Investigating OpCT-001 for treating primary photoreceptor disease
A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
This study is testing a new treatment called OpCT-001 to see if it can safely help adults with primary photoreceptor diseases, like Retinitis Pigmentosa and Usher Syndrome, improve their vision.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 54 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | BlueRock Therapeutics Industry-sponsored |
| Locations | 4 sites (Miami, Florida and 3 other locations) |
| Trial ID | NCT06789445 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and tolerability of OpCT-001 in adults with primary photoreceptor diseases, including Retinitis Pigmentosa and Usher Syndrome. It consists of two phases: Phase 1 focuses on dose escalation and safety assessment, while Phase 2 aims to gather additional safety data and assess the impact of OpCT-001 on visual function and anatomical measures. Participants will be assigned to different cohorts based on their eligibility and will receive varying doses of the treatment. The study employs a randomized, masked design to ensure unbiased results.
Who should consider this trial
Good fit: Ideal candidates are adults with a genetically confirmed diagnosis of primary photoreceptor disease and specific visual acuity criteria.
Not a fit: Patients with significant ocular conditions, recent ocular surgeries, or certain systemic health issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve visual function and quality of life for patients with inherited retinal diseases.
How similar studies have performed: While this approach is novel, similar studies targeting retinal diseases have shown promising results in the past.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Confirmed genetic diagnosis of primary photoreceptor (PR) disease * Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive. * Retinal structure examination in the study eye demonstrating regions suitable for cell administration. Key Exclusion Criteria: * Clinically relevant, active ocular inflammation or infection * Glaucoma or other significant optic neuropathy * Diabetic macular edema or diabetic retinopathy * Clinically significant cystoid macular edema * In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia * Ocular surgery ≤3 months before Screening * Monocular vision (ie, no light perception in the fellow eye) * Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated. * Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression * History of any cell therapy, gene therapy, or retinal implant at any time * Previously received a bone marrow or solid organ transplant
Where this trial is running
Miami, Florida and 3 other locations
- University of Miami, Bascom Palmer Eye Institute — Miami, Florida, United States (Recruiting)
- Mid Atlantic Retina/ Wills Eye — Philadelphia, Pennsylvania, United States (Recruiting)
- Retina Foundation of the Southwest — Dallas, Texas, United States (Recruiting)
- University of Wisconsin — Madison, Wisconsin, United States (Recruiting)
Study contacts
- Study coordinator: Patient Engagement
- Email: clinicaltrials@bluerocktx.com
- Phone: 1-877-380-3931
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.