ASTRA Study
Research type
Research Study
Full title
A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (ASTRA)
IRAS ID
1010869
Contact name
Leigh Shaw
Contact email
Sponsor organisation
SpliceBio, S.L.
Clinicaltrials.gov Identifier
Research summary
This is a first-in-human study targeting participants with moderate or advanced Stargardt’s (STGD1) disease. Treatment is with an experimental gene therapy called SB-007, administered as a one-time subretinal injection into the study eye. Only one eye will be treated with SB-007.
The study will be performed up to 11 clinic visits and the participant followed for 96 weeks post-surgery. Participants will be treated at one of three dose levels or placed in a control group (>18-year-olds only). The study will include an independent Data Safety Monitoring Board (DSMB) to monitor safety at each dose level before providing the green light to escalate.
The primary endpoint will be the safety and tolerability of SB-007 through to Week 96. The study will also measure efficacy, with the key secondary endpoint measuring a change from baseline in lesion growth on imaging, comparing the eye treated with SB-007 and the untreated control eye at Week 96.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0028
Date of REC Opinion
20 Mar 2025
REC opinion
Further Information Favourable Opinion