NCT06996756

Gene Therapy for Alpha 1- Antitrypsin Deficiency

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	AAV8hAAT(AVL)	AAV8hAAT(AVL) gene transfer vector

Timeline

• Start date: 2025-02-26
• Primary completion: 2028-04-30
• Completion: 2032-08-01
• First posted: 2025-05-30
• Last updated: 2026-03-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study