Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT06996756
PHASE1

Gene Therapy for Alpha 1- Antitrypsin Deficiency

Sponsor: Weill Medical College of Cornell University

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.

Key Details

Gender

All

Age Range

18 Years - 70 Years

Study Type

INTERVENTIONAL

Enrollment

16

Start Date

2025-02-26

Completion Date

2032-08-01

Last Updated

2026-03-13

Healthy Volunteers

No

Interventions

BIOLOGICAL

AAV8hAAT(AVL)

AAV8hAAT(AVL) gene transfer vector

Locations (1)

WCMC Department of Genetic Medicine

New York, New York, United States