Long-Term Follow-up Study of ADVM-043
Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency
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.
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.
Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043
Alpha-1 Antitrypsin Deficiency
Study Arms / Comparison Groups
Description: Patients who received ADVM-043 in a treatment protocol
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
December 27, 2018
December 6, 2021
Primary Completion Date
December 6, 2021
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)
18 Years - N/A
Accepts Healthy Volunteers
Charlton Strange, MD, ,
Adverum Biotechnologies, Inc.
Charlton Strange, MD, Principal Investigator, Medical University of South Carolina, Charleston, SC, USA