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 | NCT03767829 (ClinicalTrials.gov) | December 5, 2018 | 5/12/2018 | A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | Drug: ALN-AAT02;Drug: Placebo | Alnylam Pharmaceuticals | NULL | Terminated | 18 Years | 65 Years | All | 32 | Phase 1/Phase 2 | United Kingdom |
| 2 | EUCTR2018-001362-41-GB (EUCTR) | 03/12/2018 | 20/06/2019 | A 2-Part Safety and Tolerability Study of ALN-AAT02 in Healthy Volunteers and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease MedDRA version: 20.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALN-AAT02 Product Code: ALN-AAT02 INN or Proposed INN: ALN-77412 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 1;Phase 2 | United States;Germany;United Kingdom | ||
| 3 | EUCTR2015-001297-18-GB (EUCTR) | 29/06/2015 | 15/05/2015 | The First-in-Human Study of an Investigational Drug, ALN-AAT, in Healthy Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending, and Multiple-Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease MedDRA version: 19.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALN-AAT Product Code: ALN-AAT INN or Proposed INN: ALN-61444 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 66 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom |