Long-Term Follow-up Study of ADVM-043

May 15, 2024 updated by: Adverum Biotechnologies, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Study Type

Observational

Enrollment (Actual)

6

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients who received ADVM-043 in a treatment protocol

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Roll over
Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)
Genetic: No Intervention
Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
Time Frame: 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
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Adverum Biotechnologies, Inc.

Investigators

  • Principal Investigator: Charlton Strange, MD, Medical University of South Carolina, Charleston, SC, USA
  • Principal Investigator: Mark Brantly, MD, University of Florida, Gainesville, FL, USA

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 27, 2018

Primary Completion (Actual)

December 6, 2021

Study Completion (Actual)

December 6, 2021

Study Registration Dates

First Submitted

January 11, 2019

First Submitted That Met QC Criteria

January 11, 2019

First Posted (Actual)

January 15, 2019

Study Record Updates

Last Update Posted (Actual)

September 20, 2024

Last Update Submitted That Met QC Criteria

May 15, 2024

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ADVM-043-03

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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