A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
CNGB3 retinal gene therapy for patients with achromatopsia
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Comparison of different dosages of AAV-CNGB3
University of Michigan Kellog Eye Centre
Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom
Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
Time frame: 6 weeks
Improvements in Visual Function as Assessed by Visual Acuity
Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.
Time frame: 6 months
Improvements in Retinal Function as Assessed by Static Perimetry
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Time frame: 6 months
Quality of Life Measured by QoL Questionnaires in Children and Adolescents
Change from baseline to Week 24 in EuroQol-5D-Y Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative change from baseline reflects worsening.
Time frame: 6 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Quality of Life Measured by QoL Questionnaires in Adults
Change from baseline to Week 24 in EuroQol-5D-5L Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative change from baseline reflects worsening.
Time frame: 6 Months