A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease
Running, not enrolling · Phase 2/Phase 3
Conditions studied: Stargardt Disease
In brief
Phase 2/3 Pivotal Confirmatory Clinical Trial is a randomized, outcome assessor-masked, multicenter study, that will enroll fifty-one (51) subjects. Subjects will be enrolled in a 2:1 ratio to either the treatment group (n=34 subjects) or to an untreated control group (n=17 subjects). Phase 1 is complete and closed for enrollment. It was a multicenter, open-label, dose ranging/dose escalation study that enrolled 9 subjects. OCU410ST Phase 1- Retinal Structure and Visual Function Data Results * Safety: Favorable safety and tolerability profile No SAE deemed related to OCU410ST including intraocular inflammation, exudation, endophthalmitis, anterior ischemic optic neuropathy (AIONs) or vasculitis. * Primary Endpoint: Structural Improvement Atrophic lesions grew slower by 48% at 12M in evaluable treated eyes when compared to untreated eyes * Secondary Endpoint: Visual Function (BCVA) 100% evaluable treated eyes demonstrated stabilization or improvement when compared to untreated eyes in visual function OCU410ST Phase 1- Structural and Functional Outcomes at 12M Data Results * The GARDian3 clinical trial for ABCA4- related retinopathies including Stargardt disease builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable treated eyes compared to untreated eyes * Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.
Key facts
- Study ID
- NCT05956626
- Run by
- Ocugen
- People needed
- 51
- Starts
- 2023-08-25
- Expected to finish
- 2026-09-28
- Last updated by the study team
- 2026-07-31
Who can join
Age: 5 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Associated Retina Consultants — Phoenix, Arizona, United States
- Retinal Consultants Medical Group — Sacramento, California, United States
- Vitreo Retinal Associates, P.A. — Gainesville, Florida, United States
- Bascom Palmer Eye Institute — Miami, Florida, United States
- Advanced Research, LLC — Pompano Beach, Florida, United States
- Retina Partners Midwest, P.C. — Carmel, Indiana, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Mississippi Retina Associates — Jackson, Mississippi, United States
- The Retina Institute — St Louis, Missouri, United States
- Duke Eye Center — Durham, North Carolina, United States
- Erie Retina Research, LLC — Erie, Pennsylvania, United States
- Retina Consultants of Texas — Bellaire, Texas, United States
- Retina Foundation of the Southwest — Dallas, Texas, United States
- Valley Retina Institute — McAllen, Texas, United States
Full record on ClinicalTrials.gov
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