ORIGINAL RESEARCH article
Front. Pediatr.
Sec. Pediatric Neurology
This article is part of the Research TopicNew Insights into Pediatric Neurology: Neurological Disorders and Epileptic EncephalopathiesView all 19 articles
Ketogenic drug tricaprilin (CER-0001) for the treatment of refractory infantile epileptic spasms: a phase 1/2a study
Provisionally accepted
- 1Robert Wood Johnson Medical School, Rutgers, The State University of New Jersey, New Brunswick, United States
- 2Cerecin Inc, Denver, Colorado, United States
- 3Queensland Children's Hospital, Children's Health Queensland, Brisbane, Australia
- 4KK Women's and Children's Hospital, Singapore, Singapore
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Objective: Ketogenic diets (KD) have been used in the management of multiple pediatric and adult epilepsies. CER-0001 (tricaprilin) is an orally administered, liquid, investigational ketogenic drug with preclinical efficacy in an infantile spasms (IS) animal model. This study aimed to evaluate the safety and tolerability of CER-0001 for the treatment of drug-resistant IS in individuals not following a KD.Participants underwent a 28-day study period following baseline measurements which included a 24-hour video electroencephalogram (vEEG) and caregiver diary. CER-0001 was titrated over a period of up to 14 days, with the total daily dose divided into 4 doses every 6 hours to determine the maximum tolerated dose or to achieve seizure control. Participants who experienced improved seizure control were maintained on the dose for 7 days, while those without clinical benefit underwent a repeat vEEG on the final day of titration to confirm continuation to the maintenance phase. A one-year open-label extension phase was offered to participants in Australia who exhibited improvement in seizure control.Results: Out of the 8 participants enrolled, the majority of reported treatment emergent adverse events (TEAEs) were mild and moderate. The most common TEAEs were gastrointestinal (GI) related, observed in 75% of participants, with vomiting reported in 50% of participants. Two participants (25%) experienced severe TEAEs. Four participants showed a 50-75% improvement in seizure clusters on the vEEG, while 2 experienced a 100% improvement. Of the 8 participants who completed titration, 7 continued into maintenance, and 2 continued to the open-label extension. One participant in Singapore continued receiving CER-0001 through the Special Access Route (SAR) Program. Overall, CER-0001 was well tolerated and showed preliminary benefits for refractory IS.Significance: Our findings demonstrate that CER-0001 offers a novel potential treatment option as a prescription drug for ketogenic therapy, without the restrictions imposed by KDs.
Keywords: Epilepsy, infantile spasms, ketogenic, Seizures, tricaprilin
Received: 11 Feb 2025; Accepted: 29 Dec 2025.
Copyright: © 2025 Cantillon, Chen, Mcintyre, Henderson, Riney, Chan and Chow. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Marc Cantillon
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