A Phase 1/2 interventional study of RTx-021 in Stargardt Disease, sponsored by Ray Therapeutics, Inc.. Recruiting at 3 sites in United States. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2026-04-15.
Sponsored by Ray Therapeutics, Inc. · Phase 1/2, Interventional, and Treatment
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.
This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.
67 studies on the registry are indexed under Stargardt Disease; 26 are open to participants now.
This study's planned enrollment of 18 is below the median of 28 across 44 interventional studies indexed under Stargardt Disease.
Browse Stargardt Disease studies →Ray Therapeutics, Inc. is the lead sponsor of 4 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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.
Genetic: 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.
Genetic: 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.
Genetic: RTx-021
Optogenetic gene therapy
Incidence of Treatment-Emergent Adverse Events
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
Time frame: 6 Months
Best Corrected Visual Acuity (BCVA)
Change from Baseline to Month 6 after injection with RTx-021 in BCVA
Time frame: 6 Months
Low Luminance Visual Acuity (LLVA)
Change from Baseline to Month 6 after injection with RTx-021 in LLVA
Time frame: 6 Months
MNREAD Reading Assessment
Change from Baseline to Month 6 after injection with RTx-021 in reading acuity and reading speed.
Time frame: 6 Months
Contrast Sensitivity
Change from Baseline to Month 6 after injection with RTx-021 in contrast sensitivity
Time frame: 6 Months
Visual Field
Change from Baseline to Month 6 after injection with RTx-021 in the total area in which objects can be seen
Time frame: 6 Months
Low Vision Quality of Life (VA LV VFQ-48)
Change from Baseline to Month 6 after injection with RTx-021 in low vision quality of life questionnaire VA LV VFQ-48. A total score will be calculated with higher scores indicating an improvement.
Time frame: 6 Months
Plan to share: No — IPD is not currently being shared as this investigational treatment has not received regulatory approval.
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Ray Therapeutics, Inc.