231. Alpha-1-antitrypsin deficiency Clinical trials / Disease details
Clinical trials : 93 / Drugs : 77 - (DrugBank : 8) / Drug target genes : 10 - Drug target pathways : 36
Showing 1 to 10 of 93 diseases
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 |
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1 | NCT05643495 (ClinicalTrials.gov) | February 23, 2023 | 29/11/2022 | A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype | A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitr ... | Alpha-1 Antitrypsin Deficiency | Drug: VX-864 | Vertex Pharmaceuticals Incorporated | NULL | Recruiting | 18 Years | 80 Years | All | 20 | Phase 2 | United States |
2 | NCT05727800 (ClinicalTrials.gov) | February 8, 2023 | 3/2/2023 | A Phase 1, First-in-human Study of VX-668 | A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-668 | Alpha-1 Antitrypsin Deficiency | Drug: VX-668;Drug: Placebo | Vertex Pharmaceuticals Incorporated | NULL | Recruiting | 18 Years | 55 Years | All | 114 | Phase 1 | United States |
3 | NCT05579431 (ClinicalTrials.gov) | October 13, 2022 | 10/10/2022 | A Phase 1, First-in-human Study of VX-634 | A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634 | Alpha 1-Antitrypsin Deficiency | Drug: VX-634;Drug: Placebo | Vertex Pharmaceuticals Incorporated | NULL | Recruiting | 18 Years | 55 Years | All | 114 | Phase 1 | United States |
4 | NCT05315921 (ClinicalTrials.gov) | March 22, 2022 | 30/3/2022 | Study of OsrhAAT or Placebo in Healthy Volunteers | A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, ... | Emphysema Secondary to Congenital AATD | Drug: OsrhAAT 1 mg/kg IV;Drug: OsrhAAT 3 mg/kg IV;Drug: OsrhAAT 10 mg/kg IV;Drug: OsrhAAT 20 mg/kg IV;Drug: OsrhAAT 40 mg/kg IV;Drug: OsrhAAT 60 mg/kg IV Drug: OsrhAAT 1 mg/kg IV;Drug: OsrhAAT 3 mg/kg IV;Drug: OsrhAAT 10 mg/kg IV;Drug: OsrhAAT 20 mg/kg I ... | Healthgen Biotechnology Corp. | NULL | Recruiting | 18 Years | 55 Years | All | 48 | Phase 1 | United States |
5 | EUCTR2019-000602-30-IE (EUCTR) | 25/05/2021 | 31/03/2020 | A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%). A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficac ... | A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%). - Phase III, Efficacy and Safety of Kamada-AAT for Inhalation A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficac ... | Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%), and with no history of two or more moderate or one or more severe exacerbations of COPD during the past year. MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limit ... | Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsin (AAT) Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsi ... | Kamada Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 220 | Phase 3 | Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden | ||
6 | NCT04764448 (ClinicalTrials.gov) | February 12, 2021 | 27/1/2021 | A Study of Belcesiran in Patients With AATLD | A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Ph ... | Alpha 1-Antitrypsin Deficiency | Drug: Belcesiran;Other: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | 75 Years | All | 46 | Phase 2 | Australia;Austria;Belgium;Canada;France;Germany;Ireland;Netherlands;New Zealand;Portugal;Spain;Sweden;United Kingdom Australia;Austria;Belgium;Canada;France;Germany;Ireland;Netherlands;New Zealand;Portugal;Spain;Swede ... |
7 | 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 clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ... | 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] A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect o ... | 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] alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Class ... | 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 Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Switzerland;I ... | ||
8 | 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 clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ... | 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] A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacody ... | 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] alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Class ... | 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 | ||
9 | 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 A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 i ... | 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] Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.1;Level: PT;Cla ... | 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 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Produc ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
10 | 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 clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ... | 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] A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect o ... | 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] alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.0;Level: LLT;Class ... | 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 |