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Gene Therapy for Alpha 1- Antitrypsin Deficiency | Clinical Trials | Clareo Health

RECRUITINGPHASE1

Gene Therapy for Alpha 1- Antitrypsin Deficiency

NCT06996756•Weill Medical College of Cornell University

Summary

This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) 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 a persistent basis. We hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin deficient individuals.

Eligibility

Age

18 - 70 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM
•Emphysema as assessed by chest high resolution computational tomography (HRCT)
•Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema.
•Troponin T within normal limits
•Normal liver ultrasound and serum alpha fetoprotein
•Normal kidney function
•No contraindications to receiving corticosteroid immunosuppression

Exclusion Criteria

•Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions.
•Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes)
•Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus
•Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder
•Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin)
•Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)
•Abnormal ECG at screening with findings consistent with cardiac disease
•Females who are currently pregnant or lactating
•Any history of allergies to drugs used for bronchoscopy, including xylocaine, lidocaine, versed, valium, atropine, pilocarpine, isoproterenol, terbutaline, aminophylline, or any local anesthetic
•Individuals receiving experimental medications or participating in another experimental protocol for at least 3 months prior to entry to the study
+5 more criteria

Locations (1)

WCMC Department of Genetic Medicine

New York, New York, United States

Key Dates

Start Date

February 26, 2025

Primary Completion Date

April 30, 2028

Completion Date

August 1, 2032

Last Updated

March 13, 2026

Enrollment

ESTIMATED participants

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

AAV8hAAT(AVL)

BIOLOGICAL

Contacts

Niamh Savage

CONTACT

646-962-5527 nis2049@med.cornell.edu

Sandra Hyde

CONTACT

646-962-2672 sah2003@med.cornell.edu

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RECRUITING

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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Gene Therapy for Alpha 1- Antitrypsin Deficiency

Inclusion Criteria

Exclusion Criteria

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

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

Alpha-1 Research Registry

Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency

Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease

Alpha-1 Foundation Research Registry