Investigating OpCT-001 for treating primary photoreceptor disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Southern California /Children's Hospital of Los Angeles
  Los Angeles, California, United States Recruiting
• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.