic of the Congo;Uganda
421NCT00168025
(ClinicalTrials.gov)
September 200412/9/2005Efficacy 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 ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNULLCompleted3 Years70 YearsBoth89Phase 3NULL
422NCT00168012
(ClinicalTrials.gov)
September 200412/9/2005Efficacy 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 ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNULLCompleted3 Years70 YearsBoth42Phase 3NULL
423NCT00228852
(ClinicalTrials.gov)
April 200427/9/2005IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyPhase 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 ImmunodeficiencyT-Cell Immune Deficiency Diseases;Severe Combined ImmunodeficiencyDrug: Busulfan, Fludarabine and ATGEmory UniversityNULLCompletedN/AN/ABothPhase 1/Phase 2United States
424NCT00578643
(ClinicalTrials.gov)
March 200419/12/2007Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseHLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell InfusionBaylor College of MedicineNULLCompletedN/AN/AAll15Phase 2United States
425NCT00152100
(ClinicalTrials.gov)
February 20047/9/2005Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency SyndromeTransplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency SyndromeSevere Combined ImmunodeficiencyProcedure: Stem cell transplant;Drug: Filgrastim, Alemtuzumab;Device: Miltenyi CliniMACSSt. Jude Children's Research HospitalNULLCompletedN/A2 YearsBoth4Phase 1United States
426NCT00564759
(ClinicalTrials.gov)
January 200426/11/2007Gene Therapy for Chronic Granulomatous DiseasePhase I/II Gene Therapy Study for X-Linked Chronic Granulomatous DiseaseGranulomatous Disease, ChronicDrug: retroviral SF71-gp91phox transduced CD34+ cellsJohann Wolfgang Goethe University HospitalsGerman Federal Ministry of Education and ResearchActive, not recruiting18 YearsN/AMale2Phase 1/Phase 2Germany
427NCT01279720
(ClinicalTrials.gov)
October 200318/1/2011Gene Therapy ADA DeficiencyPhase I Gene Therapy Protocol for Adenosine Deaminase DeficiencyAdenosine Deaminase DeficiencyBiological: Intravenous infusion of transduced cellsGreat Ormond Street Hospital for Children NHS Foundation TrustNULLCompletedN/A18 YearsBoth8Phase 1/Phase 2United Kingdom
428NCT00065390
(ClinicalTrials.gov)
July 200321/7/2003Pyrimethamine to Treat Autoimmune Lymphoproliferative SyndromePilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphatic Disease;Lymphoproliferative DisorderDrug: PyrimethamineNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth8Phase 1United States
429NCT00803933
(ClinicalTrials.gov)
February 20034/12/2008Trial of DB289 for the Treatment of Stage I African TrypanosomiasisPhase II b Trial of DB289 for the Treatment of Stage I African TrypanosomiasisAfrican TrypanosomiasisDrug: DB289;Drug: PentamidineImmtech Pharmaceuticals, IncBill and Melinda Gates FoundationCompleted15 Years50 YearsBoth111Phase 2Congo
430NCT00489658
(ClinicalTrials.gov)
October 200220/6/2007Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, UgandaEfficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, UgandaTrypanosomiasis, AfricanDrug: Eflornithine plus Nifurtimox combination therapyEpicentreMedecins Sans FrontieresTerminatedN/AN/ABoth31Phase 2/Phase 3Uganda
431NCT00187057
(ClinicalTrials.gov)
September 200212/9/2005Study for Treatment of Cancer in Children With Ataxia-telangiectasiaPilot Study I for Treatment of Cancer in Children With Ataxia-TelangiectasiaAtaxia-TelangiectasiaDrug: vinblastine, vincristine, prednisone, daunorubicin;Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase;Drug: etoposide, cytarabine, mercaptopurine;Drug: dexamethasone, procarbazine;Procedure: chemotherapy, intrathecal chemotherapy, steroid therapySt. Jude Children's Research HospitalChildren's Hospital of Philadelphia;National Cancer Institute (NCI)CompletedN/A10 YearsBoth6N/AUnited States
432NCT00220766
(ClinicalTrials.gov)
August 200213/9/2005Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency PatientsIGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient PatientsImmunologic Deficiency Syndrome;Agammaglobulinemia;Severe Combined Immunodeficiency;Wiskott-Aldrich Syndrome;Common Variable ImmunodeficiencyDrug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified;Drug: Dextrose, 5% in WaterGrifols Therapeutics Inc.NULLCompleted18 Years75 YearsBoth100Phase 3United States;Canada
433NCT00579709
(ClinicalTrials.gov)
July 200220/12/2007Thymus Transplantation With ImmunosuppressionThymus Transplantation With Immunosuppression, #884DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Thymus Tissue for TransplantationM. Louise MarkertNational 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 GmbHCompletedN/AN/AAll15Phase 1United States
434NCT00157079
(ClinicalTrials.gov)
June 25, 20028/9/2005Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency DisordersA Clinical Investigation to Assess the Safety and Efficacy of Immune Globulin Intravenous (Human), 10% in Subjects With Primary Immunodeficiency DisordersPrimary Immunodeficiency Diseases (PID);Immune Thrombocytopenic Purpura (ITP);Kawasaki SyndromeBiological: Immune Globulin Intravenous (Human), 10%Baxalta now part of ShireNULLCompleted24 MonthsN/AAll61Phase 3United States
435NCT00161993
(ClinicalTrials.gov)
June 13, 20028/9/2005Safety, 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 AgammaglobulinemiaPrimary Immunodeficiency Diseases (PID);Agammaglobulinemia;HypogammaglobulinemiaDrug: Immune Globulin Intravenous (Human), 10% TVR (Triple Virally Reduced) Solution;Drug: Gammagard S/D (Solvent/Detergent)Baxalta now part of ShireNULLCompleted18 YearsN/AAll24Phase 2Finland;Sweden
436NCT00176852
(ClinicalTrials.gov)
June 200212/9/2005Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramCompletedN/A50 YearsAll22Phase 2/Phase 3United States
437NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
438NCT00661401
(ClinicalTrials.gov)
January 200214/4/2008Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous ImmunoglobulinSerum IgG Antibody to Streptococcus Pneumoniae, Haemophilus Influenzae Type b and Tetanus Toxoid in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin InfusionsCommon Variable Immunodeficiency;AgammaglobulinemiaBiological: gammaglobulinFederal University of São PauloCSL BehringCompleted2 Years75 YearsBoth5N/ABrazil
439NCT00028236
(ClinicalTrials.gov)
December 10, 200117/12/2001Stem 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 ImmunodeficiencyDrug: Gene-Transduced Autologous CD34+ Stem CellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted18 Months20 YearsAll3Phase 1United States
440NCT00024934
(ClinicalTrials.gov)
October 20019/10/2001B-Lymphocyte Stimulator (BLyS) To Treat Selective IgA DeficiencyA Phase I Dose Escalation Study of B-Lymphocyte Stimulator (BLyS) Administered Subcutaneously in Patients With Selective IgA DeficiencyIgA DeficiencyDrug: B-Lymphocyte Stimulator (BLyS)National Cancer Institute (NCI)NULLCompletedN/AN/ABoth20Phase 1United States
441NCT00023192
(ClinicalTrials.gov)
August 200129/8/2001Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of CareTreatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of CareChronic Granulomatous DiseaseDrug: T-Cell Depleted & CD34+Select/w/StemCell Enriched ProductNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth60Phase 3United States
442NCT00802594
(ClinicalTrials.gov)
August 20013/12/2008A Trial of DB289 for the Treatment of Stage I African TrypanosomiasisPhase II A Trial of DB289 for the Treatment of Stage I African TrypanosomiasisTrypanosomiasis, AfricanDrug: DB289Immtech Pharmaceuticals, IncBill and Melinda Gates FoundationCompleted16 YearsN/ABoth30Phase 2Angola;Congo
443NCT00018018
(ClinicalTrials.gov)
June 20, 200127/6/2001Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) DeficiencyTreatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA GeneSevere Combined Immunodeficiency SyndromeDrug: CD34+ cells transduced with ADA retrovirNational Human Genome Research Institute (NHGRI)NULLCompleted1 MonthN/AAll8Phase 1United States
444NCT00330148
(ClinicalTrials.gov)
March 200124/5/2006Randomized Clinical Trial of Three Drug Combinations for Late-Stage Gambiense Human African TrypanosomiasisClinical Trial Comparing the Therapeutic Combinations Melarsoprol-Nifurtimox, Melarsoprol-Eflornithine and Eflornithine-Nifurtimox in the Treatment of Gambiense Human African Trypanosomiasis in the Meningo-Encephalitic PhaseTrypanosomiasis, AfricanDrug: 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 7dEpicentreMedecins Sans Frontieres;Embassy of France in Uganda;National Sleeping Sickness Control Program, UgandaTerminatedN/AN/ABoth435Phase 3Uganda
445NCT00013689
(ClinicalTrials.gov)
March 200128/3/2001Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative SyndromePilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphoproliferative DisorderDrug: Fansidar (pyrimethamine and sulfadoxine)National Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth8Phase 1United States
446NCT00031486
(ClinicalTrials.gov)
September 20006/3/2002Long Term Treatment of Herpes Simplex Encephalitis (HSE) With ValacyclovirA Phase III Double-Blind, Placebo-Controlled Trial of Long Term Therapy of Herpes Simplex Encephalitis (HSE): An Evaluation of Valacyclovir (CASG-204)EncephalitisDrug: Valacyclovir;Drug: PlaceboNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted12 YearsN/AAll91Phase 3United States;Canada;Sweden;United Kingdom
447NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, 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 RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: 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 therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
448NCT00176878
(ClinicalTrials.gov)
June 200012/9/2005Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLCompletedN/A35 YearsAll10Phase 2/Phase 3United States
449NCT00006054
(ClinicalTrials.gov)
March 20005/7/2000Allogeneic Bone Marrow Transplantation in Patients With Primary ImmunodeficienciesImmunologic 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 SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow TransplantationFairview University Medical CenterNULLTerminatedN/A35 YearsBothN/AUnited States
450NCT00006056
(ClinicalTrials.gov)
March 20005/7/2000Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic DisordersChediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantationFairview University Medical CenterNULLActive, not recruitingN/A55 YearsBoth40N/AUnited States
451NCT00001145
(ClinicalTrials.gov)
October 19993/11/1999Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM SyndromeStudy of Immune Responses and Safety of Recombinant CD40 Ligand in Patients With X-Linked Hyper IgM SyndromeImmunoproliferative DisorderDrug: Bacteriophage;Drug: rhuCD40L;Drug: KLHNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth5Phase 2United States
452NCT00001905
(ClinicalTrials.gov)
April 19993/11/1999Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type IInterferon Gamma Administration in Leukocyte Adhesion Deficiency Type ILeukocyte Adhesion Deficiency SyndromeDrug: Interferon gammaNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth5Phase 2United States
453NCT00004695
(ClinicalTrials.gov)
September 199724/2/2000Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable ImmunodeficiencyCommon Variable ImmunodeficiencyDrug: PEG-interleukin-2Mount Sinai School of MedicineNULLCompleted2 YearsN/ABoth48N/ANULL
454NCT00008450
(ClinicalTrials.gov)
August 11, 19976/1/2001Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantInduction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAdenosine 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 ImmunodeficiencyProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 1United States
455NCT00001476
(ClinicalTrials.gov)
June 1, 19953/11/1999Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-upGene Therapy Approach for Chronic Granulomatous DiseaseChronic Granulomatous Disease;Communicable DiseaseDrug: Gene Therapy Method for CGD;Device: Isolex 300i Magnetic Cell SelectorNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted5 YearsN/AAll14Phase 1United States
456NCT00004787
(ClinicalTrials.gov)
December 199424/2/2000Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure SyndromesShwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;ThrombocytopeniaDrug: filgrastimNational Center for Research Resources (NCRR)James Whitcomb Riley Hospital for ChildrenCompletedN/AN/ABoth20Phase 2NULL
457NCT00001317
(ClinicalTrials.gov)
May 19923/11/1999A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of ChildhoodA Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of ChildhoodChronic Granulomatous DiseaseDrug: interferon-gammaNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth100Phase 4United States
458NCT00576407
(ClinicalTrials.gov)
October 199117/12/2007Thymus Transplantation in DiGeorge Syndrome #668Phase II Study of Thymus Transplantation in Complete DiGeorge Syndrome #668DiGeorge Syndrome;Complete Typical DiGeorge AnomalyBiological: Cultured Thymus Tissue for Implantation (CTTI)M. Louise MarkertNational 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 GmbHCompletedN/AN/AAll26Phase 2United States
459NCT00001280
(ClinicalTrials.gov)
January 19913/11/1999Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous DiseaseItraconazole for the Prevention of Fungal Infections in Chronic Granulomatous DiseaseMycosesDrug: itraconazoleNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth100Phase 2United States
460NCT00001255
(ClinicalTrials.gov)
September 19903/11/1999Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History StudyTreatment 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 StudySevere Combined ImmunodeficiencyDrug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced LymphocytesNational Human Genome Research Institute (NHGRI)NULLCompletedN/AN/ABoth10N/AUnited States
461EUCTR2014-003607-30-Outside-EU/EEA
(EUCTR)
26/02/2015Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement TherapyA 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 AGNULLNAFemale: yes
Male: yes
49Phase 3United States
462EUCTR2016-003438-26-SK
(EUCTR)
01/02/2017Study 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.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
463EUCTR2020-004734-37-PL
(EUCTR)
16/06/2021Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency DiseasesProspective, 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.HNULLNAFemale: yes
Male: yes
50Phase 1;Phase 3United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany
464EUCTR2014-003605-15-Outside-EU/EEA
(EUCTR)
26/02/2015Extension 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 AGNULLNAFemale: yes
Male: yes
21United States
465EUCTR2011-005015-82-Outside-EU/EEA
(EUCTR)
10/01/2012Study 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 AGNULLNAFemale: yes
Male: yes
20United States
466EUCTR2009-011434-10-Outside-EU/EEA
(EUCTR)
27/01/2012Study 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 AGNULLNAFemale: yes
Male: yes
51United States
467EUCTR2020-003445-11-CZ
(EUCTR)
11/02/2021A 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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
54Phase 2Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Belgium;Poland;Germany;Netherlands
468EUCTR2018-000338-36-PL
(EUCTR)
06/11/2018Not ApplicableOpen-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.NULLNAFemale: yes
Male: yes
155Phase 3United States;Spain;Belgium;Poland;Australia;Tunisia;Norway;Germany;United Kingdom;Italy;India
469EUCTR2014-003608-61-Outside-EU/EEA
(EUCTR)
05/01/2015Study 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 BehringNULLNAFemale: yes
Male: yes
25Japan
470EUCTR2009-011152-22-FR
(EUCTR)
09/12/2010Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WASPhase 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
GENETHONNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2France
471EUCTR2014-003772-23-Outside-EU/EEA
(EUCTR)
14/04/2015Safety 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 AGNULLNAFemale: yes
Male: yes
55United States
472EUCTR2011-005679-18-Outside-EU/EEA
(EUCTR)
07/12/2011A 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 LimitedNULLNAFemale: yes
Male: yes
25Phase 4Chile;United States
473EUCTR2015-003290-15-PL
(EUCTR)
15/12/2016A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Hungary;Czech Republic;Spain;Poland;Germany;United Kingdom
474EUCTR2016-000468-41-DE
(EUCTR)
20/01/2021Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLIAn 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.NULLNAFemale: yes
Male: yes
42Phase 2;Phase 3France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy
475EUCTR2018-003199-10-DE
(EUCTR)
04/10/2018OPEN-LABEL STUDY: THERAPEUTIC USE OF TADEKINIG ALFA IN NLRC4 MUTATION AND XIAP DEFICIENCYOpen-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.NULLNA Female: yes
Male: yes
10 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Canada;Germany
476EUCTR2020-004734-37-SK
(EUCTR)
02/06/2021Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency DiseasesProspective, 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.HNULLNAFemale: yes
Male: yes
50Phase 1;Phase 3United States;Czechia;Slovakia;Poland;Ukraine;Russian Federation;Germany
477EUCTR2016-001631-12-Outside-EU/EEA
(EUCTR)
30/01/2017The pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiencyProspective 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 KKNULLNAFemale: yes
Male: yes
10Phase 3Japan
478EUCTR2015-005241-31-PL
(EUCTR)
01/07/2016n/aA 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.NULLNot RecruitingFemale: yes
Male: yes
180Phase 3United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;India;Poland;Belgium;Australia;Germany;Norway;Tunisia
479EUCTR2014-003609-14-Outside-EU/EEA
(EUCTR)
05/01/2015Follow-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 BehringNULLNAFemale: yes
Male: yes
23Japan
480EUCTR2010-023483-41-Outside-EU/EEA
(EUCTR)
11/04/2014A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune systemA 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 BIOTECHNOLOGIESNULLNAFemale: yes
Male: yes
60Poland;Serbia;Czech Republic;France;Hungary;Lithuania;Ukraine
481EUCTR2014-003409-13-Outside-EU/EEA
(EUCTR)
05/01/2015Multicenter 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 BehringNULLNAFemale: yes
Male: yes
22Japan
482EUCTR2016-003799-33-Outside-EU/EEA
(EUCTR)
30/01/2017Safety 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 LLCNULLNAFemale: yes
Male: yes
51Phase 4United States;Canada