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.
| 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 | 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 | ||
| 3 | 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 |
| 4 | 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 | |
| 5 | 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 |