Long-Term Follow-up Study of ADVM-043
Completed
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Registration Number
- NCT03804021
- Lead Sponsor
- Adverum Biotechnologies, Inc.
- Brief Summary
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
- Detailed Description
ADVM-043-03 was a multi-center, observational study without administration of the ADVM-043 investigational product. Subjects who participated in the ADVANCE study were enrolled into this LTFU study for continued safety monitoring for a 2-year period.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 6
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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686) All adverse events of special interest and all serious adverse events related to ADVM-043
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (2)
University of Florida
🇺🇸Gainesville, Florida, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Florida🇺🇸Gainesville, Florida, United States