231. Alpha-1-antitrypsin deficiency Clinical trials / Disease details
Clinical trials : 89 / Drugs : 89 - (DrugBank : 16) / Drug target genes : 35 - Drug target pathways : 47
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 |