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

Part of paid clinical trials in Miami, Florida.

Sponsor
BlueRock Therapeutics
Study ID
NCT06789445
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Mar 10, 2025
Status verified
Apr 2026
Primary completion
Oct 31, 2029
Completion
Oct 31, 2030

Study Design

Enrollment
54 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

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.

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

Locations (4)

FacilityCityStateZIPSite coordinators
University of Miami, Bascom Palmer Eye InstituteMiamiFlorida33136
Patient Engagement
[email protected]
877-380-3931
Mid Atlantic Retina/ Wills EyePhiladelphiaPennsylvania19107
Patient Engagement
[email protected]
877-380-3931
Retina Foundation of the SouthwestDallasTexas75231
Patient Engagement
[email protected]
877-380-3931
University of WisconsinMadisonWisconsin53705
Patient Engagement
[email protected]
877-380-3931

Find similar trials in Miami, FL

By research site
University of Miami, Bascom Palmer Eye Institute· Miami, FLMid Atlantic Retina/ Wills Eye· Philadelphia, PARetina Foundation of the Southwest· Dallas, TXUniversity of Wisconsin· Madison, WI

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