65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
| 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-FR (EUCTR) | 12/01/2021 | 02/12/2020 | 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 | Not Recruiting | Female: yes Male: yes | 42 | Phase 2;Phase 3 | United States;Belarus;France;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | 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 | ||
| 3 | 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 | Not Recruiting | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 4 | EUCTR2016-000468-41-IT (EUCTR) | 01/06/2018 | 02/09/2021 | 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 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 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy | ||
| 5 | EUCTR2016-000468-41-NL (EUCTR) | 03/11/2016 | 19/09/2016 | 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 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 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Germany;Netherlands;United Kingdom;Italy | ||
| 6 | 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 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 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy | ||
| 7 | 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), APDS / PASLI | Drug: CDZ173;Other: Placebo | Novartis Pharmaceuticals | NULL | Completed | 12 Years | 75 Years | All | 37 | Phase 2/Phase 3 | United States;Belarus;Czechia;Germany;Ireland;Italy;Netherlands;Russian Federation;United Kingdom;Czech Republic;France |
| 8 | EUCTR2016-000468-41-DE (EUCTR) | 20/01/2021 | 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 | Pharming Technologies B.V. | NULL | NA | Female: yes Male: yes | 42 | Phase 2;Phase 3 | France;United States;Belarus;Czechia;Czech Republic;Ireland;Russian Federation;Netherlands;Germany;United Kingdom;Italy |