Clinical trials: Alpha 1-Antitrypsin Deficiency
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)
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
Chronic Obstructive Pulmonary Disease, Acute Exacerbation Copd
COPD Exacerbation Blood and Urine Biomarkers Study
This will be a prospective study examining serum levels of MMP-13 and alpha-1 antitrypsin as well as other biomarkers as well as urine biomarkers of smoking status and collagen degradation in the COPD patient population.
- Conditions
- Chronic Obstructive Pulmonary Disease, Acute Exacerbation Copd, Alpha-1 Anti-trypsin Deficiency
- Trial name
- NCT03823443
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Columbia University
Alpha 1-Antitrypsin Deficiency
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency
The objective of this study is to prospectively assess whether there is any interval between first symptom and initial diagnosis that is experienced by patients with newly diagnosed alpha-1 antitrypsin deficiency (AATD) and then…
- Trial name
- NCT03039335
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Biocerna LLC
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
Lung Diseases, Emphysema
Alpha1-antitrypsin Deficiency Registry
To collect data from the 37 participating clinical centers on patients with alpha1-antitrypsin deficiency, including those who received replacement therapy with an intravenous preparation of alpha1-proteinase inhibitor (A1Pi)…
- Conditions
- Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT00005292
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Obstructive Pulmonary Disease, Alpha-1-antitrypsin-deficiency
Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients
Chronic Obstructive Pulmonary Disease (COPD)-patients (caused by smoking-level) and Alpha-1-Antitrypsin-deficiency patients showed different developments during rehabilitation in regard to improvement of 6-minute-walking…
- Trial name
- NCT01380626
- Testing
- exercise training
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Schön Klinik Berchtesgadener Land
Alpha-1 Anti-trypsin Deficiency, AATD
A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110
The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with…
- Trial name
- REWRITE (NCT06677307)
- Testing
- KRRO-110
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Korro Bio, Inc.
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
Emphysema, Lung Diseases, Obstructive
Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals
Alpha 1-antitrypsin-deficient individuals develop severe destructive lung disease much earlier and their lung function declines faster than the general population of individuals with chronic obstructive lung disease.
- Conditions
- Emphysema, Lung Diseases, Obstructive, Alpha 1-Antitrypsin Deficiency
- Trial name
- NCT00001462
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Alpha 1-Antitrypsin Deficiency
Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency
The primary purpose of this Phase II/III study is to demonstrate that Kamada-API, a new API concentrate manufactured by Kamada Ltd., is comparable to a currently marketed API product.
- Trial name
- Kamada API (NCT00460096)
- Testing
- Kamada-API
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Kamada, Ltd.
Alpha-1 Antitrypsin Deficiency, AAT Deficiency
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.
- Conditions
- Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD, Cystic Fibrosis (CF)
- Trial name
- NCT01851642
- Testing
- History and physical exam., Blood draw., Pulmonary function testing.
- Where
- United States
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- University of Florida
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
Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency
This randomized trial is being conducted to show non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state.
- Trial name
- NCT03385395
- Testing
- OctaAlpha1, Glassia
- Sponsor
- Octapharma
Alpha1-Antitrypsin Deficiency
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran…
- Trial name
- NCT05677971
- Testing
- Fazirsiran Injection
- Where
- 89 sites in United States, Australia and 17 more
- Volunteers
- 160 people
- Sponsor
- Takeda
Alpha1 Anti-Trypsin Deficiency
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects
This study is composed of two parts. Part A: will test single doses of ZF874 in a double-blind, randomised, placebo-controlled and dose-escalating design (except Group 7, which will be open-label and without placebo).
- Trial name
- NCT04443192
- Testing
- ZF874
- Where
- 5 sites in United Kingdom
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Z Factor Limited
Emphysema
International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema
This is a randomised , placebo controlled, double blind , multicentre, Phase II/III study evaluating the safety and efficacy of Kamada AAT for inhalation in patients with Emphysema caused by Alpha-1 Antitrypsin (AAT) deficiency.
- Trial name
- NCT01217671
- Testing
- Kamada AAT for inhalation
- Where
- 12 sites in Canada, Denmark and 5 more
- Volunteers
- 168 people
- Sponsor
- Kamada, Ltd.
Emphysema, Chronic Obstructive Pulmonary Disease (COPD)
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability
The purpose of this research study is to learn about the safety of transplanting lungs obtained from non-heart-beating donors (NHBDs) that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and…
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis
- Trial name
- NCT01615484
- Testing
- Transplantation of lungs obtained from Non-Heart-Beating Donors (NHBDs) after ex-vivo perfusion w/ STEEN Solution™, STEEN Solution™
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- University of North Carolina, Chapel Hill
Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD)
Alpha-1 antitrypsin deficiency (AATD) is considered a rare genetic cause of chronic obstructive pulmonary disease (COPD) and liver disease.
- Trial name
- PFT (NCT01419158)
- Where
- 19 sites in United States, Puerto Rico
- Volunteers
- 3,457 people
- Sponsor
- Alpha-1 Foundation