231. Alpha-1-antitrypsin deficiency
83 clinical trials,   89 drugs   (DrugBank: 16 drugs),   35 drug target genes,   46 drug target pathways

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
18 trials found
No.TrialIDDate_
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agemin
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PhaseCountries
1NCT04167345
(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
2NCT03946449
(ClinicalTrials.gov)
December 19, 20198/5/2019Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AATA Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)Alpha 1-Antitrypsin DeficiencyDrug: ARO-AAT InjectionArrowhead PharmaceuticalsNULLRecruiting18 Years75 YearsAll12Phase 2Austria;Germany;United Kingdom
3NCT04204252
(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
4NCT04174118
(ClinicalTrials.gov)
October 24, 20196/11/2019A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver DiseaseA Phase 1/2 Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered DCR-A1AT in Healthy Adult Volunteers and Patients With Alpha1-Antitrypsin-Deficiency-Associated Liver DiseaseAlpha 1-Antitrypsin DeficiencyDrug: DCR-A1AT;Drug: PlaceboDicerna Pharmaceuticals, Inc.NULLRecruiting18 Years70 YearsAll60Phase 1;Phase 2Sweden
5NCT03945292
(ClinicalTrials.gov)
August 7, 20198/5/2019Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AATA 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 DeficiencyDrug: ARO-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsNULLRecruiting18 Years75 YearsAll120Phase 2;Phase 3United States;Canada;Ireland;Italy;Netherlands;Portugal;Spain;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03636347
(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 HealthRecruiting18 Years75 YearsAll165Phase 2United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom
7NCT03385395
(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
8NCT03362242
(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
9NCT01669421
(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
10EUCTR2007-004869-18-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
UHB NHS Foundation TrustNULLNot Recruiting Female: yes
Male: yes
26Phase 2United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT00460096
(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
12NCT00396006
(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
13NCT00301366
(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
14NCT00295061
(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
15NCT00430768
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00242385
(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
17NCT00263887
(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
18NCT00067756
(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