Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

Part of paid clinical trials in Bakersfield, California.

Sponsor
Ray Therapeutics, Inc.
Study ID
NCT07439887
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Stargardt Disease

Eligibility Criteria

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

Interventions

  • RTx-021 — GENETIC
    Optogenetic gene therapy

Study Details

A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.

Key Dates

Start date
Jan 28, 2026
Status verified
Feb 2026
Primary completion
Dec 1, 2030
Completion
Dec 1, 2030

Study Design

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

Arms

  • Experimental: Low Dose RTx-021
    Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.
  • Experimental: Middle Dose RTx-021
    Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.
  • Experimental: High Dose RTx-021
    Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Events [ Time Frame: 6 Months ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
RayTx Clinical SiteBakersfieldCalifornia93309-
UPMC Vision InstitutePittsburghPennsylvania15219
UPMC Vision Institute Clinical Trials Team
[email protected]
412-642-7888
RayTx Clinical SiteBellaireTexas77401-

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By research site
RayTx Clinical Site· Bakersfield, CAUPMC Vision Institute· Pittsburgh, PARayTx Clinical Site· Bellaire, TX

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