This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.
Age ≥ 18 years old at enrollment.
Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD.
Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria.
Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging).
Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20.
Participant with cortical visual impairment.
Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision.
Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen.
Sequential
SPVN20 (high dose)
Biological/VaccineSPVN20
SPVN20 (medium dose)
Biological/VaccineSPVN20
SPVN20 (low dose)
Biological/VaccineSPVN20
Safety and Tolerability
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Ocular safety
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
Best Corrected Visual Acuity (BCVA)
Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
Full-field stimulus threshold (FST) test
Change in FST from Baseline to Month 6 after dosing with SPVN20
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