The purpose of this study is to assess the safety, tolerability, and efficacy of tricaprilin in subjects with infantile spasms. This is a single-arm, open-label, pilot study in up to 10 subjects with infantile spasms.
Upon completion of the main phase, subjects who exhibit improvement in seizure control and who tolerate the compound will be offered continued use of the IMP until benefit-risk ratio is no longer favourable, in a one-year open-label extension phase, available to Australian participants only.
Who can participate
Age range
3 Months – 36 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male and female infants ages 3 months to 24 months, inclusive, at the time of parent/legal guardian signing the informed consent
. Clinical diagnosis of IS, confirmed by analysis of a 24-hour video-electroencephalogram (vEEG) recording, including at least one documented spasm
. Continued infantile spasms despite adequate treatment with oral prednisolone (or adrenocorticotropic hormone \[ACTH\]) and vigabatrin
. If being treated with concomitant ASDs (other than ketogenic therapies/diet), current ASDs have been at a constant daily dose for at least 1 week.
. Subject is taking no more than 3 concomitant ASDs
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Timeframe: Up to end of study (Day 5 to 21; depending on subject)
2
To determine the safety and tolerability of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Timeframe: Up to end of study (Day 5 to 21; depending on subject)
3
Extension Phase (for Australian sites only): To determine the safety of daily long-term administration of tricaprilin in subjects with Infantile Spasms (IS)
Timeframe: End of main phase treatment period to end of extension phase (1-year period)
. Subject considered by the Investigator, for any reason, to be an unsuitable candidate to receive the investigational product
. Significant and active pre-existing cardiovascular, renal, liver, infectious, or other systemic disease
. Subject has clinically significant renal impairment
. Clinically significant abnormality on ECG that, in the opinion of the Investigator, increases the safety risks of participating in the study
. Known or suspected allergy to the investigational product
. Known history of aspiration pneumonia within the past year
. Previous participation in another clinical study of the investigational product or received any investigational drug, device, or therapy within 30 days of study entry or within five half-lives of another investigational drug