The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Nanoscope Clinical Site
Miami, Florida, United States
Nanoscope Clinical Site
McAllen, Texas, United States
Type, severity, and incidence of ocular and systemic adverse events (AEs)
Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010
Time frame: 48 weeks
Effect of vMCO-010 as assessed by visual acuity
Change from baseline in BCVA at Weeks 12, 24, 48 in the study eye and the fellow eye
Time frame: 48 weeks
Effect of vMCO-010 on Light-guided Mobility
Change from baseline in Multi-Luminance Mobility Test at weeks 12, 24, 48 in the study eye and the fellow eye
Time frame: 48 Weeks
Effect of vMCO-010 on determination of shape
Change from baseline in accuracy in determination of shape using Low Vision Multi-Parameter Test (LVMPT) at weeks 12, 24, 48 in the study eye and the fellow eye
Time frame: 48 Weeks
Effect of vMCO-010 on determination of optical flow
Change from baseline in accuracy in determination of optical flow using the LVMPT at weeks 12, 24 and 48 in the study eye and the fellow eye
Time frame: 48 Weeks
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