RECRUITING

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived from IPSCs in Patients with Primary Photoreceptor Disease

Quick Facts

Study Start:2025-03-10
Study Completion:2030-10
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06789445

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Genetically confirmed diagnosis of primary photoreceptor (PR) disease
  2. * 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.
  3. * Retinal structure examination in the study eye (SE) demonstrating regions suitable for cell administration.
  1. * History of/currently active clinically relevant, ocular inflammation or infection
  2. * Glaucoma or other significant optic neuropathy
  3. * Diabetic macular edema or diabetic retinopathy
  4. * Clinically significant cystoid macular edema
  5. * Spherical equivalent refractive error of greater than 8.00 diopters myopia
  6. * Ocular surgery ≤3 months before Screening
  7. * Monocular vision (ie, no light perception in the fellow eye)
  8. * Presence of clinically significant anti-OpCT-001 HLA antibodies at Screening
  9. * Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.
  10. * Any current infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression
  11. * History of any cell therapy, gene therapy, or retinal implant at any time
  12. * Previously received a bone marrow or solid organ transplant

Contacts and Locations

Study Contact

Cheryl Fiedler
CONTACT
1-860-917-4408
clinicaltrials@bluerocktx.com

Study Locations (Sites)

Locations: University of Miami, Bascom Palmer Eye Institute
Miami, Florida, 33136
United States

Collaborators and Investigators

Sponsor: BlueRock Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-03-10
Study Completion Date2030-10

Study Record Updates

Study Start Date2025-03-10
Study Completion Date2030-10

Terms related to this study

Keywords Provided by Researchers

  • Primary Photoreceptor disease
  • CLARICO
  • Photoreceptor cells
  • Inherited retinal disease
  • Cell Therapy
  • Cellular Therapy
  • Usher Syndrome
  • Cone-Rod Disease
  • Rod-Cone Disease
  • Retinitis Pigmentosa

Additional Relevant MeSH Terms

  • Primary Photoreceptor Disease
  • Retinitis Pigmentosa (RP)
  • Usher Syndrome
  • Cone-Rod Dystrophy
  • Inherited Retinal Disease (IRD)
  • Rod-Cone Dystrophy
  • Rod-Cone Disease
  • Cone-Rod Disease
  • Retinal Degeneration