Exploring the caregiver journey through randomized controlled trials in dravet syndrome: insights from a cross-sectional survey.

Abstract

To explore how caregivers perceived their child's participation in four randomized controlled trials (RCTs) for Dravet syndrome (DS), conducted between 1996 and 2024, on stiripentol (STP), cannabidiol (CBD), fenfluramine (FFA), and soticlestat (TAK-935). A semi-structured, cross-sectional survey, in French, assessed families' motivations for enrolling in the trials, their perceptions throughout the different trial phases, and challenges encountered. Ten caregivers reported that their child had participated in the STP trial, 20 in the CBD trial, 13 in the FFA trial, and 1 in the TAK-935 trial. In most cases (39/44, 88%), trial participation was proposed by the treating (paediatric) neurologist. The main reasons for non-enrolment were satisfactory seizure control, the trial having ended before awareness, and ineligibility. Motivations for participation were mostly access to new treatments, hope, and confidence in the medical team, whereas non-participation was primarily driven by concerns about adverse events, emotional impact of uncertainty, and potential placebo assignment. Overall, trial experiences were rated positively: hope, fear of not completing the trial, and stress were the most frequently reported emotions throughout the trial, while the primary barriers related to daily schedules to accommodate regular medical appointments. Regret and solitude post-trial were scored very low. To our knowledge, this is the first survey exploring the caregivers' perceptions of participation in epilepsy clinical trials. Patient and stakeholder engagement is essential for optimizing trial recruitment, acceptance and understanding of the failures.