OpCT-001 for Retinitis Pigmentosa

(CLARICO Trial)

Overseen By Cheryl Fiedler

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: BlueRock Therapeutics

Disqualifiers: Ocular inflammation, Glaucoma, Diabetic retinopathy, Monocular vision, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

54 Participants Needed

Trial Summary

What is the purpose of this trial?

Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Eligibility Criteria

This trial is for adults with primary photoreceptor diseases like Usher Syndrome, Retinitis Pigmentosa, and other retinal degenerations. Participants should have a specific type of eye condition without any current treatments that could interfere with the study.

Inclusion Criteria

My eye is suitable for cell therapy based on a retinal exam.

BCVA at Screening for Phase 1: LogMAR 3.9 to LogMAR 1.0. BCVA at Screening for Phase 2: ETDRS letter score 20 and 60.

My eye condition is genetically confirmed.

Exclusion Criteria

I have never had cell, gene therapy, or a retinal implant.

I have or had significant eye inflammation or infection.

Spherical equivalent refractive error of greater than 8.00 diopters myopia

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Treatment

Participants receive OpCT-001 in a dose-escalation design across 4 cohorts to evaluate safety and tolerability

12-24 weeks

Phase 2 Treatment

Participants are randomized to receive one of two selected dose levels of OpCT-001 to assess safety and visual function outcomes

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• OpCT-001

Trial Overview The CLARICO study tests OpCT-001 in a two-part process: Phase 1 checks safety at different doses; Phase 2 looks at how well it works on vision and eye structure in various subgroups of patients with photoreceptor disease.

Participant Groups

Group I: Cohort 4 Experimental Treatment 1 Intervention

OpCT-001 dose level 4 will be administered via subretinal injection.

Group II: Cohort 3 Experimental Treatment 1 Intervention

OpCT-001 dose level 3 will be administered via subretinal injection.

Group III: Cohort 2 Experimental Treatment 1 Intervention

OpCT-001 dose level 2 will be administered via subretinal injection.

Group IV: Cohort 1 Experimental Treatment 1 Intervention

OpCT-001 dose level 1 will be administered via subretinal injection.

Who Is Running the Clinical Trial?

BlueRock Therapeutics

Lead Sponsor

Trials

4

Recruited

270+