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RECRUITING
NCT06942572
PHASE1/PHASE2

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)

Sponsor: Splice Bio

View on ClinicalTrials.gov

Summary

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.

Official 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

Key Details

Gender

All

Age Range

12 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

57

Start Date

2025-02-11

Completion Date

2028-12-31

Last Updated

2025-04-29

Healthy Volunteers

No

Interventions

GENETIC

SB-007

Subretinal Administration of SB-007

Locations (5)

UCHealth Sue Anschutz-Rodgers Eye Center,

Aurora, Colorado, United States

Bascom Palmer Eye Institute

Miami, Florida, United States

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States

Oregon Health & Science University

Portland, Oregon, United States

Retina Foundation of the Southwest

Dallas, Texas, United States