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

  
45 trials found
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1NCT05579431
(ClinicalTrials.gov)
October 13, 202210/10/2022A Phase 1, First-in-human Study of VX-634A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634Alpha 1-Antitrypsin DeficiencyDrug: VX-634;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLRecruiting18 Years55 YearsAll114Phase 1United States
2NCT04764448
(ClinicalTrials.gov)
February 12, 202127/1/2021A Study of Belcesiran in Patients With AATLDA 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 DiseaseAlpha 1-Antitrypsin DeficiencyDrug: Belcesiran;Other: PlaceboDicerna Pharmaceuticals, Inc.NULLRecruiting18 Years75 YearsAll46Phase 2Australia;Austria;Belgium;Canada;France;Germany;Ireland;Netherlands;New Zealand;Portugal;Spain;Sweden;United Kingdom
3EUCTR2019-000602-30-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
4NCT04167345
(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
5NCT03946449
(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
6NCT04174118
(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.NULLActive, not recruiting18 Years55 YearsAll30Phase 1New Zealand;Sweden
7EUCTR2019-000602-30-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
8NCT03945292
(ClinicalTrials.gov)
August 7, 20198/5/2019Safety, 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 DeficiencyDrug: Fazisiran Injection;Other: PlaceboArrowhead PharmaceuticalsNULLActive, not recruiting18 Years75 YearsAll40Phase 2United States;Germany;Italy;Netherlands;Portugal;Spain;Canada;Ireland;Sweden
9NCT03636347
(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 HealthCompleted18 Years75 YearsAll99Phase 2United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom
10NCT03385395
(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
11EUCTR2015-004110-23-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
12NCT03362242
(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
13NCT02796937
(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
14EUCTR2015-004110-23-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
15EUCTR2013-001870-38-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
16EUCTR2013-001870-38-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
17NCT02001688
(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
18EUCTR2013-001870-38-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
19EUCTR2013-001870-38-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
20EUCTR2013-001870-38-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
21EUCTR2013-001870-38-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
22EUCTR2013-001870-38-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
23EUCTR2013-001870-38-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): 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
24NCT01669421
(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
25NCT01379469
(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
26EUCTR2008-005326-36-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
27NCT01213043
(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
28EUCTR2008-005326-36-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
29EUCTR2008-005326-36-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
30EUCTR2008-005326-36-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 LimitedNULLNot RecruitingFemale: yes
Male: yes
200Phase 2;Phase 3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
31EUCTR2008-005326-36-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
32EUCTR2008-005326-36-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
33EUCTR2007-004869-18-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
34EUCTR2004-001688-23-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
35NCT00460096
(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
36NCT00396006
(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
37EUCTR2004-001688-23-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
38NCT00301366
(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
39NCT00295061
(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
40NCT00430768
(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
41NCT00242385
(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
42EUCTR2004-001688-23-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 LtdNULLNot RecruitingFemale: yes
Male: yes
300Phase 2Spain;Denmark;United Kingdom
43NCT00377416
(ClinicalTrials.gov)
March 200414/9/2006Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin DeficiencyPreclinical & 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 AdultsAlpha 1-Antitrypsin DeficiencyGenetic: rAAV2-CB-hAAT Gene VectorUniversity of Massachusetts, WorcesterNational Heart, Lung, and Blood Institute (NHLBI);Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR)Completed18 YearsN/AAll12Early Phase 1United States
44NCT00263887
(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
45NCT00067756
(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