M. Cantillon, S. Chen, N. McIntyre, ST. Henderson, K. Riney, DWS. Chan, J. Lawson, et al. (2025). Ketogenic drug tricaprilin (CER-0001) for the treatment of refractory infantile epileptic spasms: a phase 1/2a study. Frontiers in pediatrics, 13, 1575014. https://doi.org/10.3389/fped.2025.1575014

PubMed 41799021

[Abstract]
OBJECTIVE: Ketogenic diets (KDs) 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 aims to evaluate the safety and tolerability of CER-0001 for the treatment of drug-resistant IS in individuals not following a KD.

METHODS: Participants underwent a 28-day study period following baseline measurements that included a 24 h 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 four doses every 6 h 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 1-year open-label extension phase was offered to participants in Australia who exhibited improvement in seizure control.

RESULTS: Out of the eight participants enrolled in the study, it was found that 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%. Two participants (25%) experienced severe TEAEs. Four participants showed a 50%-75% improvement in seizure clusters on the vEEG, while two experienced a 100% improvement. Of the eight participants who completed the titration phase, seven continued into the maintenance phase of the study, and of these, three continued CER-0001 treatment beyond completion of the maintenance phase, either through the open-label extension protocol in Australia (n = 2), or through the special access route (SAR) program in Singapore (n = 1). Overall, CER-0001 was well tolerated and was associated with 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.