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
| 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 | 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 | 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 | 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 | 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 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 | 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] | Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsin (AAT) | 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 | 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 |
| 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 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 | ||
| 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 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 | ||
| 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 | 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] | 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 | 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 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 | ||
| 11 | 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 | 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] | 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 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
| 12 | 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 | 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] | 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 | 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 | ||
| 13 | 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 | 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] | 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 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Canada;Ireland;Germany;United Kingdom;Sweden | ||
| 14 | 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 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 | 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] | Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsin (AAT) | Kamada Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 220 | Phase 3 | Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden | ||
| 15 | EUCTR2019-000602-30-BE (EUCTR) | 23/07/2020 | 12/06/2020 | 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 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 | 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] | Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsin (AAT) | Kamada Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 220 | Phase 3 | Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden | ||
| 16 | EUCTR2019-000602-30-GB (EUCTR) | 02/07/2020 | 17/03/2020 | 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 Airflow Limitation (50% = FEV1 = 80% of predicted; FEV1/SVC = 70%). | 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 Airflow Limitation (50% = FEV1 = 80% of predicted; FEV1/SVC = 70%). - Phase III, Efficacy and Safety of Kamada-AAT for Inhalation | Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate Airflow Limitation (50% = 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] | Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsin (AAT) | Kamada Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 220 | Phase 3 | Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden | ||
| 17 | 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 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 | 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] | 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 | 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 | ||
| 18 | EUCTR2019-003650-92-SE (EUCTR) | 08/05/2020 | 09/12/2019 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects | Alpha-1 Antitrypsin Deficiency;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: VX-814 INN or Proposed INN: VX-814 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 2 | United States;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 19 | EUCTR2019-002501-22-GB (EUCTR) | 04/05/2020 | 30/10/2019 | A Multi-Center, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety and Tolerability of the Neutrophil Elastase Inhibitor PHP-303 in Adults with Alpha-1 Antitrypsin Deficiency (AATD) | A Multi-Center, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety and Tolerability of the Neutrophil Elastase Inhibitor PHP-303 in Adults with Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Code: PHP-303 INN or Proposed INN: Not applicable Other descriptive name: (4S)-4-[4-cyano-2-(methylsulfonyl)phenyl]-3,6-dimethyl-2-oxo-1-[3-(trifluoromethyl)phenyl]-1,2,3,4-tetrahydropyrimidine-5-carbonitrile | pH Pharma Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United Kingdom | ||
| 20 | EUCTR2019-003650-92-IE (EUCTR) | 09/04/2020 | 10/03/2020 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects | Alpha-1 Antitrypsin Deficiency;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: VX-814 INN or Proposed INN: VX-814 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 2 | United States;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 21 | EUCTR2019-003650-92-GB (EUCTR) | 28/02/2020 | 09/12/2019 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects | Alpha-1 Antitrypsin Deficiency;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: VX-814 INN or Proposed INN: VX-814 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 2 | United States;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 22 | EUCTR2019-003650-92-DE (EUCTR) | 11/02/2020 | 04/12/2019 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects | Alpha-1 Antitrypsin Deficiency;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: VX-814 INN or Proposed INN: VX-814 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 2 | United States;Ireland;Netherlands;Germany;United Kingdom;Sweden | ||
| 23 | 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 | ||
| 24 | 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 | 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 |
| 25 | 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 |
| 26 | 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 | ||
| 27 | 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 | ||
| 28 | 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 | ||
| 29 | 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 | 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 |
| 30 | 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 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 | 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] | 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 | Kamada Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 220 | Phase 3 | Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden | ||
| 31 | 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 | ||
| 32 | EUCTR2018-001309-95-BE (EUCTR) | 06/09/2019 | 02/07/2019 | A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiency | 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. | Alpha-1 antitrypsin deficiency MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Alvelestat Product Code: MPH966 INN or Proposed INN: Alvelestat tosylate Other descriptive name: ALVELESTAT | Mereo BioPharma 4 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 182 | Phase 2 | United States;Canada;Spain;Poland;Belgium;Denmark;United Kingdom;Sweden | ||
| 33 | 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 | ||
| 34 | 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] | 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 |
| 35 | 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 | ||
| 36 | NCT03815396 (ClinicalTrials.gov) | July 19, 2019 | 21/1/2019 | Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency | An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency;AATD | Drug: INBRX-101/rhAAT-Fc | Inhibrx, Inc. | NULL | Completed | 18 Years | 80 Years | All | 31 | Phase 1 | United States;New Zealand;United Kingdom |
| 37 | 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 | ||
| 38 | EUCTR2018-001309-95-PL (EUCTR) | 05/04/2019 | 10/12/2018 | A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 (PiZZ or null genotype/phenotype) antitrypsin deficiency | 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 (PiZZ or null genotype/phenotype) antitrypsin deficiency. | Alpha-1 antitrypsin deficiency MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Alvelestat Product Code: MPH966 INN or Proposed INN: Alvelestat tosylate Other descriptive name: ALVELESTAT | Mereo BioPharma 4 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 182 | Phase 2 | United States;Canada;Spain;Belgium;Poland;Denmark;United Kingdom;Sweden | ||
| 39 | NCT03804021 (ClinicalTrials.gov) | January 11, 2019 | 11/1/2019 | Long-Term Follow-up Study of ADVM-043 | Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency | Alpha-1 Antitrypsin Deficiency | Genetic: ADVM-043 | Adverum Biotechnologies, Inc. | NULL | Enrolling by invitation | 18 Years | N/A | All | 6 | United States | |
| 40 | NCT03767829 (ClinicalTrials.gov) | December 5, 2018 | 5/12/2018 | A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | Drug: ALN-AAT02;Drug: Placebo | Alnylam Pharmaceuticals | NULL | Terminated | 18 Years | 65 Years | All | 32 | Phase 1/Phase 2 | United Kingdom |
| 41 | EUCTR2018-001362-41-GB (EUCTR) | 03/12/2018 | 20/06/2019 | A 2-Part Safety and Tolerability Study of ALN-AAT02 in Healthy Volunteers and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALN-AAT02 Product Code: ALN-AAT02 INN or Proposed INN: ALN-77412 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 1;Phase 2 | United States;Germany;United Kingdom | ||
| 42 | EUCTR2018-001309-95-GB (EUCTR) | 30/11/2018 | 31/07/2018 | A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiency | 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. | Alpha-1 antitrypsin deficiency 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] - Respiratory Tract Diseases [C08] | Product Name: Alvelestat Product Code: MPH966 INN or Proposed INN: Alvelestat tosylate Other descriptive name: ALVELESTAT | Mereo BioPharma 4 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 182 | Phase 2 | United States;Canada;Poland;Belgium;Spain;Denmark;United Kingdom;Sweden | ||
| 43 | EUCTR2018-001309-95-DK (EUCTR) | 14/11/2018 | 13/09/2018 | A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiency | 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. | Alpha-1 antitrypsin deficiency 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] - Respiratory Tract Diseases [C08] | Product Name: Alvelestat Product Code: MPH966 INN or Proposed INN: Alvelestat tosylate Other descriptive name: ALVELESTAT | Mereo BioPharma 4 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 182 | Phase 2 | Canada;Sweden;Belgium;United States;Denmark;Poland;United Kingdom;Spain | ||
| 44 | 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 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. | 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 |
| 45 | EUCTR2018-001309-95-SE (EUCTR) | 16/10/2018 | 06/09/2018 | A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiency | 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. | Alpha-1 antitrypsin deficiency 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] - Respiratory Tract Diseases [C08] | Product Name: Alvelestat Product Code: MPH966 INN or Proposed INN: Alvelestat tosylate Other descriptive name: ALVELESTAT | Mereo BioPharma 4 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 182 | Phase 2 | Canada;Sweden;Belgium;United States;Denmark;Poland;United Kingdom;Spain | ||
| 46 | 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 | 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 | Alpha 1-Antitrypsin Deficiency | Drug: OctaAlpha1;Drug: Glassia | Octapharma | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | NULL |
| 47 | EUCTR2015-004110-23-PL (EUCTR) | 19/04/2018 | 16/02/2018 | A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), ModifiedProcess 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Estonia;Canada;Poland;Denmark;Australia;Russian Federation;New Zealand;Sweden | ||
| 48 | 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 |
| 49 | NCT03172455 (ClinicalTrials.gov) | May 1, 2017 | 29/5/2017 | Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) | Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) | Acute-graft-versus-host Disease;Steroid Refractory Acute Graft Versus Host Disease;Graft-versus-host-disease;Graft Vs Host Disease;Alpha 1-Antitrypsin Deficiency;Alpha-1 Proteinase Inhibitor;Alpha-1 Protease Inhibitor Deficiency;Acute Graft-Versus-Host Reaction Following Bone Marrow Transplant | Drug: Glassia | Impatients N.V. trading as myTomorrows | Kamada, Ltd. | No longer available | N/A | N/A | All | NULL | ||
| 50 | NCT03114020 (ClinicalTrials.gov) | March 22, 2017 | 5/4/2017 | Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency | Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema | Emphysema;Alpha 1-Antitrypsin Deficiency | Drug: Hyaluronic Acid Inhalation Solution;Drug: Placebo Inhalation Solution | Gerard Turino | NULL | Completed | 18 Years | 80 Years | All | 27 | Phase 2 | United States;Canada |
| 51 | NCT03008915 (ClinicalTrials.gov) | January 2017 | 22/12/2016 | Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency | Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency | Alpha-1 Antitrypsin Deficiency;Emphysema;Chronic Obstructive Pulmonary Disease | Drug: Aspirin;Drug: Placebo;Other: Withdrawal from alpha1 antitrypsin replacement therapy | Columbia University | Alpha-1 Foundation;Stony Wold-Herbert Fund, Inc. | Active, not recruiting | 40 Years | N/A | All | 15 | Phase 2 | United States |
| 52 | NCT02900183 (ClinicalTrials.gov) | October 2016 | 9/9/2016 | Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels | An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as Evidenced by Changes in Liver Biopsy in Patients With Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency | Drug: ARC-AAT Injection | Arrowhead Pharmaceuticals | NULL | Withdrawn | 18 Years | 75 Years | All | 0 | Phase 2 | Canada;Ireland;Italy;Sweden |
| 53 | EUCTR2016-000917-59-SE (EUCTR) | 04/08/2016 | 08/06/2016 | 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) | An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as evidenced by changes in liver biopsy in Patients with Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency related liver disease MedDRA version: 19.0;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: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API-AAT Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance Product Name: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API-AAT Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance Product Name: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API-AAT Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance | Arrowhead Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Canada;Ireland;Sweden | ||
| 54 | NCT02796937 (ClinicalTrials.gov) | July 2016 | 3/6/2016 | Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency | Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency | Biological: Alpha-1 MP | Grifols Therapeutics LLC | NULL | Enrolling by invitation | 20 Years | 72 Years | All | 250 | Phase 3 | United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden |
| 55 | EUCTR2016-000917-59-IE (EUCTR) | 29/06/2016 | 13/04/2016 | 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) | An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as evidenced by changes in liver biopsy in Patients with Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency related liver disease MedDRA version: 19.1;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: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API-AAT Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance | Arrowhead Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Canada;Ireland;Sweden | ||
| 56 | EUCTR2015-004110-23-DK (EUCTR) | 29/03/2016 | 29/01/2016 | A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alphal-Proteinase Inhibitor (Human), ModifiedProcess 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Estonia;Spain;Russian Federation;France;Canada;Argentina;Brazil;Poland;Romania;Denmark;Australia;Germany;New Zealand;Sweden | ||
| 57 | EUCTR2013-001870-38-DE (EUCTR) | 21/09/2015 | 27/03/2015 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 19.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 339 | Phase 3 | United States;Estonia;Finland;Spain;Ireland;Russian Federation;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Sweden | ||
| 58 | EUCTR2015-001297-18-GB (EUCTR) | 29/06/2015 | 15/05/2015 | The First-in-Human Study of an Investigational Drug, ALN-AAT, in Healthy Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending, and Multiple-Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease MedDRA version: 19.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALN-AAT Product Code: ALN-AAT INN or Proposed INN: ALN-61444 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 66 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 59 | NCT02810327 (ClinicalTrials.gov) | February 2015 | 20/6/2016 | Alpha-1 Carrier Genomics Study | Alpha-1 Carrier Genomics Study | Alpha-1 Antitrypsin Deficiency;Emphysema;COPD;Smoking | Genetic: Genetic Sequencing | Medical University of South Carolina | CSL Behring | Completed | 18 Years | N/A | All | 117 | United States | |
| 60 | NCT02363946 (ClinicalTrials.gov) | February 2015 | 2/2/2015 | A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) | A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect of Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients With Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency | Drug: ARC-AAT Injection;Other: Placebo | Arrowhead Pharmaceuticals | NULL | Terminated | 18 Years | 70 Years | All | 65 | Phase 1 | Australia;Germany;Netherlands;United Kingdom |
| 61 | EUCTR2013-001870-38-PL (EUCTR) | 30/10/2014 | 29/08/2014 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 17.1;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 339 | United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden | |||
| 62 | NCT02001688 (ClinicalTrials.gov) | April 2014 | 24/11/2013 | Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation | Phase II, Double-Blind, Placebo-Controlled Study to Explore the ELF and Plasma Concentration as Well as Safety of Inhaled Alpha-1 Antitrypsin in Alpha-1 Antitrypsin Deficiency Subjects | Alpha-1 Antitrypsin Deficiency | Drug: Kamada-AAT for Inhalation, 80mg;Drug: Placebo;Drug: Kamada-AAT for Inhalation, 160mg | Kamada, Ltd. | NULL | Completed | 18 Years | 65 Years | All | 36 | Phase 2 | United States |
| 63 | EUCTR2013-001870-38-IE (EUCTR) | 21/03/2014 | 27/09/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 16.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 339 | Phase 3 | Serbia;United States;Estonia;Slovakia;Finland;Spain;Ireland;Lithuania;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Brazil;Denmark;Australia;Germany;Netherlands;Latvia;Norway;New Zealand;Sweden | ||
| 64 | EUCTR2013-001870-38-FI (EUCTR) | 30/01/2014 | 30/12/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 339 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Serbia;United States;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Moldova, Republic of;Sweden | ||
| 65 | EUCTR2013-001870-38-SE (EUCTR) | 09/01/2014 | 23/09/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 339 | Phase 3 | United States;Serbia;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Brazil;Poland;Romania;Australia;Denmark;Germany;Moldova, Republic of;New Zealand;Sweden | ||
| 66 | EUCTR2013-001870-38-ES (EUCTR) | 26/12/2013 | 18/10/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 14.1;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 339 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Serbia;Estonia;Slovakia;Finland;Spain;Ireland;Lithuania;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Australia;Denmark;Germany;Norway;Netherlands;Latvia;New Zealand;Sweden | ||
| 67 | EUCTR2013-001870-38-DK (EUCTR) | 20/11/2013 | 04/10/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 339 | Phase 3 | Serbia;United States;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Argentina;Poland;Brazil;Romania;Denmark;Australia;Germany;Moldova, Republic of;New Zealand;Sweden | ||
| 68 | EUCTR2013-001870-38-EE (EUCTR) | 12/11/2013 | 01/10/2013 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN | Grifols Therapeutics LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 339 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Serbia;United States;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Moldova, Republic of;Sweden | ||
| 69 | NCT01669421 (ClinicalTrials.gov) | July 2012 | 14/8/2012 | Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation. | Effect of a Higher Dose of Alpha-1 Antitrypsin Augmentation Therapy on Lung Inflammation in Subjects With Alpha-1 Antitrypsin Deficiency. | Alpha 1 Antitrypsin Deficiency | Drug: Alpha-1 Antitrypsin (human) | Michael Campos, MD | CSL Behring | Completed | 18 Years | 75 Years | All | 10 | Phase 2 | United States |
| 70 | NCT01379469 (ClinicalTrials.gov) | January 2012 | 15/6/2011 | Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency | A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency | Alpha-1-antitrypsin Deficiency;Liver Cirrhosis | Drug: Drug-Carbamazepine (Tegretol XR);Drug: Carbamazepine (Tegretol XR) Placebo | Washington University School of Medicine | Novartis;National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of Pittsburgh | Terminated | 14 Years | 80 Years | All | 20 | Phase 2 | United States |
| 71 | EUCTR2008-005326-36-SE (EUCTR) | 06/10/2011 | 11/09/2009 | A clinical trial looking at the safety and effectiveness of a new drug for inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with Emphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Product Code: Kamada-AAT for inhalation Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2/3 | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | ||
| 72 | EUCTR2009-014286-20-IE (EUCTR) | 21/12/2010 | 31/05/2010 | A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha 1 Antitrypsin (rAAV1-CB-hAAT) in Patients with Alpha 1 Antitrypsin Deficiency - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency | A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha 1 Antitrypsin (rAAV1-CB-hAAT) in Patients with Alpha 1 Antitrypsin Deficiency - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency | Alpha 1 antitrypsin deficiency.This is an inherited, genetic condition characterized by reduced serum levels of alpha-1 antitrypsin (AAT) which increases the risk of developing emphysema and liver disease MedDRA version: 12.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency | Product Name: rAAV1-CB-hAAT Product Code: rAAV1-CB-hAAT | Applied Genetic Technologies Corporation | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Ireland | ||
| 73 | NCT01213043 (ClinicalTrials.gov) | November 2010 | 29/9/2010 | Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency | A Randomized Double-blind Crossover Study to Assess the Safety and Pharmacokinetics of Two Different Doses of Weekly Intravenous Administration of Alpha1-Proteinase Inhibitor (Human) Prolastin®-C in Subjects With Alpha1-Antitrypsin Deficiency | Emphysema;Alpha 1-antitrypsin Deficiency (AATD) | Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kg | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | 70 Years | All | 30 | Phase 2 | United States |
| 74 | EUCTR2008-005326-36-DE (EUCTR) | 01/10/2010 | 28/04/2010 | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 15.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Product Code: Kamada-AAT for inhalation Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2/3 | Ireland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 75 | EUCTR2008-005326-36-IE (EUCTR) | 09/07/2010 | 12/05/2010 | A clinical trial looking at the safety and effectiveness of a new drug for inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with Emphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Product Code: Kamada-AAT for inhalation Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2/3 | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | ||
| 76 | NCT01054339 (ClinicalTrials.gov) | June 2010 | 21/1/2010 | Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency | A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha-1 Antitrypsin (rAAV1-CB-hAAT) in Patients With Alpha-1 Antitrypsin Deficiency | Alpha-1 Antitrypsin Deficiency | Drug: rAAV1-CB-hAAT | Applied Genetic Technologies Corp | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 18 Years | 75 Years | All | 9 | Phase 2 | United States;Ireland |
| 77 | EUCTR2008-005326-36-NL (EUCTR) | 05/02/2010 | 21/08/2009 | A clinical trial looking at the safety and effectiveness of a new drug forinhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients withEmphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Product Code: Kamada-AAT for inhalation Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2;Phase 3 | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | ||
| 78 | EUCTR2008-005326-36-DK (EUCTR) | 13/01/2010 | 14/09/2009 | A clinical trial looking at the safety and effectiveness of a new drug forinhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients withEmphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Product Code: Kamada-AAT for inhalation Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2;Phase 3 | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | ||
| 79 | EUCTR2008-005326-36-GB (EUCTR) | 02/11/2009 | 07/08/2009 | A clinical trial looking at the safety and effectiveness of a new drug for inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with Emphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Other descriptive name: ALPHA-1-ANTITRYPSIN | Kamada Limited | NULL | Not Recruiting | Female: yes Male: yes | 200 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | ||
| 80 | EUCTR2007-004869-18-GB (EUCTR) | 03/07/2008 | 04/04/2008 | Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD | Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD | The medical condition is emphysema and will be investigated in subjects with alpha 1-antitrypsin deficiency. MedDRA version: 9.1;Level: LLT;Classification code 10001811;Term: Alpha-1 proteinase inhibitor deficiency MedDRA version: 9.1;Classification code 10014563;Term: Emphysema pulmonary MedDRA version: 9.1;Classification code 10010952;Term: COPD MedDRA version: 9.1;Classification code 10006458;Term: Bronchitis chronic | Trade Name: Prolastin Product Name: Prolastin INN or Proposed INN: alpha 1-proteinase inhibitor (human) Other descriptive name: ALPHA-1-ANTITRYPSIN | UHB NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 26 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): Therapeutic use (Phase 4): | United Kingdom | ||
| 81 | EUCTR2004-001688-23-GB (EUCTR) | 18/02/2008 | 19/10/2005 | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | Product Name: Not applicable Product Code: RO3300074 INN or Proposed INN: None assigned Other descriptive name: Retinoic Acid Receptor gamma Agonist Product Name: Not applicable Product Code: RO3300074 Other descriptive name: Retinoic Acid Receptor gamma Agonist | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 300 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): Therapeutic use (Phase 4): | Spain;Denmark;United Kingdom | ||
| 82 | NCT00460096 (ClinicalTrials.gov) | March 2007 | 12/4/2007 | Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency | Randomized Double-Blind Comparison of an Alpha-1 Proteinase Inhibitor (Kamada API) With a Currently Marketed API Product in Individuals With Alpha-1 Antitrypsin Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: Kamada-API | Kamada, Ltd. | NULL | Completed | 18 Years | N/A | Both | 50 | Phase 2/Phase 3 | United States |
| 83 | NCT00396006 (ClinicalTrials.gov) | October 2006 | 3/11/2006 | Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor) | The Effect of Augmentation Therapy With ARALAST Fraction IV-1 (ARALAST) Alpha1-Proteinase Inhibitor (a1-PI) on the Level of a1-PI and Other Analytes in the Bronchoalveolar (BAL) Epithelial Lining Fluid (ELF) | Alpha 1-Antitrypsin Deficiency | Biological: Alpha1-Proteinase Inhibitor | Baxalta now part of Shire | NULL | Completed | 18 Years | N/A | All | 21 | Phase 4 | Australia;New Zealand |
| 84 | EUCTR2004-001688-23-DK (EUCTR) | 05/07/2006 | 30/05/2006 | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 5 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 5 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | Product Name: Not applicable Product Code: RO3300074 INN or Proposed INN: None assigned Other descriptive name: Retinoic Acid Receptor gamma Agonist | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 300 | United Kingdom;Denmark;Spain | |||
| 85 | NCT00301366 (ClinicalTrials.gov) | June 2006 | 8/3/2006 | The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency | Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: alpha-1 proteinase inhibitor (human) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 38 | Phase 3 | United States;United Kingdom;Netherlands;Poland |
| 86 | NCT00295061 (ClinicalTrials.gov) | May 2006 | 20/2/2006 | Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults | Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency. | Alpha 1-Antitrypsin Deficiency | Drug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 24 | Phase 3 | United States |
| 87 | NCT00430768 (ClinicalTrials.gov) | February 2006 | 31/1/2007 | Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency | Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults | Alpha 1-Antitrypsin Deficiency | Biological: rAAV1-CB-hAAT | University of Massachusetts, Worcester | National Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR) | Completed | 18 Years | N/A | All | 9 | Phase 1 | United States |
| 88 | NCT00242385 (ClinicalTrials.gov) | December 2005 | 19/10/2005 | Pharmacokinetic Study of ARALAST (Human Alpha1- PI) | Single-Dose, Double-Blind, Crossover Study to Evaluate the Pharmacokinetic Comparability of ARALAST Fraction IV-1 Alpha1-Proteinase Inhibitor (ARALAST Fr. IV-1) and ARALAST | Alpha 1-Antitrypsin Deficiency | Biological: Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor;Biological: Dose of 60 mg/kg alpha1-proteinase inhibitor | Baxalta now part of Shire | Baxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia) | Completed | 18 Years | N/A | All | 25 | Phase 1 | Australia;New Zealand |
| 89 | EUCTR2004-001688-23-ES (EUCTR) | 27/10/2004 | 21/10/2004 | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | Product Name: Not applicable Product Code: RO3300074 INN or Proposed INN: None assigned Other descriptive name: Retinoic Acid Receptor gamma Agonist | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 2 | Spain;Denmark;United Kingdom | ||
| 90 | NCT00377416 (ClinicalTrials.gov) | March 2004 | 14/9/2006 | Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency | Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV2-CB-hAAT) Gene Vector to AAT-Deficient Adults | Alpha 1-Antitrypsin Deficiency | Genetic: rAAV2-CB-hAAT Gene Vector | University of Massachusetts, Worcester | National Heart, Lung, and Blood Institute (NHLBI);Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR) | Completed | 18 Years | N/A | All | 12 | Early Phase 1 | United States |
| 91 | NCT00263887 (ClinicalTrials.gov) | December 2003 | 12/9/2005 | Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE) | Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency. | Alpha 1-Antitrypsin Deficiency | Drug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 77 | Phase 2 | Denmark;Sweden;United Kingdom |
| 92 | NCT00067756 (ClinicalTrials.gov) | November 2001 | 26/8/2003 | 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? | 4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals | Alpha 1-Antitrypsin Deficiency | Drug: 4-PBA | University of Florida | Alpha-1 Foundation;Brantly, Mark L., M.D. | Completed | 18 Years | 65 Years | Both | 12 | Phase 2 | United States |
| 93 | 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 |