NCT07161544PHASE1, PHASE2Recruiting

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

AAVantgarde Bio Srl

Start Date

9/29/2025

Completion Date

7/1/2032

Summary

The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.

Eligibility Criteria

Age Range: 8 years to 55 years

Inclusion Criteria: * Molecular diagnosis of Stargardt disease due to ABCA4 mutation * Willingness to adhere to protocol per informed consent Exclusion Criteria: * Unwillingness to meet the requirements of the study * Participation in a clinical study with another Investigation Medicinal Product * Previous participation in another gene or cell therapy trial * Any condition that would preclude subretinal surgery * Complicating ocular and/or systemic diseases

Interventions

BIOLOGICAL

AAVB-039

Interested in This Trial?

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Conditions

Stargardt Disease

Locations

Associated Retina Consultants

Phoenix, Arizona 85020

United States

Children's Hospital Los Angeles

Los Angeles, California 90027

United States

Retina Vitreous Associates Medical Group

Los Angeles, California 90211

United States

University of Florida Health Science Center

Jacksonville, Florida 32209

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Retina Consultants of Texas

Bellaire, Texas 77401

United States

Retina Foundation of the Southwest

Dallas, Texas 75231

United States

University of Utah

Salt Lake City, Utah 84112

United States

Oslo University Hospital

Oslo, Kirkeveien 166

Norway

Moorfields Eye Hospital NHS Foundation Trust

London, England EC1V 2PD

United Kingdom

The Retina Clinic

London, England W1G 9AY

United Kingdom