Long-Term Follow-up Study of ADVM-043

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 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

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