Clinical Trial

Gene Therapy for Alpha 1- Antitrypsin Deficiency

Recruiting Phase 1
View on ClinicalTrials.gov →
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.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2025-05-21; most recent amendment 2026-03-11.
Status change: Not Yet Recruiting → Recruiting 2025-09-22
Trial Details
NCT Number NCT06996756
Lead Sponsor Weill Medical College of Cornell University
Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
Conditions Alpha 1-Antitrypsin Deficiency
Enrollment 16 participants
Start Date 2025-02-26
Primary Completion 2028-04-30 (estimated)
Study Completion 2032-08-01 (estimated)
Updated on ClinicalTrials.gov 2026-03-13