A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease
NCT ID: NCT05956626
Last Updated: 2025-10-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
51 participants
INTERVENTIONAL
2023-08-25
2026-09-28
Brief Summary
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Phase 1 is complete and closed for enrollment. It was a multicenter, open-label, dose ranging/dose escalation study that enrolled 9 subjects.
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Detailed Description
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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).
Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
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.
TREATMENT
SINGLE
Study Groups
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Phase 2/3 Randomized Treatment Arm
Subjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL
OCU410ST
Subretinal Administration of OCU410ST
Phase 2/3 Randomized Control Arm
Subjects will not receive any active study intervention
No interventions assigned to this group
Interventions
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OCU410ST
Subretinal Administration of OCU410ST
Eligibility Criteria
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Inclusion Criteria
2. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies).
3. 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).
4. 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 mm2 (approximately 7-disc areas).
5. Have detectable outer nuclear layer (ONL) in the macular region
Exclusion Criteria
2. Participation in any experimental treatment or research study within 60 days before screening (any previous eye treatments involving gene therapy, stem cells, implanted retinal chips, injections into the eye, or participation in an Alkeus ALK-001 study within the past 6 months)
3. Macular atrophy secondary to any disease other than Stargardt Disease (STGD).
4. Presence of genetic mutations that mimic Stargardt Disease like ELOVL4, or PROM1.
5. 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
5 Years
ALL
No
Sponsors
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Ocugen
INDUSTRY
Responsible Party
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Principal Investigators
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Murthy Chavali, MD, Ph.D.
Role: STUDY_DIRECTOR
Ocugen., Inc.
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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OCU410ST-101
Identifier Type: OTHER
Identifier Source: secondary_id
OCU410ST-101/301
Identifier Type: -
Identifier Source: org_study_id
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