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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
12 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-003385-14-GB
(EUCTR)
19/10/202028/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;United Kingdom;Sweden
2EUCTR2018-003385-14-SE
(EUCTR)
31/01/202025/03/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;Sweden
3NCT03946449
(ClinicalTrials.gov)
December 19, 20198/5/2019Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AATA 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 DeficiencyDrug: ARO-AAT InjectionArrowhead PharmaceuticalsNULLRecruiting18 Years75 YearsAll12Phase 2Austria;Germany;United Kingdom
4EUCTR2019-000068-86-AT
(EUCTR)
14/11/201912/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom
5EUCTR2018-003385-14-NL
(EUCTR)
07/11/201924/07/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Ireland;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2018-003385-14-PT
(EUCTR)
04/11/201909/07/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Poland;Spain;Ireland;Netherlands;Italy;Sweden
7EUCTR2018-003385-14-ES
(EUCTR)
11/09/201911/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Poland;Spain;Ireland;Netherlands;Italy;Sweden
8NCT03945292
(ClinicalTrials.gov)
August 7, 20198/5/2019Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AATA 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 DeficiencyDrug: ARO-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsNULLRecruiting18 Years75 YearsAll120Phase 2;Phase 3United States;Canada;Ireland;Italy;Netherlands;Portugal;Spain;Sweden
9EUCTR2018-003385-14-IE
(EUCTR)
06/08/201921/03/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;Sweden
10EUCTR2019-000068-86-GB
(EUCTR)
01/07/201903/04/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03362242
(ClinicalTrials.gov)
March 12, 201830/11/2017Study of ARO-AAT in Normal Adult VolunteersA 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 VolunteersAlpha 1-Antitrypsin DeficiencyDrug: ARO-AAT Injection;Other: Sterile Normal Saline (0.9% NaCl)Arrowhead PharmaceuticalsNULLCompleted18 Years55 YearsAll45Phase 1New Zealand
12EUCTR2019-000068-86-DE
(EUCTR)
04/06/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom