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Exploring the caregiver journey through randomized controlled trials in dravet syndrome: insights from a

Caroline Delattre1, Gianluca D'Onofrio2, Anne-Emmanuel Leiber1

  • 1Alliance Syndrome de Dravet, 3 sentier des Larris, 45330 Malesherbes, France.

Epilepsy & Behavior : E&B
|August 8, 2026
PubMed
Abstract

Insights

Caregivers generally had positive experiences in Dravet syndrome (DS) clinical trials, motivated by access to new treatments and hope. Key challenges involved managing daily schedules for appointments, but regret was minimal.

Area of Science:

  • Neurology
  • Clinical Trials
  • Rare Diseases

Background:

  • Dravet syndrome (DS) is a severe form of epilepsy often requiring multiple treatments.
  • Clinical trials are crucial for developing new therapies for DS.
  • Understanding caregiver perspectives is vital for successful trial design and recruitment.

Purpose of the Study:

  • To investigate caregiver perceptions of their child's participation in four randomized controlled trials (RCTs) for Dravet syndrome.
  • To explore motivations for enrollment, experiences during trials, and challenges faced by families.

Main Methods:

  • A cross-sectional survey in French was administered to caregivers.
  • The survey used semi-structured questions to assess motivations, perceptions, and challenges.
  • Data were collected from caregivers whose children participated in trials for stiripentol, cannabidiol, fenfluramine, or soticlestat.

Main Results:

  • Most trial participation was initiated by neurologists (88%).
  • Motivations for participation included access to novel treatments and hope; reasons for non-enrollment centered on seizure control, ineligibility, or trial timing.
  • Caregivers reported positive overall trial experiences, with hope and stress being common emotions, and daily schedule disruptions as the main barrier.

Conclusions:

  • This is the first survey to explore caregiver perceptions of epilepsy clinical trial participation.
  • Positive caregiver experiences highlight the importance of patient and stakeholder engagement in optimizing trial recruitment and acceptance.
  • Understanding caregiver perspectives can help improve future clinical trial design and reduce trial failures.
Keywords:
Caregiver burdenEpilepsyPatient participationQuestionnaireRandomized controlled trial

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