Condition: Alpha 1-Antitrypsin Deficiency · Sponsor: Weill Medical College of Cornell University
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.
This description comes directly from the study's public registry record.
Niamh Savage · 646-962-5527 · nis2049@med.cornell.edu
Sandra Hyde · 646-962-2672 · sah2003@med.cornell.edu
Always discuss trial participation with your own doctor first.
| WCMC Department of Genetic Medicine | New York, New York, United States | Recruiting |
Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.
We email about this public record only. Unsubscribe anytime with one click. Never medical advice.
This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT06996756