A phase 3, randomized clinical trial of soticlestat as adjunctive therapy for Lennox–Gastaut syndrome

Soticlestat did not demonstrate efficacy versus placebo in reducing MMD seizure frequency; the safety findings were consistent with those of previous studies. These data suggest that targeting cholesterol 24-hydroxylase is not a suitable pharmacotherapeutic strategy for LGS.

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Publication Details

Journal
Epilepsia
Published
2026-03-27
DOI
https://doi.org/10.1002/epi.70216
Primary Topic
Epilepsy research and treatment
Type
article
Field-Weighted Citation Impact
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article

A phase 3, randomized clinical trial of soticlestat as adjunctive therapy for Lennox–Gastaut syndrome

Mahnaz Asgharnejad, P. P. Mitra, Ružica Kravljanac, Cecil D. Hahn et al.
Epilepsia
Epilepsy research and treatment
article

A phase 3, randomized clinical trial of soticlestat as adjunctive therapy for Lennox–Gastaut syndrome

Mahnaz Asgharnejad, P. P. Mitra, Ružica Kravljanac, Cecil D. Hahn, Anna Altmann, Philipp von Rosenstiel, Dennis Dlugos, E. Neil G. Marsh, Jimmy Schiemann, Wei‐Ping Liao, Venkatesha Murthy, S. Sheikh, Yasir Khan, Stéphane Auvin, Katsumi Imai, Renzo Guerrini
article en

Abstract

Soticlestat did not demonstrate efficacy versus placebo in reducing MMD seizure frequency; the safety findings were consistent with those of previous studies. These data suggest that targeting cholesterol 24-hydroxylase is not a suitable pharmacotherapeutic strategy for LGS.

Epilepsia
Children's Hospital of Philadelphia (US), Inserm (FR), Délégation Paris 7 (FR), Institut Universitaire de France (FR), Hospital for Sick Children (CA), Sorbonne Paris Cité (FR), National Epilepsy Center (JP), Meyer Children's Hospital (IT), Szent János Kórház (HU), Hôpital Robert-Debré (FR), Institute of Public Health of Serbia (RS), Takeda (United States) (US), NeuroDiderot (FR), University of Florence (IT), University of Pennsylvania (US), Guangzhou Medical University (CN)
Good health and well-being
Openalex Percentile: Top 14%
Epilepsy research and treatment
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