A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
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)
AAVantgarde Bio Srl
75 participants
Sep 29, 2025
INTERVENTIONAL
Conditions
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
Inclusion Criteria2
- Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- Willingness to adhere to protocol per informed consent
Exclusion Criteria5
- 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
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Single subretinal administration
Locations(6)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07161544