65. 原発性免疫不全症候群
［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04618211 (ClinicalTrials.gov) | January 2021 | 26/10/2020 | Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema | A Phase II, Double-blind, Placebo-controlled, Randomized, Cross-over, Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-inhibitor Deficiency Type I and II | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor Deficiency;C1 Inhibitor Deficiency | Drug: PHA-022121;Drug: Placebo | Pharvaris Netherlands B.V. | NULL | Not yet recruiting | 18 Years | 75 Years | All | 54 | Phase 2 | Canada |
| 2 | NCT04414046 (ClinicalTrials.gov) | July 22, 2020 | 24/5/2020 | TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Primary Immune Deficiency Disorders;Metabolic Disease | Biological: Haploidentical Hematopoietic Cell Transplantation | Johns Hopkins All Children's Hospital | NULL | Recruiting | N/A | 21 Years | All | 17 | Phase 2 | United States |
| 3 | EUCTR2019-001693-28-DE (EUCTR) | 06/02/2020 | 02/09/2019 | Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older | Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with congenital C1-INH deficiency and hereditary angioedema - CONE-01 | Hereditary angioedema type I and type II MedDRA version: 20.0;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: C1 Esterase Inhibitor Human Product Code: OCTA-C1-INH Other descriptive name: C1 ESTERASE INHIBITOR (HUMAN) | Octapharma Pharmazeutika Produktionsges.m.b.H. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Serbia;Belarus;Hungary;Czech Republic;Poland;Ukraine;Russian Federation;Bulgaria;Germany | ||
| 4 | EUCTR2018-004489-32-AT (EUCTR) | 02/10/2019 | 07/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;United Kingdom | ||
| 5 | EUCTR2018-004489-32-PL (EUCTR) | 29/08/2019 | 29/05/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT04208412 (ClinicalTrials.gov) | July 2, 2019 | 20/12/2019 | A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | A Randomized, Double-blind, Placebo-controlled, Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: KVD900;Drug: Placebo | KalVista Pharmaceuticals, Ltd. | NULL | Active, not recruiting | 18 Years | N/A | All | 68 | Phase 2 | United States;Austria;Czechia;Germany;Hungary;Italy;Netherlands;North Macedonia;Poland;United Kingdom |
| 7 | EUCTR2018-004489-32-DE (EUCTR) | 24/06/2019 | 12/02/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Poland;Austria;North Macedonia;Netherlands;Germany;United Kingdom | ||
| 8 | EUCTR2018-004489-32-NL (EUCTR) | 19/06/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Germany;Netherlands;United Kingdom | ||
| 9 | EUCTR2018-004489-32-HU (EUCTR) | 21/05/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | KalVista Pharmaceuticals Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom | |||
| 10 | EUCTR2018-004489-32-GB (EUCTR) | 13/03/2019 | 27/12/2018 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Phase 1 | United Kingdom | |||
| 12 | EUCTR2018-001489-41-SE (EUCTR) | 02/07/2018 | 08/05/2018 | A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions. | Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease | Dyskeratosis congenita / telomere disease MedDRA version: 20.0;Level: PT;Classification code 10001756;Term: Allogenic bone marrow transplantation therapy;System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Karolinska Universitetssjukhuset | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 2 | United States;Sweden | |||
| 13 | NCT03576469 (ClinicalTrials.gov) | June 13, 2018 | 15/6/2018 | A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | CVI - Common Variable Immunodeficiency | Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) | IMMUNOe Research Centers | NULL | Recruiting | 18 Years | N/A | All | 20 | Phase 4 | United States |
| 14 | NCT03828279 (ClinicalTrials.gov) | October 1, 2017 | 23/1/2019 | Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and II | The Global Registry on Hereditary Angioedema Type I and II | Hereditary Angioedema Type I and II | Diagnostic Test: functional and antigenic C1 inhibitor | HAE Global Registry Foundation | NULL | Recruiting | N/A | N/A | All | 220 | Italy | |
| 15 | EUCTR2017-001275-23-GB (EUCTR) | 21/09/2017 | 03/07/2017 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells Product Code: OTL-101 INN or Proposed INN: There is no recommended INN Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT04136028 (ClinicalTrials.gov) | September 25, 2015 | 21/10/2019 | IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease | A Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Kineret | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Completed | N/A | 18 Years | All | 13 | Early Phase 1 | Russian Federation |
| 17 | EUCTR2010-023483-41-FR (EUCTR) | 24/04/2013 | 04/01/2011 | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | a primary immunodeficiency as defined by the ESID and validated by a reference centre :• X-linked agammaglobulinemia (XLA)• Autosomal recessive inherited agammaglobulinemia (including autosomal recessive hyper-IgM syndrome)• Common variable immunodeficiency (CVID)• IgG subclass deficiency (at least 2 subclasses) with clinical manifestations of immunodeficiency MedDRA version: 12.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | France;Hungary | |||
| 18 | EUCTR2010-024253-36-GB (EUCTR) | 03/08/2012 | 09/05/2012 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1aS-ADA lentiviral vector mediated gene modification of autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced patient CD34+ cells INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 19 | EUCTR2005-003201-81-DE (EUCTR) | 20/12/2006 | 19/01/2007 | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | Herpes simplex virus type-1 (HSV-1) is the most frequent cause of fatal sporadic encephalitis in humans. Herpes simplex encephalitis (HSE) was one of the first viral infections to be successfully treated with antiviral drugs. Mortality has been significantly reduced since the introduction of acyclovir, a specific inhibitor of HSV replication. Despite appropriate and promptly initiated antiviral therapy the incidence of persistent neurological deficits remain unacceptably high. | Trade Name: Fortecortin Inject 40 mg Amp. Product Name: Fortecortin INN or Proposed INN: dexamethasone 21-dihydrogen phosphate | Universitätsklinikum Heidelberg | NULL | Not Recruiting | Female: yes Male: yes | 450 | Phase 3 | Germany | ||
| 20 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT00325078 (ClinicalTrials.gov) | May 2006 | 11/5/2006 | Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease | Tumor Necrosis Factor Alpha Inhibitor (Lnfliximab, Adalimumab) Treatment for Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease: A Phase I/II Study Assessing Clinical and Immune Responses to Treatment and Genetic Influences | Chronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD) | Drug: Infliximab | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 10 Years | N/A | All | 40 | Phase 1;Phase 2 | United States |
| 22 | NCT00263237 (ClinicalTrials.gov) | December 2, 2005 | 7/12/2005 | STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable Immunodeficiency | A Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: STA-5326 | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Years | 75 Years | All | 10 | Phase 1 | United States |
| 23 | NCT00119431 (ClinicalTrials.gov) | September 2005 | 4/7/2005 | Kinetics, Efficacy and Safety of C1-Esteraseremmer-N | Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema | Hereditary Angioedema Type I;Angioneurotic Edema | Drug: C1 inhibitor concentrate | Sanquin | NULL | Completed | 18 Years | N/A | Both | 12 | Phase 2 | Netherlands |
| 24 | NCT00004787 (ClinicalTrials.gov) | December 1994 | 24/2/2000 | Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes | Shwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;Thrombocytopenia | Drug: filgrastim | National Center for Research Resources (NCRR) | James Whitcomb Riley Hospital for Children | Completed | N/A | N/A | Both | 20 | Phase 2 | NULL |