Gene Therapy for Alpha 1- Antitrypsin Deficiency
Sponsor: Weill Medical College of Cornell University • ClinicalTrials.gov ID: NCT06996756
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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.
Interventions
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AAV8hAAT(AVL)
(Biological)
AAV8hAAT(AVL) gene transfer vector
Eligibility criteria
Conditions
Locations
- WCMC Department of Genetic Medicine — New York, New York, United States
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Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
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