Long-Term Follow-up Study of ADVM-043

Completed

• Conditions: Alpha-1 Antitrypsin Deficiency
• Interventions: Genetic: No Intervention

• 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.

Study Timeline

• Study Start: 2018-12-27
• Study First Submitted: 2019-01-11
• Study First Submitted QC: 2019-01-11
• Study First Posted: 2019-01-15
• Primary Completion: 2021-12-06
• Study Completion: 2021-12-06
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-16
• Last Update Posted: 2022-03-31

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

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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)

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Roll over	No Intervention	Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

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

Trial Locations

Locations (2)

University of Florida; 🇺🇸 Gainesville, Florida, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States