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 : 93 / 薬物数 : 77 - (DrugBank : 8) / 標的遺伝子数 : 10 - 標的パスウェイ数 : 36
Showing 1 to 10 of 45 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 | 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 |
2 | 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 ... |
3 | EUCTR2019-000602-30-SE (EUCTR) | 29/06/2020 | 24/04/2020 | 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) Other descriptive name: RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE 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 | Argentina;Finland;Belgium;Ireland;Netherlands;United Kingdom;Italy;Sweden | ||
4 | NCT04167345 (ClinicalTrials.gov) | January 13, 2020 | 15/11/2019 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 i ... | Alpha 1-Antitrypsin Deficiency | Drug: VX-814;Drug: Placebo | Vertex Pharmaceuticals Incorporated | NULL | Terminated | 18 Years | 80 Years | All | 48 | Phase 2 | United States;Canada;Germany;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) A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patien ... | 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 | NCT04174118 (ClinicalTrials.gov) | October 24, 2019 | 6/11/2019 | Study of DCR-A1AT in Healthy Adult Volunteers | A Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered Belcesiran in Healthy Adult Volunteers A Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study ... | Alpha 1-Antitrypsin Deficiency | Drug: belcesiran;Drug: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Active, not recruiting | 18 Years | 55 Years | All | 30 | Phase 1 | New Zealand;Sweden |
7 | EUCTR2019-000602-30-NL (EUCTR) | 30/09/2019 | 15/05/2019 | 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) Other descriptive name: RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE 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 | ||
8 | NCT03945292 (ClinicalTrials.gov) | August 7, 2019 | 8/5/2019 | Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) | A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, 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 | Drug: Fazisiran Injection;Other: Placebo | Arrowhead Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 75 Years | All | 40 | Phase 2 | United States;Germany;Italy;Netherlands;Portugal;Spain;Canada;Ireland;Sweden |
9 | NCT03636347 (ClinicalTrials.gov) | October 29, 2018 | 20/6/2018 | A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH9 ... | A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 Antitrypsin Deficiency. A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group ... | Alpha 1-Antitrypsin Deficiency;Emphysema;COPD | Drug: Placebo Oral Tablet;Drug: Alvelestat oral tablet - dose 1;Drug: Alvelestat oral tablet - dose 2 | Mereo BioPharma | Syneos Health | Completed | 18 Years | 75 Years | All | 99 | Phase 2 | United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom |
10 | NCT03385395 (ClinicalTrials.gov) | July 2018 | 1/12/2017 | Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® ... | A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed Preparation Glassia® (Kamada Ltd.) in Subjects With Alpha-1-antitrypsin Deficiency A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intr ... | Alpha 1-Antitrypsin Deficiency | Drug: OctaAlpha1;Drug: Glassia | Octapharma | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | NULL |