Clinical trials: Alpha1-Proteinase Inhibitor Deficiency
Alpha-1 Antitrypsin Deficiency
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
- Trial name
- NCT05643495
- Testing
- VX-864
- Where
- 13 sites in United States, Germany and 2 more
- Volunteers
- 14 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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 (AATD)
Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.
To define the course of AATD-associated liver disease. To use the obtained samples for biomedical research which includes: 1. Search for serum-based disease biomarkers and the associated molecular pathways. 2.
- Trial name
- NCT07639996
- Testing
- Multi-omic spatial analysis
- Volunteers
- 45 people
- Sponsor
- Assiut University
Diabetes Mellitus, Type 1
A Research Trial of Aralast in New Onset Diabetes (RETAIN)
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to…
- Trial name
- RETAIN (NCT01183468)
- Testing
- Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
- Where
- 15 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Alpha-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
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat…
- Conditions
- Alpha-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 COPD
- Trial name
- ATALANTa (NCT03679598)
- Testing
- Alvelestat (MPH966)
- Where
- 10 sites in United States
- Volunteers
- 63 people
- Sponsor
- University of Alabama at Birmingham
Alpha-1-antitrypsin Deficiency, Emphysema
Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency
Emphysema linked to alpha-1-antitrypsin deficiency (DAAT): towards a better prediction of risks Emphysema caused by alpha-1-antitrypsin deficiency (DAAT) is a rare genetic disorder that can lead to serious complications, such as…
- Conditions
- Alpha-1-antitrypsin Deficiency, Emphysema, Artificial Intelligence (AI), Predictive Learning Models
- Trial name
- FLARE (NCT07715617)
- Where
- 4 sites in France
- Volunteers
- 230 people
- Sponsor
- University Hospital, Bordeaux
Alpha 1 Antitrypsin Deficiency
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
The current treatment of individuals with alpha-1 antitrypsin deficiency (AATD) who develop lung disease (COPD) is the administration of intravenous purified alpha-1 antitrypsin (augmentation therapy) at a fixed dose of 60 mg/kg…
- Trial name
- NCT01669421
- Testing
- Alpha-1 Antitrypsin (human)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Michael Campos, MD
Alpha-1 Antitrypsin Deficiency
The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency
Individuals with a deficiency of alpha-1 antitrypsin (AAT) often develop emphysema. Traditional lung function tests may not be the most accurate way to measure the progression of emphysema.
- Trial name
- QUANTUM-1 (NCT00532805)
- Where
- 7 sites in United States
- Volunteers
- 49 people
- Sponsor
- Medical University of South Carolina
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
Alpha 1-Antitrypsin Deficiency, Emphysema
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema
- Trial name
- ELEVAATE OLE (NCT05897424)
- Testing
- SAR447537
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 185 people
- Sponsor
- Sanofi