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

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.

Conditions

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

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Study Details

Study overview

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.

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

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

Condition
Primary Photoreceptor Disease
Intervention / Treatment

-

Contacts and Locations

Miami

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

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Genetically confirmed diagnosis of primary photoreceptor (PR) disease
  • * 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.
  • * Retinal structure examination in the study eye (SE) demonstrating regions suitable for cell administration.
  • * History of/currently active clinically relevant, ocular inflammation or infection
  • * Glaucoma or other significant optic neuropathy
  • * Diabetic macular edema or diabetic retinopathy
  • * Clinically significant cystoid macular edema
  • * 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)
  • * Presence of clinically significant anti-OpCT-001 HLA antibodies at Screening
  • * 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 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

BlueRock Therapeutics,

Study Record Dates

2030-10