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alpha-1-antitrypsin-deficiency Clinical Trials

A listing of alpha-1-antitrypsin-deficiency medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 13 clinical trials
A Ann M. Collins

Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"

The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg/day can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD). The main question it aims to answer is: …

18 - 65 years of age All Phase 3

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).

18 - 70 years of age All Phase 1/2
S Sandra Hyde

Gene Therapy for Alpha-1 Antitrypsin Deficiency

This is a study of gene therapy to treat alpha-1 antitrypsin deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on …

18 - 70 years of age All Phase 1
D David LaFon, MD

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function and respiratory infections. To investigate this hypothesis, we will compare immune …

19 years of age All Phase 4

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with …

18 - 70 years of age All Phase 1
S Semi Yoon

PiMZ Longitudinal Cohort (PiMZ Logic)

Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. Presentations are variable in the heterozygous population, the most predominant genotype being PiMZ. The purpose of this study in PiMZ heterozygous patients is to examine the density of the lung as measured by chest computed tomography …

18 years of age All Phase N/A
S Sarah Svenningsen, PhD

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 lung structure and function and evaluate response to therapy delivered as a part of clinical care.

18 - 85 years of age All Phase N/A
o ozlem sengoren dikis, associate prof.

Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction

Brief Summary: This study aims to find out if a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD) is more common in people who have shortness of breath and signs of airway obstruction on their breathing tests. Alpha-1 antitrypsin (AAT) is a protein that protects the lungs from damage. AATD is …

18 years of age All Phase N/A
A Alison Keaveny, MBBS

Alpha-1 Research Registry

The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1.

years of age All Phase N/A
J Jesse West

The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study

The Alpha-1 Coded Testing (ACT) Study was established to study genetic testing and outcomes of individuals at risk for alpha-1 antitrypsin deficiency.

years of age All Phase N/A

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