CELESTE
Research type
Research Study
Full 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)
IRAS ID
1012354
Contact name
Michael Murtagh
Contact email
Sponsor organisation
AAVantgarde Bio UK Ltd
Research summary
Study 039-101 (CELESTE) is an early-stage clinical trial looking at a potential treatment called AAVB-039 for people with Stargardt disease (STGD1), a genetic eye condition that causes vision loss.
The study has two parts:
• Part A: The researchers will test up to three different dose levels of the treatment in adults (18 years and older) to see which dose is safe and how the body reacts.
• Part B: Once a safe and well-tolerated dose is found that dose will be tested in about 60 participants (40 adults and 20 children aged 8 or older) to further study its effects.
An independent group of experts (data monitoring committee) will help review the safety data. If the results are promising, more studies may follow. People who receive the treatment will be monitored for up to five years to track long-term safety and results.REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0225
Date of REC Opinion
17 Sep 2025
REC opinion
Further Information Favourable Opinion