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A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
Sponsor: AAVantgarde Bio Srl
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.
Official title: An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)
Key Details
Gender
All
Age Range
8 Years - 55 Years
Study Type
INTERVENTIONAL
Enrollment
75
Start Date
2025-09-29
Completion Date
2032-07
Last Updated
2026-03-09
Healthy Volunteers
No
Conditions
Interventions
AAVB-039
Single subretinal administration
Locations (6)
Retina Vitreous Associates Medical Group
Los Angeles, California, United States
Mayo Clinic
Rochester, Minnesota, United States
Retina Consultants of Texas
Bellaire, Texas, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Moorfields Eye Hospital NHS Foundation Trust
London, England, United Kingdom
The Retina Clinic
London, England, United Kingdom