231. Alpha-1-antitrypsin deficiency
83 clinical trials, 89 drugs (DrugBank: 16 drugs), 35 drug target genes, 46 drug target pathways
Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
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 | EUCTR2018-003385-14-GB (EUCTR) | 19/10/2020 | 28/06/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.0;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10001806 - Alpha-1 anti-trypsin deficiency;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;United Kingdom;Sweden | ||
| 2 | EUCTR2018-003385-14-SE (EUCTR) | 31/01/2020 | 25/03/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.0;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10001806 - Alpha-1 anti-trypsin deficiency;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;Sweden | ||
| 3 | NCT03946449 (ClinicalTrials.gov) | December 19, 2019 | 8/5/2019 | Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AAT | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection | Arrowhead Pharmaceuticals | NULL | Recruiting | 18 Years | 75 Years | All | 12 | Phase 2 | Austria;Germany;United Kingdom |
| 4 | EUCTR2019-000068-86-AT (EUCTR) | 14/11/2019 | 12/06/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 2 | Austria;Germany;United Kingdom | ||
| 5 | EUCTR2018-003385-14-NL (EUCTR) | 07/11/2019 | 24/07/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Canada;Spain;Ireland;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden | ||
| 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 |
| 6 | EUCTR2018-003385-14-PT (EUCTR) | 04/11/2019 | 09/07/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) | alpha-1 antitrypsin deficiency (AATD)-associated 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] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Poland;Spain;Ireland;Netherlands;Italy;Sweden | ||
| 7 | EUCTR2018-003385-14-ES (EUCTR) | 11/09/2019 | 11/06/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) | alpha-1 antitrypsin deficiency (AATD)-associated 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] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Poland;Spain;Ireland;Netherlands;Italy;Sweden | ||
| 8 | NCT03945292 (ClinicalTrials.gov) | August 7, 2019 | 8/5/2019 | Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection;Other: Placebo | Arrowhead Pharmaceuticals | NULL | Recruiting | 18 Years | 75 Years | All | 120 | Phase 2;Phase 3 | United States;Canada;Ireland;Italy;Netherlands;Portugal;Spain;Sweden |
| 9 | EUCTR2018-003385-14-IE (EUCTR) | 06/08/2019 | 21/03/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.0;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10001806 - Alpha-1 anti-trypsin deficiency;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;Sweden | ||
| 10 | EUCTR2019-000068-86-GB (EUCTR) | 01/07/2019 | 03/04/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 2 | Austria;Germany;United Kingdom | ||
| 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 |
| 11 | NCT03362242 (ClinicalTrials.gov) | March 12, 2018 | 30/11/2017 | Study of ARO-AAT in Normal Adult Volunteers | A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection;Other: Sterile Normal Saline (0.9% NaCl) | Arrowhead Pharmaceuticals | NULL | Completed | 18 Years | 55 Years | All | 45 | Phase 1 | New Zealand |
| 12 | EUCTR2019-000068-86-DE (EUCTR) | 04/06/2019 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001 | Arrowhead Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 2 | Austria;Germany;United Kingdom |