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

First listed
Feb 27, 2026
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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