Recruiting
Phase 1
Phase 2

OpCT-001

Sponsor:

BlueRock Therapeutics

Code:

NCT06789445

Conditions

Primary Photoreceptor Disease

Retinitis Pigmentosa (RP)

Usher Syndrome

Inherited Retinal Disease (IRD)

Rod-Cone Dystrophy

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

OpCT-001

OpCT-001

Study Details

Brief summary:

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 approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses 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.

Conditions

Primary Photoreceptor Disease

Retinitis Pigmentosa (RP)

Usher Syndrome

Inherited Retinal Disease (IRD)

Rod-Cone Dystrophy

Study ID

NCT06789445

Start date

Mar 10, 2025

Status verified date

Aug, 2026

Completion date

Oct, 2030

Anticipated

Primary completion date

Oct, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

54 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

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

experimental: Cohort 2

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

experimental: Cohort 3 (optional)

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

experimental: Cohort 4

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

Interventions

OpCT-001

Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).

OpCT-001

Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Primary outcome measure

  • Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit [ Time Frame: From OpCT-001 administration through the Week 52 visit ]

Central Contacts and Locations

Central contacts

Patient Engagement

1-877-380-3931clinicaltrials@bluerocktx.com

Locations

University of Southern California /Children's Hospital of Los Angeles

Recruiting

Los Angeles, California, United States, 90007

Contacts

Patient Engagement

877-380-3931clinicaltrials@bluerocktx.com

Hilary Schwartz-Gale

hschwartz@chla.usc.edu

University of Miami, Bascom Palmer Eye Institute

Recruiting

Miami, Florida, United States, 33136

Contacts

Patient Engagement

877-380-3931clinicaltrials@bluerocktx.com

Mid Atlantic Retina/ Wills Eye

Recruiting

Philadelphia, Pennsylvania, United States, 19107

Contacts

Patient Engagement

877-380-3931clinicaltrials@bluerocktx.com

Study Coordinator

215-928-3092alemus@chla.usc.edu

Retina Foundation of the Southwest

Recruiting

Dallas, Texas, United States, 75231

Contacts

Patient Engagement

877-380-3931clinicaltrials@bluerocktx.com

Jennifer Herrera

214-363-3911jherrera@retinafoundation.org

University of Wisconsin

Recruiting

Madison, Wisconsin, United States, 53705

Contacts

Patient Engagement

877-380-3931clinicaltrials@bluerocktx.com

More Information

Sponsor

BlueRock Therapeutics

Last update posted

Aug 19, 2026

Last verified

Aug, 2026

Keywords

  • CLARICO
  • Photoreceptor cells
  • Cell Therapy
  • Cellular Therapy
  • Usher Syndrome
  • Retinitis Pigmentosa
  • Inherited retinal disease (IRD)
  • Primary Photoreceptor disease (PPD)
  • Rod-Con Disease (R-CD)
  • Cone-Rose disease (C-RD)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by BlueRock Therapeutics on 2026-08-19.