Status: Recruiting

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75 subjects

Participants needed: 75

Trial details

Age: 12-65Biological sex: AllType: ObservationalSponsor: Splice BioUpdated: Sep 19, 2025Locations: 20

Eligibility criteria

Provide written consent [+9]

Are an immediate family member (e.g., child, sibling) of the Sponsor or study si... [+10]

Status: Recruiting

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)

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.

Participants needed: 57

Trial details

Phase: Phase 1, Phase 2Age: 12-65Biological sex: AllType: InterventionalSponsor: Splice BioUpdated: Apr 29, 2025Locations: 5

Eligibility criteria

Provide written consent. Subjects under legal age will also provide informed ass... [+11]

Have had any intraocular surgery (including cataract surgery) or thermal laser w... [+10]

Clinical trials

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

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)