A Phase 1/2, First-in-Human 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)

NCTID	NCT06942572 (View at clinicaltrials.gov)
Description	This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1). This is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007. (Show More)
Development Status	Active
Indication	Stargardt Disease
Disease Ontology Term	DOID:0110015
Compound Name	SB-007
Sponsor	Splice Bio
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	57 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	ABCA4
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	dual AAV8
Editor Type	none
Dose 1	Undisclosed dose escalation, 3 levels
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States