U.S., May 31 -- ClinicalTrials.gov registry received information related to the study (NCT06996756) titled 'Gene Therapy for Alpha-1 Antitrypsin Deficiency' on May 21.
Brief Summary: This is a study of gene therapy to treat alpha-1 antitrypsin 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 (AAT) deficient individuals.
Study Start Date: Aug. 01, 2025
Study Type: INTERVENTIONAL
Condition:
Alpha 1-...