RecruitingPhase 1NCT06996756

Gene Therapy for Alpha 1- Antitrypsin Deficiency

Sponsor

Weill Medical College of Cornell University

Enrollment

16 participants

Start Date

Feb 26, 2025

Study Type

INTERVENTIONAL

Conditions

Alpha 1-Antitrypsin Deficiency

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

Min Age: 18 YearsMax Age: 70 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called AAV8hAAT(AVL) for people with alpha 1-antitrypsin deficiency. The study is currently recruiting participants at 1 location. People eligible for this study include aged 18 Years to 70 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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