Long-Term Follow-up Study of ADVM-043

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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 study of subjects in the prior ADVM 043-01 gene therapy
      clinical study (ADVANCE) for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
    

Detailed Description

      ADVM-043-03 is a long-term follow-up safety study of subjects in the ADVM 043 01 gene therapy
      clinical study (ADVANCE) delivering ADVM-043 for treatment of Alpha-1 Antitrypsin (A1AT)
      deficiency. Subjects will roll over after 12 months in the ADVANCE study into this study for
      continued safety monitoring. ADVM-043-03 is a multi-site observational study, without
      administration of investigational product, evaluating the long-term safety of ADVM-043
      administered in a prior study. Subjects who received ADVM-043 will be followed for up to 3
      years post-treatment
    


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:  Subjects who received a dose of ADVM-043 in a prior clinical study

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Genetic

Estimated Enrollment

6

Start Date

January 11, 2019

Completion Date

December 31, 2021

Primary Completion Date

September 30, 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

Administrative Informations


NCT ID

NCT03804021

Organization ID

ADVM043-03


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

October 2019