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Inh    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
65Primary immunodeficiency26

65. Primary immunodeficiency    [ 413 clinical trials,   581 drugs,   (DrugBank: 97 drugs),   68 drug target genes,   202 drug target pathways]
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.
26 / 413 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT04618211
(ClinicalTrials.gov)		January 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.NULLNot yet recruiting18 Years75 YearsAll54Phase 2Canada
2NCT04528355
(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
3NCT04414046
(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
4EUCTR2019-001693-28-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: 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		20Phase 2Serbia;Belarus;Hungary;Czech Republic;Poland;Ukraine;Russian Federation;Bulgaria;Germany
5EUCTR2018-004489-32-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;United Kingdom
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6EUCTR2018-004489-32-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		60Phase 2United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;United Kingdom
7NCT04208412
(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.NULLActive, not recruiting18 YearsN/AAll68Phase 2United States;Austria;Czechia;Germany;Hungary;Italy;Netherlands;North Macedonia;Poland;United Kingdom
8EUCTR2018-004489-32-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		60Phase 2United States;Hungary;Poland;Austria;North Macedonia;Netherlands;Germany;United Kingdom
9EUCTR2018-004489-32-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 2Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Germany;Netherlands;United Kingdom
10EUCTR2018-004489-32-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]KalVista Pharmaceuticals Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		60Phase 2Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
11EUCTR2018-004489-32-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]KalVista Pharmaceuticals LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		60Phase 2United States;Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom
12EUCTR2018-000673-68-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]		Great Ormond Street Hospital for Children NHS TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes		5Phase 1United Kingdom
13EUCTR2018-001489-41-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]		Karolinska UniversitetssjukhusetNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		40Phase 2United States;Sweden
14NCT03576469
(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 CentersNULLRecruiting18 YearsN/AAll20Phase 4United States
15NCT03828279
(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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
16EUCTR2017-001275-23-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
17NCT04136028
(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
18NCT01962415
(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
19EUCTR2010-023483-41-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 BIOTECHNOLOGIESNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		60France;Hungary
20EUCTR2010-024253-36-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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
21EUCTR2005-003201-81-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
22NCT00358657
(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
23NCT00325078
(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
24NCT00263237
(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
25NCT00119431
(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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
26NCT00004787
(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

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