All Times EDT
Keywords: myopia, pediatric population, group sequential, Type I error control, interim analysis, patient reported outcome measure, patient’s voice
Pediatric myopia has become a global public health concern as it increases serious sight-threatening diseases risk (cataract, glaucoma, retinal detachment etc). Early onset of myopia and moderate myopia at young age are risk factors for severe myopia. To address this highly unmet need, JJVCI developed a soft contact lens to reduce myopia progression in children. This presentation highlights the patient’s voice embedded in 2 key design elements of a global pediatric double-masked, randomized, two arms (Test vs Control lens) trial to demonstrate the safety and myopia control effectiveness of a bilateral daily-disposable lens: acceleration of time to market and improved assessment of benefit/risk profile. For early detection of benefits, a group-sequential design links the study duration to interim analyses (IA) results, with superiority testing of 2 efficacy endpoints every 6 months between 1 and 4 years follow-up. The overall Type 1 error rate is controlled at 1-sided 0.025 level by a “two-dimensional” alpha control strategy, where multiplicity dimensions are: the multiple IAs and the multiple changes from baseline within each IA. The 2 efficacy co-primary endpoints will be analyzed separately using a linear repeated measures mixed model. To enhance the safety profile assessment with patient’s perspectives, JJVCI developed a new fit-for-purpose Patient Reported Outcome Measure (PROM) that evaluates visual symptoms, near visual function, distance visual function and school-related impact in children. This Pediatric Myopia Control Questionnaire (PMCQ) was created and validated as no established instrument existed in this context. While the effectiveness of the myopia control treatment is promising, the novel optical lens design may slightly reduce the quality of vision compared to conventional single-vision contact lenses. Hence, direct patient-reported assessments of visual symptoms, function and vision related quality of life are critical safety related outcome.