A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.
This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa or choroideremia. Up to 4 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
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.
Retina Vitreous Associates Medical Group
Beverly Hills, California, United States
RECRUITINGUCI Alpha Clinic
Orange, California, United States
RECRUITINGUPMC Vision Institute
Pittsburgh, Pennsylvania, 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: 12 months
Best Corrected Visual Acuity (BCVA) at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in BCVA
Time frame: 6 and 12 Months
Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in LLVA
Time frame: 6 and 12 Months
Multi luminance mobility at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Visual Change from baseline to Month 6 and 12 in mobility performance at multiple luminance levels
Time frame: 6 and 12 Months
Contrast Sensitivity at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Contrast Sensitivity
Time frame: 6 and 12 Months
Full-field static visual field testing at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in the total area in which objects can be seen
Time frame: 6 and 12 Months
Low Vision Quality of Life at Month 6 and Month 12
Change from baseline in low vision quality of life at Month 6 and Month 12. An algorithm will be applied to assess a total score. Higher scores are a worsening of the condition.
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Retina Consultants of Texas Research Centers
Bellaire, Texas, United States
RECRUITINGTime frame: 6 and 12 Months