231. α1−アンチトリプシン欠乏症 Alpha-1-antitrypsin deficiency Clinical trials / Disease details
臨床試験数 : 93 / 薬物数 : 77 - (DrugBank : 8) / 標的遺伝子数 : 10 - 標的パスウェイ数 : 36
| 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 | NCT05643495 (ClinicalTrials.gov) | February 23, 2023 | 29/11/2022 | A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype | A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks | Alpha-1 Antitrypsin Deficiency | Drug: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Recruiting | 18 Years | 80 Years | All | 20 | Phase 2 | United States |
| 2 | EUCTR2019-004881-16-IE (EUCTR) | 29/10/2020 | 09/07/2020 | Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects | Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.1;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
| 3 | EUCTR2019-004881-16-DE (EUCTR) | 17/09/2020 | 10/07/2020 | Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects | Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.1;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
| 4 | EUCTR2019-004881-16-GB (EUCTR) | 14/09/2020 | 21/07/2020 | Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects - VX19-864-101 Efficacy and Safety of VX-864 | Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 5 | EUCTR2019-004881-16-SE (EUCTR) | 11/09/2020 | 14/07/2020 | Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects | Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.1;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden |