The purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)
This open label dose-escalation study evaluated 2 dose levels in up to 11 subjects of retinitis pigmentosa (3 in low dose and 8 in high dose per dose) with active vMCO-010. Subjects with confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination and dilated fundus examination, were considered for participation in this study. The primary endpoint for this study is safety and tolerability of vMCO-I at 16 weeks. All subjects were assessed for 52 weeks following treatment with vMCO-I
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
11
The vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
JPM Rotary Club of Cuttack Eye Hospital and Research Institute
Cuttack, Odisha, India
The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
Safety and tolerability of vMCO-l treatment at Week 16, by assessments based on local and systemic safety issues, as assessed by incidence of Adverse Events.
Time frame: 16 Weeks
Evaluate the treatment effect of vMCO-l as assessed by visual acuity
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with the Freiburg Visual Acuity (FrACT) to provide automated, self-paced, monitored measurement
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Light-guided Mobility assays, performed at different light intensities, to provide functional vision measures using the time to find lighted panel
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Light-guided Mobility assays, performed at different light intensities, to provide functional vision measures using the score based on correct choice
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Static Shape recognition assay, performed at different light intensities, to provide visual function measures using size determination threshold
Time frame: 52 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Static Shape recognition assay, performed at different light intensities, to provide visual function measures using % shape recognition accuracy
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Optical Flow assay, performed at different speeds, to provide visual function measures using the %accuracy in determining direction of flow
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Optical Flow assay, performed at different speeds, to provide visual function measures using the Upper speed limit to determine correct optical flow
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire
Assessment of the treatment effect on Quality of Life changes from baseline to Week 52 with Visual Function Questionnaire-25 (VFQ-25).VFQ25 is a 25-item questionnaire with 47 questions, each question has several responses scored on a scale from 0-5, 0-6, or 0-10. Values are calculated in percentages.
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field
Assessment of the treatment effect with the change from baseline to Week 52 with Humphrey Visual Field (30-2). Visual Field Index (VFI) is calculated in % and Mean Deviation (MD) values are calculated in dB.
Time frame: 52 weeks