231. α1−アンチトリプシン欠乏症 Alpha-1-antitrypsin deficiency Clinical trials / Disease details
臨床試験数 : 89 / 薬物数 : 89 - (DrugBank : 16) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 47
Showing 1 to 10 of 89 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05146882 (ClinicalTrials.gov) | December 2021 | 9/11/2021 | An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Di ... | A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Pa ... | Alpha 1-Antitrypsin Deficiency | Drug: Belcesiran | Dicerna Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 18 Years | 75 Years | All | 54 | Phase 2 | New Zealand |
| 2 | NCT04764448 (ClinicalTrials.gov) | February 12, 2021 | 27/1/2021 | A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease | A Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency A Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and ... | Alpha 1-Antitrypsin Deficiency | Drug: belcesiran;Other: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | 70 Years | All | 54 | Phase 2 | Australia;New Zealand;Spain;United Kingdom |
| 3 | 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 ... | ||
| 4 | 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 | United States;Portugal;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | EUCTR2019-004881-16-DE (EUCTR) | 17/09/2020 | 10/07/2020 | 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 | ||
| 8 | EUCTR2019-004881-16-GB (EUCTR) | 14/09/2020 | 21/07/2020 | 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 - VX19-864-101 Efficacy and Safety of VX-864 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: 23.0;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: 23.0;Level: PT;Cla ... | Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: ... | Vertex Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 9 | EUCTR2019-004881-16-SE (EUCTR) | 11/09/2020 | 14/07/2020 | 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: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Produ ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
| 10 | EUCTR2019-000602-30-FI (EUCTR) | 08/09/2020 | 08/05/2020 | 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 |