Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders

Background/aims:Pediatric rare disease presents a challenging situation of high unmet need and a limited pool of potential clinical trial participants. Understanding perspectives of parents of children who have not participated in trials may facilitate approaches to optimize participation rates. The objective of this study was to explore factors associated with parental interest in enrolling children with pediatric neuromuscular disorders in clinical trials.Methods:Parents of individuals with Duchenne or Becker muscular dystrophy and spinal muscular atrophy were recruited through advocacy organizations, a registry, and clinics. These parents (N = 203) completed a questionnaire including assessments of barriers and facilitators to clinical trial participation, parents’ interest in trial participation, and their perceptions of others’ views about participation in a clinical trial.Results:Trial interest in participating parents was high (64% combined group). The most highly endorsed barrier to participation ...