Long-Term Follow-up Study of ADVM-043

Plain-language summary

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.

Trial details

Phase

N/A

Status

Completed

Sponsor

Adverum Biotechnologies, Inc.

Intervention

No Intervention

Start date

12/27/2018

Estimated completion

12/6/2021

Countries

United States

Related genes

Locations

• Gainesville, United States
• Charleston, United States

Eligibility criteria

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)