This study aims to provide long-term follow-up for individuals with alpha-1 antitrypsin (A1AT) deficiency, an inherited condition, who had previously received the gene therapy ADVM-043. No new treatment is given; individuals who had received ADVM-043 and agreed to the assessments in the long-term follow-up protocol were included. This was a phase-not-applicable study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * The subject has A1AT deficiency and has previously received ADVM-043 gene therapy Key Exclusion Criteria: * The subject is unwilling or unable to participate in all required study evaluations in the long-term follow-up protocol * The subject is participating in another investigational treatment protocol * The subject is unable to understand the purpose and risks of the study or cannot provide a signed and dated informed consent form (ICF)