231. α1−アンチトリプシン欠乏症 Alpha-1-antitrypsin deficiency Clinical trials / Disease details


臨床試験数 : 89 薬物数 : 89 - (DrugBank : 16) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 47

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05146882
(ClinicalTrials.gov)
December 20219/11/2021An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver DiseaseAlpha 1-Antitrypsin DeficiencyDrug: BelcesiranDicerna Pharmaceuticals, Inc.NULLEnrolling by invitation18 Years75 YearsAll54Phase 2New Zealand
2NCT04764448
(ClinicalTrials.gov)
February 12, 202127/1/2021A Study of Belcesiran in Patients With A1ATD-Associated Liver DiseaseA Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: belcesiran;Other: PlaceboDicerna Pharmaceuticals, Inc.NULLRecruiting18 Years70 YearsAll54Phase 2Australia;New Zealand;Spain;United Kingdom
3EUCTR2018-003385-14-AT
(EUCTR)
28/01/202114/09/2020A 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.NULLNot RecruitingFemale: yes
Male: yes
120Phase 2;Phase 3Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
4EUCTR2018-003385-14-DE
(EUCTR)
05/01/202125/05/2020A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2United States;Portugal;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden
5EUCTR2019-004881-16-IE
(EUCTR)
29/10/202009/07/2020Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;Canada;Ireland;Germany;United Kingdom;Sweden
6EUCTR2018-003385-14-GB
(EUCTR)
19/10/202028/06/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 23.0;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10001806 - Alpha-1 anti-trypsin deficiency;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;United Kingdom;Sweden
7EUCTR2019-004881-16-DE
(EUCTR)
17/09/202010/07/2020Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;Canada;Ireland;Germany;United Kingdom;Sweden
8EUCTR2019-004881-16-GB
(EUCTR)
14/09/202021/07/2020Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeA 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 IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 2United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden
9EUCTR2019-004881-16-SE
(EUCTR)
11/09/202014/07/2020Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;Canada;Ireland;Germany;United Kingdom;Sweden
10EUCTR2019-000602-30-FI
(EUCTR)
08/09/202008/05/2020A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
220Phase 3Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden
11EUCTR2019-000602-30-GB
(EUCTR)
02/07/202017/03/2020A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
220Phase 3Belgium;Ireland;Netherlands;United Kingdom;Sweden;Finland
12EUCTR2019-000602-30-SE
(EUCTR)
29/06/202024/04/2020A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
220Phase 3Argentina;Finland;Belgium;Ireland;Netherlands;United Kingdom;Italy;Sweden
13EUCTR2019-003650-92-SE
(EUCTR)
08/05/202009/12/2019Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
43Phase 2United States;Ireland;Netherlands;Germany;United Kingdom;Sweden
14EUCTR2019-002501-22-GB
(EUCTR)
04/05/202030/10/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2United Kingdom
15EUCTR2019-003650-92-IE
(EUCTR)
09/04/202010/03/2020Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
43Phase 2Sweden;United Kingdom;Germany;Netherlands;Ireland;United States
16EUCTR2019-003650-92-GB
(EUCTR)
28/02/202009/12/2019Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
43Phase 2United States;Ireland;Netherlands;Germany;United Kingdom;Sweden
17EUCTR2019-003650-92-DE
(EUCTR)
11/02/202004/12/2019Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZGenotypeA 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 IncorporatedNULLNot RecruitingFemale: yes
Male: yes
43Phase 2United States;Ireland;Netherlands;Germany;United Kingdom;Sweden
18EUCTR2018-003385-14-SE
(EUCTR)
31/01/202025/03/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]A Placebo-Controlled, Multi-dose, Phase 2 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.NULLNot RecruitingFemale: yes
Male: yes
36Phase 2Portugal;United States;Canada;Spain;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden;Ireland
19NCT04167345
(ClinicalTrials.gov)
January 13, 202015/11/2019Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ GenotypeA Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ SubjectsAlpha 1-Antitrypsin DeficiencyDrug: VX-814;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLTerminated18 Years80 YearsAll48Phase 2United States;Canada;Germany;Ireland
20NCT03946449
(ClinicalTrials.gov)
December 19, 20198/5/2019Study 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 DeficiencyDrug: ARO-AAT InjectionArrowhead PharmaceuticalsNULLActive, not recruiting18 Years75 YearsAll16Phase 2Austria;Germany;United Kingdom
21NCT04204252
(ClinicalTrials.gov)
November 25, 201917/12/2019Evaluate Efficacy and Safety of Kamada-AAT for Inhalation in Patients With AATDA 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 DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: Alpha 1-Antitrypsin;Drug: PlacebosKamada, Ltd.Syneos HealthRecruiting18 Years65 YearsAll220Phase 3Netherlands
22EUCTR2019-000068-86-AT
(EUCTR)
14/11/201912/06/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom
23EUCTR2018-003385-14-NL
(EUCTR)
07/11/201924/07/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Placebo-Controlled, Multi-dose, Phase 2 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2United States;Portugal;Canada;Spain;Ireland;Austria;Germany;Netherlands;United Kingdom;Italy;Sweden
24EUCTR2018-003385-14-PT
(EUCTR)
04/11/201909/07/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Placebo-Controlled, Multi-dose, Phase 2 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2United States;Portugal;Canada;Spain;Poland;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden
25NCT04174118
(ClinicalTrials.gov)
October 24, 20196/11/2019Study of DCR-A1AT in Healthy Adult VolunteersA Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered Belcesiran in Healthy Adult VolunteersAlpha 1-Antitrypsin DeficiencyDrug: belcesiran;Drug: PlaceboDicerna Pharmaceuticals, Inc.NULLRecruiting18 Years55 YearsAll36Phase 1New Zealand;Sweden
26EUCTR2019-000602-30-NL
(EUCTR)
30/09/201915/05/2019A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
220Phase 3Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden
27EUCTR2018-003385-14-ES
(EUCTR)
11/09/201911/06/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 20.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Poland;Spain;Ireland;Netherlands;Italy;Sweden
28EUCTR2018-001309-95-BE
(EUCTR)
06/09/201902/07/2019A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiencyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
182Phase 2United States;Canada;Spain;Poland;Belgium;Denmark;United Kingdom;Sweden
29EUCTR2018-003385-14-IT
(EUCTR)
05/09/201919/01/2021A 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 PHARMACEUTICALSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
118Phase 2Portugal;United States;Spain;Poland;Ireland;Germany;Netherlands;United Kingdom;Italy;Sweden
30NCT03945292
(ClinicalTrials.gov)
August 7, 20198/5/2019Safety, Tolerability and Pharmacodynamic Effect of ARO-AATA 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 DeficiencyDrug: ARO-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsNULLActive, not recruiting18 Years75 YearsAll40Phase 2United States;Germany;Italy;Netherlands;Portugal;Spain;Sweden;Canada;Ireland
31EUCTR2018-003385-14-IE
(EUCTR)
06/08/201921/03/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 23.0;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10001806 - Alpha-1 anti-trypsin deficiency;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
120Phase 2;Phase 3United States;Portugal;Canada;Spain;Poland;Ireland;Germany;Netherlands;United Kingdom;Italy;Sweden
32NCT03815396
(ClinicalTrials.gov)
July 19, 201921/1/2019Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin DeficiencyAn 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;AATDDrug: INBRX-101/rhAAT-FcInhibrx, Inc.NULLRecruiting18 Years80 YearsAll30Phase 1United States;New Zealand;United Kingdom
33EUCTR2019-000068-86-GB
(EUCTR)
01/07/201903/04/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom
34NCT03679598
(ClinicalTrials.gov)
April 8, 201918/9/2018Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin DeficiencyA First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan DiseaseAlpha-1 Antitrypsin Deficiency (AATD);Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations;Emphysema or COPDDrug: Alvelestat (MPH966);Other: PlaceboUniversity of Alabama at BirminghamNational Institutes of Health (NIH);Mereo BioPharmaRecruiting18 Years80 YearsAll66Phase 2United States
35EUCTR2018-001309-95-PL
(EUCTR)
05/04/201910/12/2018A 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 deficiencyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
182Phase 2United States;Canada;Spain;Belgium;Poland;Denmark;United Kingdom;Sweden
36NCT03767829
(ClinicalTrials.gov)
December 5, 20185/12/2018A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseA 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 DiseaseZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseDrug: ALN-AAT02;Drug: PlaceboAlnylam PharmaceuticalsNULLTerminated18 Years65 YearsAll32Phase 1/Phase 2United Kingdom
37EUCTR2018-001362-41-GB
(EUCTR)
03/12/201820/06/2019A 2-Part Safety and Tolerability Study of ALN-AAT02 in Healthy Volunteers and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseA 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.NULLNot RecruitingFemale: yes
Male: yes
96Phase 1;Phase 2United States;Germany;United Kingdom
38EUCTR2018-001309-95-GB
(EUCTR)
30/11/201831/07/2018A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiencyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
182Phase 2United States;Canada;Poland;Belgium;Spain;Denmark;United Kingdom;Sweden
39EUCTR2018-001309-95-DK
(EUCTR)
14/11/201813/09/2018A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiencyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
182Phase 2United States;Canada;Belgium;Spain;Poland;Denmark;United Kingdom;Sweden
40NCT03636347
(ClinicalTrials.gov)
October 29, 201820/6/2018A 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;COPDDrug: Placebo Oral Tablet;Drug: Alvelestat oral tablet - dose 1;Drug: Alvelestat oral tablet - dose 2Mereo BioPharmaSyneos HealthActive, not recruiting18 Years75 YearsAll165Phase 2United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom
41EUCTR2018-001309-95-SE
(EUCTR)
16/10/201806/09/2018A Phase 2 placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of alvelestat (MPH966) in participants with alpha-1 antitrypsin deficiencyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
182Phase 2United States;Canada;Spain;Poland;Belgium;Denmark;United Kingdom;Sweden
42NCT03385395
(ClinicalTrials.gov)
July 20181/12/2017Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin DeficiencyA 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 DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: OctaAlpha1;Drug: GlassiaOctapharmaNULLWithdrawn18 YearsN/AAll0Phase 2NULL
43EUCTR2015-004110-23-PL
(EUCTR)
19/04/201816/02/2018A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin DeficiencyAn 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 LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;Estonia;Canada;Poland;Denmark;Australia;Russian Federation;New Zealand;Sweden
44NCT03362242
(ClinicalTrials.gov)
March 12, 201830/11/2017Study of ARO-AAT in Normal Adult VolunteersA Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult VolunteersAlpha 1-Antitrypsin DeficiencyDrug: ARO-AAT Injection;Other: Sterile Normal Saline (0.9% NaCl)Arrowhead PharmaceuticalsNULLCompleted18 Years55 YearsAll45Phase 1New Zealand
45NCT03172455
(ClinicalTrials.gov)
May 1, 201729/5/2017Early 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 TransplantDrug: GlassiaImpatients N.V. trading as myTomorrowsKamada, Ltd.No longer availableN/AN/AAllNULL
46NCT03114020
(ClinicalTrials.gov)
March 22, 20175/4/2017Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin DeficiencyPhase 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 EmphysemaEmphysema;Alpha 1-Antitrypsin DeficiencyDrug: Hyaluronic Acid Inhalation Solution;Drug: Placebo Inhalation SolutionGerard TurinoNULLCompleted18 Years80 YearsAll27Phase 2United States;Canada
47NCT03008915
(ClinicalTrials.gov)
January 201722/12/2016Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin DeficiencyTargeting Pulmonary Perfusion in Alpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency;Emphysema;Chronic Obstructive Pulmonary DiseaseDrug: Aspirin;Drug: Placebo;Other: Withdrawal from alpha1 antitrypsin replacement therapyColumbia UniversityAlpha-1 Foundation;Stony Wold-Herbert Fund, Inc.Active, not recruiting40 YearsN/AAll15Phase 2United States
48NCT02900183
(ClinicalTrials.gov)
October 20169/9/2016Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin LevelsAn 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 DeficiencyDrug: ARC-AAT InjectionArrowhead PharmaceuticalsNULLWithdrawn18 Years75 YearsAll0Phase 2Canada;Ireland;Italy;Sweden
49EUCTR2016-000917-59-SE
(EUCTR)
04/08/201608/06/2016A 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 IncNULLNot RecruitingFemale: yes
Male: yes
12Phase 2Canada;Ireland;Sweden
50NCT02796937
(ClinicalTrials.gov)
July 20163/6/2016Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin DeficiencyAn 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 DeficiencyPulmonary Emphysema in Alpha-1 Antitrypsin DeficiencyBiological: Alpha-1 MPGrifols Therapeutics LLCNULLEnrolling by invitation20 Years72 YearsAll250Phase 3United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden
51EUCTR2016-000917-59-IE
(EUCTR)
29/06/201613/04/2016A 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, IncNULLNot RecruitingFemale: yes
Male: yes
12Phase 2Canada;Ireland;Sweden
52EUCTR2015-004110-23-DK
(EUCTR)
29/03/201629/01/2016A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin DeficiencyAn 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 LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;Estonia;Spain;Russian Federation;France;Canada;Argentina;Brazil;Poland;Romania;Denmark;Australia;Germany;New Zealand;Sweden
53EUCTR2013-001870-38-DE
(EUCTR)
21/09/201527/03/2015A 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 DeficiencyA 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.NULLNot RecruitingFemale: yes
Male: yes
339Phase 3United States;Estonia;Finland;Spain;Ireland;Russian Federation;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Sweden
54EUCTR2015-001297-18-GB
(EUCTR)
29/06/201515/05/2015The First-in-Human Study of an Investigational Drug, ALN-AAT, in Healthy Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver DiseaseA 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.NULLNot 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): noUnited States;United Kingdom
55NCT02363946
(ClinicalTrials.gov)
February 20152/2/2015A 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 DeficiencyDrug: ARC-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsNULLTerminated18 Years70 YearsAll65Phase 1Australia;Germany;Netherlands;United Kingdom
56EUCTR2013-001870-38-PL
(EUCTR)
30/10/201429/08/2014A 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 DeficiencyA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
339United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden
57NCT02001688
(ClinicalTrials.gov)
April 201424/11/2013Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for InhalationPhase 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 SubjectsAlpha-1 Antitrypsin DeficiencyDrug: Kamada-AAT for Inhalation, 80mg;Drug: Placebo;Drug: Kamada-AAT for Inhalation, 160mgKamada, Ltd.NULLCompleted18 Years65 YearsAll36Phase 2United States
58EUCTR2013-001870-38-IE
(EUCTR)
21/03/201427/09/2013A 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 DeficiencyA 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.NULLNot RecruitingFemale: yes
Male: yes
339Phase 3Serbia;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
59EUCTR2013-001870-38-FI
(EUCTR)
30/01/201430/12/2013A 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 DeficiencyA 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 LLCNULLAuthorised-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): noSerbia;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
60EUCTR2013-001870-38-SE
(EUCTR)
09/01/201423/09/2013A 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 DeficiencyA 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 LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
339Phase 3United States;Serbia;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Brazil;Poland;Romania;Australia;Denmark;Germany;Moldova, Republic of;New Zealand;Sweden
61EUCTR2013-001870-38-ES
(EUCTR)
26/12/201318/10/2013A 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 DeficiencyA 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.NULLNot 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): noUnited 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
62EUCTR2013-001870-38-DK
(EUCTR)
20/11/201304/10/2013A 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 DeficiencyA 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 LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
339Phase 3Serbia;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
63EUCTR2013-001870-38-EE
(EUCTR)
12/11/201301/10/2013A 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 DeficiencyA 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 LLCNULLAuthorised-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): noFinland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Moldova, Republic of;Sweden;Estonia;United States;Serbia
64NCT01810458
(ClinicalTrials.gov)
October 20136/3/2013Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin DeficiencyLiver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATDDevice: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: OndansetronUniversity of FloridaNULLCompleted18 Years70 YearsAll109United States
65NCT01669421
(ClinicalTrials.gov)
July 201214/8/2012Effect 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 DeficiencyDrug: Alpha-1 Antitrypsin (human)Michael Campos, MDCSL BehringCompleted18 Years75 YearsAll10Phase 2United States
66NCT01379469
(ClinicalTrials.gov)
January 201215/6/2011Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin DeficiencyA Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin DeficiencyAlpha-1-antitrypsin Deficiency;Liver CirrhosisDrug: Drug-Carbamazepine (Tegretol XR);Drug: Carbamazepine (Tegretol XR) PlaceboWashington University School of MedicineNovartis;National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of PittsburghTerminated14 Years80 YearsAll20Phase 2United States
67EUCTR2008-005326-36-SE
(EUCTR)
06/10/201111/09/2009A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
200Phase 2/3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
68EUCTR2009-014286-20-IE
(EUCTR)
21/12/201031/05/2010A 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 DeficiencyA 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 CorporationNULLNot RecruitingFemale: yes
Male: yes
9Phase 2Ireland
69NCT01213043
(ClinicalTrials.gov)
November 201029/9/2010Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin DeficiencyA 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 DeficiencyEmphysema;Alpha 1-antitrypsin Deficiency (AATD)Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kgGrifols Therapeutics Inc.NULLCompleted18 Years70 YearsAll30Phase 2United States
70EUCTR2008-005326-36-DE
(EUCTR)
01/10/201028/04/2010A 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 EmphysemaA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
200Phase 2/3Ireland;Denmark;Netherlands;Germany;United Kingdom;Sweden
71EUCTR2008-005326-36-IE
(EUCTR)
09/07/201012/05/2010A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
200Phase 2/3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
72NCT01054339
(ClinicalTrials.gov)
June 201021/1/2010Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin DeficiencyA 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 DeficiencyAlpha-1 Antitrypsin DeficiencyDrug: rAAV1-CB-hAATApplied Genetic Technologies CorpNational Heart, Lung, and Blood Institute (NHLBI)Completed18 Years75 YearsAll9Phase 2United States;Ireland
73EUCTR2008-005326-36-NL
(EUCTR)
05/02/201021/08/2009A 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 LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 2/3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
74EUCTR2008-005326-36-DK
(EUCTR)
13/01/201014/09/2009A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
200Phase 2;Phase 3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
75EUCTR2008-005326-36-GB
(EUCTR)
02/11/200907/08/2009 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 LimitedNULLNot 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): noIreland;Denmark;Germany;Netherlands;United Kingdom;Sweden
76EUCTR2007-004869-18-GB
(EUCTR)
03/07/200804/04/2008Evaluation 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 AATDEvaluation 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 TrustNULLNot 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
77EUCTR2004-001688-23-GB
(EUCTR)
18/02/200819/10/2005A 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 deficiencyA 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 deficiencyProduct 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 LtdNULLNot 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
78NCT01851642
(ClinicalTrials.gov)
September 200725/4/2013Lung Disease and Its Affect on the Work of White Blood Cells in the LungsThe Role of Conformational Diseases on Macrophage FunctionAlpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD;Cystic Fibrosis (CF)Procedure: History and physical exam.;Procedure: Blood draw.;Procedure: Pulmonary function testing.;Drug: Albuterol inhaler.University of FloridaNULLRecruiting18 YearsN/AAll220United States
79NCT00460096
(ClinicalTrials.gov)
March 200712/4/2007Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin DeficiencyRandomized Double-Blind Comparison of an Alpha-1 Proteinase Inhibitor (Kamada API) With a Currently Marketed API Product in Individuals With Alpha-1 Antitrypsin DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: Kamada-APIKamada, Ltd.NULLCompleted18 YearsN/ABoth50Phase 2/Phase 3United States
80NCT00396006
(ClinicalTrials.gov)
October 20063/11/2006Efficacy 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 DeficiencyBiological: Alpha1-Proteinase InhibitorBaxalta now part of ShireNULLCompleted18 YearsN/AAll21Phase 4Australia;New Zealand
81EUCTR2004-001688-23-DK
(EUCTR)
05/07/200630/05/2006A 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 deficiencyA 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 deficiencyProduct Name: Not applicable
Product Code: RO3300074
INN or Proposed INN: None assigned
Other descriptive name: Retinoic Acid Receptor gamma Agonist
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
300United Kingdom;Denmark;Spain
82NCT00301366
(ClinicalTrials.gov)
June 20068/3/2006The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) DeficiencyMulti-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: alpha-1 proteinase inhibitor (human)Grifols Therapeutics Inc.NULLCompleted18 YearsN/AAll38Phase 3United States;United Kingdom;Netherlands;Poland
83NCT00295061
(ClinicalTrials.gov)
May 200620/2/2006Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient AdultsMulti-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 DeficiencyDrug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human)Grifols Therapeutics Inc.NULLCompleted18 YearsN/AAll24Phase 3United States
84NCT00430768
(ClinicalTrials.gov)
February 200631/1/2007Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) DeficiencyPreclinical & 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 AdultsAlpha 1-Antitrypsin DeficiencyBiological: rAAV1-CB-hAATUniversity of Massachusetts, WorcesterNational Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR)Completed18 YearsN/AAll9Phase 1United States
85NCT00242385
(ClinicalTrials.gov)
December 200519/10/2005Pharmacokinetic 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 ARALASTAlpha 1-Antitrypsin DeficiencyBiological: Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor;Biological: Dose of 60 mg/kg alpha1-proteinase inhibitorBaxalta now part of ShireBaxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia)Completed18 YearsN/AAll25Phase 1Australia;New Zealand
86EUCTR2004-001688-23-ES
(EUCTR)
27/10/200421/10/2004A 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 deficiencyA 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 deficiencyProduct Name: Not applicable
Product Code: RO3300074
INN or Proposed INN: None assigned
Other descriptive name: Retinoic Acid Receptor gamma Agonist
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
300United Kingdom;Denmark;Spain
87NCT00263887
(ClinicalTrials.gov)
December 200312/9/2005Alpha-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 DeficiencyDrug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)Grifols Therapeutics Inc.NULLCompleted18 YearsN/AAll77Phase 2Denmark;Sweden;United Kingdom
88NCT00067756
(ClinicalTrials.gov)
November 200126/8/20034-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 IndividualsAlpha 1-Antitrypsin DeficiencyDrug: 4-PBAUniversity of FloridaAlpha-1 Foundation;Brantly, Mark L., M.D.Completed18 Years65 YearsBoth12Phase 2United States
89EUCTR2019-000068-86-DE
(EUCTR)
04/06/2019A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) alpha-1 antitrypsin deficiency (AATD)-associated liver disease
MedDRA version: 23.1;Level: LLT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARO-AAT Injection
Product Code: ARO-AAT
INN or Proposed INN: ADS-001
Arrowhead Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Austria;Germany;United Kingdom