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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004489-32-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
2EUCTR2018-004489-32-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
3EUCTR2018-004489-32-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
4EUCTR2018-004489-32-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
5JPRN-jRCTs031180398
30/12/201720/03/2019A 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
Kato MotohiroNULLRecruitingNot applicable< 25age oldBoth22Phase 2None (Japan only);Japan