OpCT-001 for Retinitis Pigmentosa

(CLARICO Trial)

Recruiting at 3 trial locations

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?

This trial tests a new treatment called OpCT-001 for individuals with retinitis pigmentosa, a condition that causes vision loss. The study aims to determine the safety of OpCT-001 and its effects on vision. Participants will receive different doses of the treatment through an injection into the eye. Individuals with a confirmed diagnosis of primary photoreceptor disease and specific vision capabilities may qualify for this trial. As a Phase 1 and Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group. This offers participants a chance to be among the first to benefit from this innovative therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that OpCT-001 is likely to be safe for humans?

Research shows that OpCT-001 is under study to determine its safety and tolerability. This treatment is being tested in humans for the first time. In these early stages, the main focus is on ensuring its safety.

Early studies did not report severe side effects. However, because the research is still in its initial phases, complete safety information is not yet available. The trial gradually increases the dose to help researchers identify the safest amount for participants.

If approved in the future, this treatment could offer new hope to people with eye diseases like retinitis pigmentosa. For now, ongoing studies must provide more information to fully understand its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for retinitis pigmentosa, which often involve vitamin A supplementation or retinal implants, OpCT-001 is administered directly into the eye using a subretinal injection. Researchers are excited about OpCT-001 because it targets the retina more precisely, potentially leading to improved effectiveness. Additionally, the multiple dose levels being tested, ranging from level 1 to level 4, offer flexibility and customization in treatment, which could result in better outcomes for different patients. This innovative approach could pave the way for more personalized and effective therapies for those affected by this challenging condition.

What evidence suggests that OpCT-001 might be an effective treatment for retinitis pigmentosa?

Research has shown that OpCT-001 aims to help people with retinitis pigmentosa by replacing damaged cells in the eye with new, functioning ones. Early results suggest this treatment can improve vision, particularly in low light. Some patients have seen more lines on an eye chart in dim lighting. The treatment uses special stem cells, capable of becoming different cell types, to restore vision. Although still in the early stages, this approach appears promising for those with primary photoreceptor diseases. ^{1 2 6 7 8}

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+

Citations

1. bluerocktx.com bluerocktx.com/bluerock-therapeutics-announces-first-patient-receives-investigational-therapy-in-phase-1-2a-clinical-trial-of-opct-001-for-the-treatment-of-primary-photoreceptor-diseases/

OpCT-001 is the first iPSC-derived investigational cell ... OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06789445

A Study to Investigate the Safety of OpCT-001 in Adults ... 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 ...

3. fightingblindness.org fightingblindness.org/news/retinitis-pigmentosa-research-advances-899

Retinitis Pigmentosa Research Advances The trial investigators reported mean improvements in low luminance visual acuity (LLVA), the ability to read lines on an eye chart in dim light, for patients ...

4. appliedclinicaltrialsonline.com appliedclinicaltrialsonline.com/view/bluerock-begins-first-human-trial-ipsc-cell-therapy-primary-photoreceptor-diseases

BlueRock Begins First Human Trial of iPSC Cell Therapy ... OpCT-001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease, ...

5. pharmacytimes.com pharmacytimes.com/view/opct-001-stem-cell-derived-therapy-for-primary-photoreceptor-diseases-receives-fda-fast-track-designation

OpCT-001, Stem Cell-Derived Therapy for Primary ... OpCT-001's mechanism of action allows for it to aid in the recovery of vision for patients with primary photoreceptor diseases. The therapy is ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06789445?cond=(CONE-ROD%20DYSTROPHY)%20OR%20(TTLL5)&rank=3

NCT06789445 | A Study to Investigate the Safety of OpCT- ... 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 ...

7. bayer.com bayer.com/media/en-us/bluerock-therapeutics-announces-fda-clearance-of-ind-application-for-investigational-ipsc-derived-cell-therapy-opct-001-for-the-treatment-of-primary-photoreceptor-diseases/

BlueRock Therapeutics announces FDA clearance of IND ... The Phase 1/2a study is designed as a first-in-human study to evaluate the safety and tolerability of subretinal administration of OpCT-001 in ...

8. bjo.bmj.com bjo.bmj.com/content/early/2025/08/18/bjo-2024-326480

Safety and vision outcomes of subretinal gene ... PDE6A-associated retinitis pigmentosa (RP) is an inherited retinal disorder causing severe vision loss, with no current treatment available.