Brief Title
Long-Term Follow-up Study of ADVM-043
Official Title
Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency
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.
Study Type
Observational
Primary Outcome
Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043
Condition
Alpha-1 Antitrypsin Deficiency
Intervention
ADVM-043
Study Arms / Comparison Groups
1
Description: Patients who received ADVM-043 in a treatment protocol
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
6
Start Date
December 27, 2018
Completion Date
December 6, 2021
Primary Completion Date
December 6, 2021
Eligibility Criteria
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)
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Charlton Strange, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03804021
Organization ID
ADVM-043-03
Responsible Party
Sponsor
Study Sponsor
Adverum Biotechnologies, Inc.
Study Sponsor
Charlton Strange, MD, Principal Investigator, Medical University of South Carolina, Charleston, SC, USA
Verification Date
March 2022