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 range3 Months – 36 Months
SexALL
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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
✕. 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
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)
. 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