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.
Male and female patients >= 16 years of age
Able to comply with the study visit schedule and all protocol assessments
Diagnosis of Stargardt Disease (genetic testing required)
Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months
Concurrent participation in another interventional clinical ocular study
Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications
Sequential
RTx-021
GeneticOptogenetic 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
Best Corrected Visual Acuity (BCVA)
Change from Baseline to Month 6 after injection with RTx-021 in BCVA
Low Luminance Visual Acuity (LLVA)
Change from Baseline to Month 6 after injection with RTx-021 in LLVA
MNREAD Reading Assessment
Change from Baseline to Month 6 after injection with RTx-021 in reading acuity and reading speed.
Contrast Sensitivity
Change from Baseline to Month 6 after injection with RTx-021 in contrast sensitivity
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