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)

Recruiting Phase 1 Phase 2 Interventional Study
Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
12 – 65
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
57 (estimated)
Sponsor
Splice Bio · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: To be eligible for study participation, subjects must meet the following criteria: 1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same. 2. Are male or female adolescents and adults, aged as follows…
Contacts

SpliceBio

+34 934 02 04 56

clinicaltrials@splice.bio

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