Long-Term Follow-up Study of ADVM-043

Adverum Biotechnologies

Status: Completed

Conditions: Alpha-1 Antitrypsin Deficiency

Treatments: Genetic: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT03804021

ADVM-043-03

Details and patient eligibility

About

ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.

Full 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 (ADVM-043-01; NCT02168686) were enrolled into this LTFU study for continued safety monitoring for a 2-year period.

Enrollment: 6 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)

Trial design

6 participants in 1 patient group

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Description:

Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Treatment:

Genetic: No Intervention