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 | EUCTR2016-000468-41-GB (EUCTR) | 25/02/2019 | 12/03/2018 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An 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 AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Italy;United Kingdom | ||
| 2 | EUCTR2016-000468-41-IE (EUCTR) | 25/10/2018 | 27/02/2018 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An 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 AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;France;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2016-000468-41-CZ (EUCTR) | 19/07/2016 | 25/05/2016 | Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI | An 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 | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;France;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 4 | NCT02435173 (ClinicalTrials.gov) | August 24, 2015 | 24/2/2015 | Study of Efficacy of CDZ173 in Patients With APDS/PASLI | An 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/PASLI | Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells;Lymphadenopathy and Immunodeficiency (PASLI) | Drug: CDZ173 | Novartis Pharmaceuticals | NULL | Recruiting | 12 Years | 75 Years | All | 36 | Phase 2;Phase 3 | United States;Belarus;Czechia;Ireland;Italy;Netherlands;Russian Federation;United Kingdom;Czech Republic |