Long-Term Follow-up Study of ADVM-043

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.

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Key information

Conditions

Alpha-1 Antitrypsin Deficiency Chronic Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Disease Attributes Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Lung Diseases, Obstructive Pathologic Processes Pathological Conditions, Signs and Symptoms Pulmonary Disease, Chronic Obstructive Respiratory Tract Diseases Subcutaneous Emphysema alpha 1-Antitrypsin Deficiency

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Florida, Gainesville, Florida, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

No intervention

Genetic

Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Primary outcomes

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

Sponsors and collaborators

Lead sponsor

Adverum Biotechnologies, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start: 2018
Primary completion: 2021
Study completion: 2021
First posted: Jan 15, 2019
Registry last updated: Sep 20, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed