Clinical trials: Alpha1-Antitrypsin Deficiency
Asthma, Chronic Obstructive Pulmonary Disease
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease
This is a single centre exploratory study that aims to apply hyperpolarized xenon-129 (129Xe) magnetic resonance imaging (MRI) methods and measurements in individual patients with and without lung disease to better understand…
- Conditions
- Asthma, Chronic Obstructive Pulmonary Disease, Bronchiectasis, Emphysema
- Trial name
- NCT03455686
- Testing
- Hyperpolarized 129Xe MRI
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- McMaster University
Post Cardiac Surgery Systemic Inflammatory Response
Single Dose Administration of Alpha-1 Anti-Trypsin for the Amelioration of Organ Injury in Patients Undergoing Cardiac Surgery
Protocol Summary STUDY DESIGN A pilot, prospective, double blind, randomized, placebo controlled study. STUDY POPULATION Patients assigned to elective CABG with cardiopulmonary bypass (CPB) at the Department of Cardiothoracic…
- Trial name
- NCT02191839
- Testing
- Alpha 1-Antitrypsin
- Volunteers
- 20 people
- Sponsor
- Soroka University Medical Center
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
Neoplasms, Antioxidants
Oxidoreductive Balance and Lysosomal Activity in Cancer Patients.
The research aims to determine the parameters of oxidative stress and inflammatory processes and compare these parameters with the image obtained using positron emission tomography (PET) with 2-deoxy-2-[fluorine-18]fluoro…
- Conditions
- Neoplasms, Antioxidants, Oxidative Stress, Lipid Peroxidation
- Trial name
- NCT03470857
- Testing
- Patients
- Where
- 3 sites in Poland
- Volunteers
- 83 people, healthy volunteers welcome
- Sponsor
- Nicolaus Copernicus University
Alpha 1-Antitrypsin Deficiency
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT…
- Trial name
- NCT00377416
- Testing
- rAAV2-CB-hAAT Gene Vector
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- University of Massachusetts, Worcester
Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease
A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM647a, an investigational Prime Editing therapy, in adults and adolescents with Alpha-1 Antitrypsin Deficiency…
- Conditions
- Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease, Alpha-1 Antitrypsin Deficiency (AATD), AATD
- Trial name
- PM647a (NCT07887347)
- Testing
- PM647a
- Volunteers
- 54 people
- Sponsor
- Prime Medicine, Inc.
COPD, Lung Function Decreased
Permian Health Women's Lung Health Study
The goal of this cross-sectional observational study is to estimate the prevalence of lung function impairment as measured by spirometry in a population of Gambian women aged 15 and older.
- Conditions
- COPD, Lung Function Decreased, Alpha 1-Antitrypsin
- Trial name
- WLHS (NCT06029543)
- Where
- The Gambia
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Permian Health Lung Institute
Alpha 1-Antitrypsin Deficiency
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to evaluate the long-term safety and further characterize the pharmacodynamics (PD) of belcesiran in adult patients with PiZZ AATLD.
- Trial name
- NCT05146882
- Testing
- Belcesiran
- Where
- New Zealand
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Alpha 1-Antitrypsin Deficiency, Emphysema
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
- Trial name
- ELEVAATE (NCT05856331)
- Testing
- SAR447537, Zemaira
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 99 people
- Sponsor
- Sanofi
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, 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
Alpha 1-Antitrypsin Deficiency
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver…
- Trial name
- NCT03946449
- Testing
- ARO-AAT
- Where
- 4 sites in Austria, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Arrowhead Pharmaceuticals
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
Chronic Obstructive Pulmonary Disease
Effects of Pulmonary Rehabilitation on Skeletal Muscle in COPD Patients
In a former study, the investigator observed significant differences in the response to pulmonary rehabilitation between COPD patients with the "normal" genetic variant of alpha-1 antitrypsin (PiMM) and those with a homozygous…
- Trial name
- NCT02915614
- Testing
- Pulmonary rehabilitation
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Schön Klinik Berchtesgadener Land
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.
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
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Emphysema, Chronic Obstructive Pulmonary Disease (COPD)
Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant Suitability
The purpose of this research study is to perfect the technique of EVLP and learn about the safety of transplanting lungs that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and perfused…
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis
- Trial name
- NCT01241942
- Testing
- Steen Solution™, Conventional Lung Transplant
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of North Carolina, Chapel Hill
Alpha1-antitrypsin Deficiency
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study
The purpose of the study is 2-fold: (1) to evaluate the safety and potential immunogenicity of GLASSIA following intravenous (IV) administration via in-line filtration; and (2) to assess the effects of GLASSIA augmentation…
- Trial name
- NCT02525861
- Testing
- GLASSIA
- Where
- 18 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Baxalta now part of Shire
Alpha 1-Antitrypsin Deficiency, Emphysema
Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema
The aim of this study is to describe the natural history of patients with alpha-1 antitrypsin associated emphysema and to figure out associated prognostic factors.
- Trial name
- NCT00700934
- Where
- France
- Volunteers
- 300 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris