Parental Decision Making for Fragile X Syndrome Clinical Trials

Background: There is a paucity of research evaluating parental proxy decision making for pediatric clinical trial participation. Fragile X Syndrome (FXS) is a rare genetic condition involving intellectual disability as its main symptom. Clinical trials have been offered and are under development. As intellectual disability directly affects an individual’s personality, perceptions about trial participation and the potential for a disease-modifying therapy is likely different from the perceptions of parents raising a child with a physical disability or illness. Objective: To improve understanding of parental proxy decision making for clinical trial participation of children with intellectual disability. Methods: Interviews were conducted with parents from two groups: those who chose to 1) enroll their child with FXS in a trial; and 2) decline trial participation for their child with FXS. Parents were recruited through support groups. Interviews were transcribed and coded using thematic analysis. Results: The most prevalent contextual decisional factor was attitudes about FXS medications. The most frequent trial-specific decisional factors were parental perceptions of the mechanism of the experimental drug, barriers and risks, and the match between parentally perceived purpose of the trial and their child’s specific symptomatology. Parents’ decision making processes involved weighing the risks and benefits of participation. Many parents reported making trial decisions primarily alone or with the support of their partner. All parents reported low decisional regret, though decisional conflict was found to range from low to high. Conclusions: The most prevalent, primary decisional factors synthesized by parents within their decision-making process were their strongly negative or positive attitudes towards medicating their child’s FXS symptoms, excessive travel to trial site and high number of required appointments, and perceived risk of physical side effects. Potential for direct individual benefit from participation most directly shaped parents’ trial expectations and hopes, providing ultimate motivation for participation and resulting in high therapeutic optimism amongst parents who elected trial participation. Our results offer insight into potential targets of downstream research evaluating interventions to facilitate these decisions and reduce undesirable decisional outcomes