Long-Term Follow-up Study of ADVM-043

• ClinicalTrials.gov Identifier: NCT03804021
• Recruitment Status: Completed
• First Posted: January 15, 2019
• Last Update Posted: March 31, 2022
• Sponsor: Adverum Biotechnologies, Inc.
• Information provided by (Responsible Party): Adverum Biotechnologies, Inc.

Tracking Information

• First Submitted Date: January 11, 2019
• First Posted Date: January 15, 2019
• Last Update Posted Date: March 31, 2022
• Actual Study Start Date: December 27, 2018
• Actual Primary Completion Date: December 6, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 16, 2022)

Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043 [ Time Frame: 2 years ]

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

Original Primary Outcome Measures (submitted: January 11, 2019)

Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043 [ Time Frame: 2 years ]

• Safety will be assessed by collection of Adverse Events of interest and Serious Adverse Events related to ADVM-043

• Current Secondary Outcome Measures: Not Provided

Original Secondary Outcome Measures (submitted: January 11, 2019)

Plasma Concentration of anti-A1AT [ Time Frame: 2 years ]

Change in plasma concentration of anti-A1AT antibody compared to the Baseline value obtained in the ADVM-043 treatment study

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• Study Design: Observational Model: Other; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Patients who received ADVM-043 in a treatment protocol
• Condition: Alpha-1 Antitrypsin Deficiency

Intervention

Genetic: ADVM-043

Long term follow-up of subjects that received ADVM-043

Study Groups/Cohorts

1

Patients who received ADVM-043 in a treatment protocol

Intervention: Genetic: ADVM-043

• Publications *: Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 11, 2019): 6
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: December 6, 2021
• Actual Primary Completion Date: December 6, 2021 (Final data collection date for primary outcome measure)

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)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03804021
• Other Study ID Numbers: ADVM-043-03
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Adverum Biotechnologies, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Adverum Biotechnologies, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

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

• PRS Account: Adverum Biotechnologies, Inc.
• Verification Date: March 2022