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

NCT05956626 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: OCU410ST-101/301OCU410ST-101
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 14

Brief 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.

Conditions

Stargardt Disease

Keywords

ABCA4-associated retinopathies

Outcome Measures

Primary Outcomes (2)

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

  Change in the area of atrophy will be evaluated from the baseline measurements, using FAF to assess the loss of retinal layers.

  12 months (Screening to 12 months post OCU410ST administration)

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

  Safety will be determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).

  12 months (Screening to 12 months post OCU410ST administration)

Secondary Outcomes (2)

• Change from baseline in Low Luminance Visual Acuity (LLVA)

  12 months (Screening to 12 months post OCU410ST administration)

• Change from baseline in Best Corrected Visual Acuity (BCVA)

  12 months (Screening to 12 months post OCU410ST administration)

Other Outcomes (2)

• Patients Global Impression of Change (PGIC) score

  12 months (Screening to 12 months post OCU410ST administration)

• Care Giver Administered Global Impression of Change score

  12 months (Screening to 12 months post OCU410ST administration)

Study Arms (2)

Phase 2/3 Randomized Treatment Arm

EXPERIMENTAL

Subjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL

Drug: OCU410ST

Phase 2/3 Randomized Control Arm

NO INTERVENTION

Subjects will not receive any active study intervention

Interventions

OCU410ST DRUG

Subretinal Administration of OCU410ST

Phase 2/3 Randomized Treatment Arm

Eligibility Criteria

• Age: 5 Years+
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).
• Any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, intravitreal or subretinal injections, or participation in an Alkeus ALK-001 study within the past 6 months.
• Macular atrophy secondary to any disease other than Stargardt Disease (STGD).
• Presence of genetic mutations that mimic Stargardt Disease like ELOVL4, or PROM1.
• Contraindication to subretinal injection or use of anesthesia (local and/or general).
• Phase 1 was a multicenter, open-label, dose-ranging/dose escalation study. Enrollment is complete for Phase 1

Sponsors & Collaborators

• Ocugen: lead

Study Sites (14)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

Location

Retinal Consultants Medical Group

Sacramento, California, 95825, United States

Location

Vitreo Retinal Associates, P.A.

Gainesville, Florida, 32607, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Advanced Research, LLC

Pompano Beach, Florida, 33064, United States

Location

Retina Partners Midwest, P.C.

Carmel, Indiana, 46032, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Mississippi Retina Associates

Jackson, Mississippi, 39202, United States

Location

The Retina Institute

St Louis, Missouri, 63128, United States

Location

Duke Eye Center

Durham, North Carolina, 27710, United States

Location

Erie Retina Research, LLC

Erie, Pennsylvania, 16505, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Valley Retina Institute

McAllen, Texas, 78503, United States

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jennifer Henrick, RN, MS

  Ocugen., Inc.

  STUDY DIRECTOR

• Mohamed Genead, MD

  Ocugen., Inc

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: The masked biostatistician and other study team members identified by the masking plan
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study will be conducted in two phases. Phase 2/3 Pivotal Confirmatory Clinical Trial is a randomized, outcome assessor-masked, multicenter study. A total of fifty-one (51) 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). Treatment group: 34 subjects. Subjects will receive a single subretinal injection of 200 µL OCU410ST in concentration of 1.5×10E11 vg/mL. Control group: 17 subjects. Subjects who are enrolled in the untreated control group of the study will not receive any treatment. They will be followed according to the same treatment schedule as the treated subjects. Phase 1 was a multicenter, open-label, dose-ranging/dose escalation study. A 3+3 study design will be used for the sequential dose-escalation cohorts in which subjects will receive a single subretinal injection of OCU410ST. Enrollment is complete for Phase 1.

Study Record Dates

• First Submitted: June 30, 2023
• First Posted: July 21, 2023
• Study Start: August 25, 2023
• Primary Completion (Estimated): September 28, 2026
• Study Completion (Estimated): September 28, 2026
• Last Updated: July 31, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)