Tricaprilin, an Investigational Ketogenic Drug in Refractory Infantile Spasms
Abstract number :
3.425
Submission category :
4. Clinical Epilepsy / 4C. Clinical Treatments
Year :
2023
Submission ID :
1409
Source :
www.aesnet.org
Presentation date :
12/4/2023 12:00:00 AM
Published date :
Authors :
Presenting Author: Marc Cantillon, MD FAAN – Cerecin
Lilian Chow, MMRD – Cerecin; Sam Henderson, PhD – Cerecin; Eric Kosoff, MD – Hopkins; john Lawson, MD – NSW Health
Rationale:
Ketogenic diets (KD) have been used in the management of multiple pediatric and adult epilepsies. Tricaprilin is an orally administered, liquid, investigational ketogenic agent with preclinical IS model efficacy.
Methods:
A pilot study to evaluate safety and tolerability of drug Tricaprilin for treatment of drug-resistant Infantile Spasms (IS), a disorder characterized by epileptic spasms, developmental regression, and hypsarrhythmia. Tricaprilin is an orally administered, liquid, investigational ketogenic agent with preclinical IS model efficacy. This study investigated use of Tricaprilin in IS, whilst allowing the continuation of the current source of nutrition.
Results:
Overall the treatment was well tolerated with primarily GI adverse events. Two events were graded as severe: one bronchiolitis considered unrelated to treatment and one aspiration because of feeding tube misplacement. There were no lab or other abnormalities. Micronutrients are under evaluation. Random sampling of blood β-hydroxybutyrate (BHB) levels indicates all eight subjects were in ketosis by the end of the titration phase and in the maintenance phase. In four out of eight subjects, efficacy on vEEG reduction in cluster number and duration of epileptic spasms was observed with caregiver diaries and clinician rating in the same direction.
Conclusions:
Positive safety results and benefit observed in some subjects in this Phase 2a Tricaprilin study for the treatment of IS and warrant continued investigation.
Funding: Cerecin supported the trial.
Clinical Epilepsy