Long-Term Follow-up Study of ADVM-043

Alpha-1 Antitrypsin Deficiency Clinical Trial

Official title:

Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03804021
• Other study ID #: ADVM-043-03
• Status: Completed
• Start date: December 27, 2018
• Est. completion date: December 6, 2021

Study information

• Verified date: March 2022
• Source: Adverum Biotechnologies, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: December 6, 2021
• Est. primary completion date: December 6, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Alpha-1 Antitrypsin Deficiency

Intervention

Genetic:
• ADVM-043
Long term follow-up of subjects that received ADVM-043

Locations

Country	Name	City	State
United States	Medical University of South Carolina	Charleston	South Carolina
United States	University of Florida	Gainesville	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Adverum Biotechnologies, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043	Safety was assessed by collection of severity and relatedness of Adverse Events to ADVM-043, Adverse Events of Interest and Serious Adverse Events related to ADVM-043	2 years