Develop an endpoint strategy, including parent-reported measures for evaluation of interventions in clinical trials for infants and young children with cleft lip with or without cleft palate (CL P), 2) Evaluate measurement model, reliability, ad validity of developed parent measures in relation to existing clinical outcome assessments, 3) Evaluate the ability of parent measures to detect improvements associated with primary cleft repair, 4) Finalize the endpoint strategy and implement parent measures in clinical trials with guidance from field leaders. Significance: Infants and children with CL P often experience significant challenges with feeding, nutrition, hearing, speech production, breathing and sleeping. For this reason, they frequently undergo many surgical and non-surgical interventions between birth and 18 months of age, including orthodontic molding of the lip and nose, cleft lip and palate repair, and placement of tympanostomy tubes. Despite published care guidelines however, tremendous variation exists in care for infants with CL P, nationally and internationally. This is in part due to a lack of standard outcome measures that can be applied in this age range across diverse treatments. Innovation: This project will contribute to outcomes development over the life span of persons born with CL P and will provide an endpoint strategy to complement the efforts of the two largest cleft consortiums, Americleft and Eurocleft. We propose to develop novel outcomes to measure observable outcomes reported by parents. These methods are being explored by the FDA for qualifying instruments in device and drug development. The project will assess measurement properties of the parent measures in relation to clinical assessments agreed upon by leaders in the field. We address the entire endpoint strategy in clinical trials and augment existing measures with patient-centered outcomes, a high priority of research for NIH. The potential impact of the project is high, given our involvement of Americleft and Eurocleft leaders and our plans for involving leaders in the field from across the US and abroad in evaluation of the endpoint strategy we develop. Methods and Analysis: We will conduct Phase I interviews and focus group with parents and expert panels to obtain consensus on parent measure content and selection of clinical assessments. In Phase II, we will assess measurement properties of parents' measures and convene with expert panels to finalize parent measures and the endpoint strategy, including implementation of parent measures in clinical trials. Investigators: A multidisciplinary team of experienced researchers and clinicians (pediatrician, surgeons, clinical psychologists, clinical social workers biostatisticians, outcomes scientists) with a history of collaboration is well positioned to successfully accomplish the study aims.
Clefts of the lip with or without cleft palate (CL P) represent one of the most common birth defects with an estimated worldwide prevalence of 1 in 700 live births leaving infants to experience significant challenges with feeding, nutrition, hearing, speech production, breathing and sleeping. Despite published guidelines, tremendous variation in healthcare for infants with clefts exists, nationally and worldwide, due at least in part to a shortage of validated outcome measures for assessing interventions in this population. This project will design an endpoint strategy and develop and validate a Cleft Lip and Palate Observer-Reported Outcomes instrument (COO) for evaluation of interventions in clinical trials.