Active — Not RecruitingPhase 2/3

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

United States51 participantsStarted 2023-08-25

Plain-language summary

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.

Who can participate

Age range5 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Males or females aged ≥5 years at the time of consent.
✓. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies).
✓. Adult subjects who have BCVA of 75 letters or less in the study eye (20/32 Snellen equivalent) and Pediatric subjects who have a BCVA of 35 letters or better in the study eye (20/200 Snellen equivalent).
✓. The study eye should have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns, and total lesion size must add to less than or equal to ≤ 18 mm\^2 (approximately 7-disc areas).
✓. Have detectable outer nuclear layer (ONL) in the macular region

Exclusion criteria

✕. Participation in ongoing antiretroviral therapy treatment.
✕. Participation in any investigational therapy study or receipt of investigational treatment within 60 days prior to screening or 5 half-lives (whichever is longer).
✕. Macular atrophy secondary to any disease other than Stargardt Disease (STGD).

What they're measuring

Change from baseline in atrophic lesion size as measured by Fundus Auto Fluorescence

Timeframe: 12 months (Screening to 12 months post OCU410ST administration)

Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))

Timeframe: 12 months (Screening to 12 months post OCU410ST administration)

Trial details

NCT IDNCT05956626

SponsorOcugen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-09-28

View on ClinicalTrials.gov More Stargardt Disease trials

Plain-language summary

Who can participate

Age range5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Males or females aged ≥5 years at the time of consent.
✓. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies).
✓. Adult subjects who have BCVA of 75 letters or less in the study eye (20/32 Snellen equivalent) and Pediatric subjects who have a BCVA of 35 letters or better in the study eye (20/200 Snellen equivalent).
✓. The study eye should have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns, and total lesion size must add to less than or equal to ≤ 18 mm\^2 (approximately 7-disc areas).
✓. Have detectable outer nuclear layer (ONL) in the macular region

Exclusion criteria

✕. Participation in ongoing antiretroviral therapy treatment.
✕. Participation in any investigational therapy study or receipt of investigational treatment within 60 days prior to screening or 5 half-lives (whichever is longer).
✕. Macular atrophy secondary to any disease other than Stargardt Disease (STGD).

What they're measuring

Change from baseline in atrophic lesion size as measured by Fundus Auto Fluorescence

Timeframe: 12 months (Screening to 12 months post OCU410ST administration)

Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))

Timeframe: 12 months (Screening to 12 months post OCU410ST administration)