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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Tricaprilin will be emulsified in infant formula/milk
Sydney Children's Hospital
Randwick, New South Wales, Australia
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
KK Women's and Children's Hospital
Singapore, Singapore
To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Treatment emergent adverse events
Time frame: Up to end of study (Day 5 to 21; depending on subject)
To determine the safety and tolerability of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Brussels Infant and Toddler Stool Scale; the scale consists of 4 categories: hard, formed, loose or watery. The category of stools per subject will be compared between baseline and during the study.
Time frame: Up to end of study (Day 5 to 21; depending on subject)
Extension Phase (for Australian sites only): To determine the safety of daily long-term administration of tricaprilin in subjects with Infantile Spasms (IS)
Treatment emergent adverse events
Time frame: End of main phase treatment period to end of extension phase (1-year period)
Change in spasm frequency based on caregiver spasm/seizure diary
Number of clusters and mean cluster duration
Time frame: Baseline (1-week period) to end of treatment period (1-week period)
Change in spasm frequency based on 24-hour video-EEG
Number of clusters and mean cluster duration
Time frame: Baseline (1-week period) to end of treatment period (1-week period)
Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary
Number of clusters and mean cluster duration
Time frame: End of main phase treatment period to end of extension phase (1-year period)
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