231. Alpha-1-antitrypsin deficiency Clinical trials / Disease details
Clinical trials : 93 / Drugs : 77 - (DrugBank : 8) / Drug target genes : 10 - Drug target pathways : 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 | EUCTR2018-003385-14-AT (EUCTR) | 28/01/2021 | 14/09/2020 | 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 | Not Recruiting | Female: yes Male: yes | 120 | Phase 2;Phase 3 | Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 2 | EUCTR2018-003385-14-DE (EUCTR) | 05/01/2021 | 25/05/2020 | 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 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of 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 | 36 | Phase 2 | Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 3 | 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 | ||
| 4 | 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 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of 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 | Not Recruiting | Female: yes Male: yes | 36 | Phase 2 | Portugal;United States;Canada;Spain;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden;Ireland | ||
| 5 | NCT03946449 (ClinicalTrials.gov) | December 19, 2019 | 8/5/2019 | Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (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 | Drug: ARO-AAT Injection | Arrowhead Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 75 Years | All | 16 | Phase 2 | Austria;Germany;United Kingdom |
| 6 | 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 | ||
| 7 | 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 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of 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 | 36 | Phase 2 | Portugal;United States;Canada;Spain;Ireland;Austria;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 8 | 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 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of 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 | 36 | Phase 2 | United States;Portugal;Canada;Spain;Poland;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 9 | 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 | ||
| 10 | EUCTR2018-003385-14-IT (EUCTR) | 05/09/2019 | 19/01/2021 | 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). | 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) - AROAAT2001 | 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] | ARROWHEAD PHARMACEUTICALS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 118 | Phase 2 | Portugal;United States;Spain;Poland;Ireland;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 11 | 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 | Not Recruiting | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Portugal;Canada;Spain;Poland;Ireland;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 12 | 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 | ||
| 13 | 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 |
| 14 | 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 |