Fragile X syndrome clinical trials: exploring parental decision-making.

Area of Science:

• Pediatric pharmacology
• Genetics and heredity
• Clinical trial management

Background:

• Understanding parental decision-making for pediatric drug trials in Fragile X syndrome (FXS) is crucial.
• Identifying factors influencing trial participation among children with FXS is essential for recruitment and trial design.

Purpose of the Study:

• To explore parental preferences, motivations, barriers, and decision-making ease regarding drug trial participation for children with FXS.
• To compare decision-making experiences between parents who enrolled their child (Joiners) and those who declined (Decliners).

Main Methods:

• Qualitative study involving semi-structured interviews with parents of children with FXS.
• Analysis of data from 16 parents who enrolled their child and 15 who declined participation.
• Inductive content analysis to interpret themes in parental decision-making.

Main Results:

• Key factors influencing decisions included attitudes toward symptom management, perceived direct benefit, logistical convenience, and risk of side effects.
• The primary motivation for participation was the potential for direct benefit to the child.
• No parents reported decisional regret, though decision-making ease varied from easy to difficult.

Conclusions:

• Parents of children with FXS may be more risk-averse in trial decisions compared to parents of children with life-threatening conditions.
• Future trials should clearly explain outcome variable rationales and target symptoms parents find most concerning.
• Interventions to aid parents in deliberating motivations and barriers for FXS drug trial participation are recommended.