Peer-reviewed article
Exploring the caregiver journey through randomized controlled trials in dravet syndrome: insights from a cross-sectional survey.
Delattre C, D'Onofrio G, Leiber AE, Nabbout R ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This peer-reviewed study surveyed caregivers of children with Dravet syndrome who participated in four randomized controlled trials of anti-seizure medications between 1996 and 2024. The research explored why families enrolled in trials, their experiences during participation, and barriers to enrollment. Most caregivers reported positive trial experiences, with hope and access to new treatments as primary motivations, though concerns about adverse events and placebo assignment deterred some families. The study identifies trial logistics and emotional impacts as key factors in caregiver decision-making.
Key points
- 88% of trial participation was proposed by pediatric neurologists, indicating physician-driven enrollment pathways
- Main motivations for enrollment were access to new treatments, hope, and confidence in the medical team
- Primary barriers to non-participation were concerns about adverse events, emotional uncertainty, and potential placebo assignment
- Hope, fear of not completing the trial, and stress were most frequently reported emotions during participation
- Daily schedule accommodations for medical appointments were the primary practical barrier reported
Limitations
This is a small cross-sectional survey (44 caregivers total) with retrospective reporting spanning trials from 1996-2024, which may introduce recall bias and survivorship bias since only families who completed trials were surveyed. The survey was conducted in French, limiting generalizability to French-speaking populations.
Abstract
Objective 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). Methods 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. Results 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. Conclusions 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.