Clinical trials: Alpha-1 Protease Inhibitor Deficiency
Alpha 1-Antitrypsin Deficiency
A Phase 1, First-in-human Study of VX-634
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.
- Trial name
- NCT05579431
- Testing
- VX-634
- Where
- 5 sites in United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alpha 1 Antitrypsin Deficiency
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.
- Trial name
- RepAIR1 (NCT07431112)
- Testing
- AIR-001 SAD dose level 1, AIR-001 SAD dose level 2, AIR-001 SAD dose level 3
- Where
- 13 sites in Australia, Georgia and 7 more
- Volunteers
- 54 people
- Sponsor
- AIRNA Corporation
Alpha-1 Antitrypsin Deficiency, AATD
AATD-Liver Scan Study
The goal of this study is to provide liver stiffness screenings (FibroScans) to patients with Alpha-1 Antitrypsin Deficiency for the following purposes: To evaluate the prevalence of elevated liver stiffness among homozygous and…
- Conditions
- Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 Protease Inhibitor Deficiency, Alpha-1 AT Deficiency
- Trial name
- NCT07898553
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Alpha-1 Foundation
Alpha-1-Antitrypsin Deficiency, Chronic Obstructive Lung Disease
Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency
The objective of this study is to evaluate the safety and effectiveness of the RePneu Lung Volume Reduction Coil (LVRC) in patients with Alpha-1-Antitrypsin deficiency (AATD) caused emphysema.
- Conditions
- Alpha-1-Antitrypsin Deficiency, Chronic Obstructive Lung Disease, Emphysema
- Trial name
- LuReCAA (NCT02273349)
- Testing
- Lung Volume Reduction Coil (PneumrX Inc.)
- Where
- 2 sites in Denmark, Germany
- Sponsor
- University Hospital, Saarland
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Alpha-1 Antitrypsin Deficiency
Long-Term Follow-up Study of ADVM-043
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin…
- Trial name
- NCT03804021
- Testing
- No Intervention
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Adverum Biotechnologies, Inc.
Children With a Deficiency of Alpha-1 Antitrypsin
Polygen Defi-Alpha: Genetic Polymorphisms Study in Children With Alpha-1 Antitrypsin Deficiency, Included in the DEFI-ALPHA Cohort
The deficiency of alpha-1 antitrypsin (DA1AT) is a genetic disorder of variable clinical expression, initially described in adults with pulmonary emphysema patients.
- Trial name
- Polygen (NCT01862211)
- Testing
- blood sampling
- Where
- 20 sites in France
- Volunteers
- 296 people
- Sponsor
- Hospices Civils de Lyon
Alpha 1-Antitrypsin Deficiency
Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency
OBJECTIVES: I. Establish cell lines from patients with alpha 1-antitrypsin deficiency in order to examine genetic traits that predispose to liver injury.
- Trial name
- NCT00005098
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Center for Research Resources (NCRR)