OCU400 for Retinitis Pigmentosa

(OCU400 Trial)

Recruiting at 8 trial locations

Overseen By Huma Qamar

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Ocugen

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests OCU400, an eye injection, in patients with specific genetic mutations causing RP and LCA. The treatment aims to correct faulty genes to prevent vision loss.

Research Team

Huma Qamar, MD, MPH, CMI

Principal Investigator

Ocugen

Eligibility Criteria

This trial is for adults over 18 with certain genetic mutations causing retinitis pigmentosa or Leber Congenital Amaurosis. Participants must have a specific level of visual impairment and be able to perform a mobility test in low light. They can't join if they're pregnant, breastfeeding, have had recent eye surgery, previous gene therapy, or any condition that might affect the study results.

Inclusion Criteria

Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations for Subgroup 1, autosomal dominant NR2E3 mutation for Subgroup 2, or autosomal dominant RHO mutations for Subgroup 3.

Males or females ≥18 years of age at the time of informed consent.

For the sentinel subject of Cohort 1-3, BCVA ≤ 20/160 in the study eye or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in the study eye.

Exclusion Criteria

Breastfeeding, pregnancy, sperm donation, or inability to practice strict contraception within the Treatment Observation Period.

Any intraocular surgery within 6 months.

Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis)

Treatment Details

Interventions

• OCU400 (Gene Therapy)

Trial Overview The trial tests three doses (low, medium, high) of OCU400 on patients with retinitis pigmentosa and Leber Congenital Amaurosis to evaluate its safety and effectiveness. It includes an initial phase followed by a natural history study across multiple centers involving up to 124 subjects.

Participant Groups

Group I: Phase 2 (High Dose) Experimental Treatment 1 Intervention

Following DSMB confirmation, adult LCA subjects with CEP290 mutation will receive a medium dose concentration of OCU400.

Group II: Pediatric Arm Experimental Treatment 1 Intervention

Pediatric subjects will receive the medium dose concentration and will have subjects with RP and LCA

Group III: Cohort 3 (High Dose) Experimental Treatment 1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup and LCA patients with CEP290

Group IV: Cohort 2 (Mid Dose) Experimental Treatment 1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup

Group V: Cohort 1 (Low Dose) Experimental Treatment 1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup

Group VI: Adult Arm Experimental Treatment 1 Intervention

Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup and LCA patients with CEP290

Group VII: Natural History Study (OCU400-104) Active Control 1 Intervention

A Prospective and Retrospective Natural History Study of RP and LCA: This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects. The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with earliest timepoint on or after the date of their diagnosis of RP or LCA. Subjects will be seen up to a total of four times during the 12 months of the Observational Period, at baseline, 3 months, 6 months and 12 months. A total of up to 100 subjects will be enrolled in the study, including: Approximately 76 newly enrolled subjects consisting of 50 adult RP subjects 6 adult LCA subjects 20 pediatric RP/LCA subjects. Up to 24 subjects that reconsent from the OCU400-101 study (subjects from OCU400-101 will provide data on their untreated eye)

OCU400 is already approved in Canada for the following indications:

Approved in Canada as OCU400 for:

• Retinitis Pigmentosa (Phase III clinical trial)

Who Is Running the Clinical Trial?

Ocugen

Lead Sponsor

Trials

12

Recruited

1,100+