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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Optogenetic gene therapy
RayTx Clinical Site
Bakersfield, California, United States
RECRUITINGUPMC Vision Institute
Pittsburgh, Pennsylvania, United States
RECRUITINGRayTx Clinical Site
Bellaire, Texas, United States
RECRUITINGIncidence 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
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