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.
This is an open-label, non-randomized, multi-center, dose-escalation and dose-expansion, first-in-human, Phase I/IIa study. Participants will be followed for a total of five years after receiving a single unilateral intravitreal injection of SPVN20 in their study eye, and will be monitored by an independent Data Safety Monitoring Committee. Eligible patients will be assigned by sequential enrollment to one of the following three cohorts: Cohort 1: low dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 2: medium dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 3: high dose of SPVN20 will be evaluated in a total of 9 participants.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
27
SPVN20
SPVN20
SPVN20
Ghent University Hospital
Ghent, Belgium
RECRUITINGCHNO XV-XX Paris - CIC 1423
Paris, France
RECRUITINGRoyal Victoria Eye And Ear Hospital
Dublin, Ireland
RECRUITINGSafety and Tolerability
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 6 months
Ocular safety
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
Time frame: 6 months
Best Corrected Visual Acuity (BCVA)
Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
Time frame: 6 months
Full-field stimulus threshold (FST) test
Change in FST from Baseline to Month 6 after dosing with SPVN20
Time frame: 6 months
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