OpCT-001 for Retinitis Pigmentosa

(CLARICO Trial)

Recruiting at 1 trial location

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

What You Need to Know Before You Apply

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.

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+