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

Not Applicable

Recruiting

• Conditions: Stargardt Disease
• Interventions: Drug: OCU410ST

• Registration Number: NCT05956626
• Lead Sponsor: Ocugen

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.

Detailed Description

Name of Investigational Product: OCU410ST Name of Active Ingredient: Adeno-associated viral vector 5 human RORA (AAV5-hRORA)

Title of Study: A PHASE 1 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE AND PHASE 2/3 PIVOTAL CONFIRMATORY CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE

Study Center(s): Approximately fifteen study centers in the US.

Background: Stargardt disease is an eye disease that causes vision loss in children and young adults. It is an inherited disease caused by faulty genes that cause buildup of fat deposits in the eye. Currently, there is no approved treatment available for Stargardt disease.

OCU410ST Product Information:

OCU410ST is an Adeno-Associated Virus serotype 5 containing human RORA for the treatment of Stargardt disease. Dysregulation in lipid metabolism, oxidative stress, and anti-inflammatory mechanisms are critical for pathogenesis and progression of Stargardt disease. The role of hRORA in regulating these gene pathways strongly suggests OCU410ST could restore homeostasis in the eye and thereby serve as a therapeutic candidate for Stargardt disease.

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.

Note: Data will be collected for the untreated eye at Screening, 4-month, 8-month, 12-month, four (4) long-term safety follow-up visits, and early termination visit (if applicable).

Data will be collected for the treated eye at Screening, treatment Day 1, Day 2, Day 15, 4-month, 8-month, 12-month, four (4) long-term safety follow-up visits, and early termination visit (if applicable).

Enrollment in the Phase 1 study is complete. Phase 1 enrolled a total of nine subjects in low, medium and high dose cohorts.

Low Dose Cohort (3.75×10E10 vg/mL):

Three (3) Subjects received a single subretinal injection of 200 µL OCU410ST in low dose concentration (3.75×10E10 vg/mL).

Medium Dose Cohort (7.5×10E10 vg/mL):

Three (3) Subjects received a single subretinal injection of 200 µL OCU410ST in medium dose concentration (7.5×10E10 vg/mL).

High Dose Cohort (2.25×10E11 vg/mL):

Three (3) Subjects received a single subretinal injection of 200 µL OCU410ST in high dose concentration (2.25×10E11 vg/mL).

Study Timeline

• Study First Submitted: 2023-06-30
• Study First Submitted QC: 2023-07-13
• Study First Posted: 2023-07-21
• Study Start: 2023-08-25
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-24
• Last Update Posted: 2025-07-29
• Primary Completion: 2026-09-28
• Study Completion: 2026-09-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 51

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Phase 2/3 Randomized Treatment Arm	OCU410ST	Subjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL

Primary Outcome Measures

Name	Time	Method
Change from baseline in atrophic lesion size as measured by Fundus Auto Fluorescence	12 months (Screening to 12 months post OCU410ST administration)	Change in the area of atrophy will be evaluated from the baseline measurements, using FAF to assess the loss of retinal layers.
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))	12 months (Screening to 12 months post OCU410ST administration)	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).

Secondary Outcome Measures

Name	Time	Method
Change from baseline in Best Corrected Visual Acuity (BCVA)	12 months (Screening to 12 months post OCU410ST administration)	Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
Change from baseline in Low Luminance Visual Acuity (LLVA)	12 months (Screening to 12 months post OCU410ST administration)	Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Low Luminance Visual Acuity (LLVA) letter score. A higher score represents better vision.

Trial Locations

Locations (13)

Vitreo Retinal Associates, P.A.; 🇺🇸 Gainesville, 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

The Retina Institute; 🇺🇸 Saint Louis, Missouri, United States

Erie Retina Research, LLC; 🇺🇸 Erie, Pennsylvania, United States

Valley Retina Institute; 🇺🇸 McAllen, Texas, United States

Associated Retina Consultants; 🇺🇸 Phoenix, Arizona, United States

Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States

Mississippi Retina Associates; 🇺🇸 Jackson, Mississippi, United States

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