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Recruiting NCT06789445

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

Phase I / Phase II Interventional Primary Photoreceptor Disease Retinitis Pigmentosa (RP) Usher Syndrome Inherited Retinal Disease (IRD)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: OpCT-001, OpCT-001.
Who it may be relevant to
Registry conditions: Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD). Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

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 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.

Detailed description

Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner.

Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (\~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.

Interventions

  • Biological OpCT-001
    Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
  • Biological OpCT-001
    Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Primary outcome measures

  • 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]
Secondary outcome measures (1)
  • Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT) [Time frame: From baseline through the Week 52 visit]

Eligibility criteria

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.

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 5 centers
  • University of Southern California /Children's Hospital of Los Angeles — Los Angeles
  • University of Miami, Bascom Palmer Eye Institute — Miami
  • Mid Atlantic Retina/ Wills Eye — Philadelphia
  • Retina Foundation of the Southwest — Dallas
  • University of Wisconsin — Madison

Identifiers

NCT: NCT06789445 · OpCT-001-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗