Tricaprilin Infantile Spasms Pilot Study

• Register: ClinicalTrials.gov
• Last refreshed on: 30 September 2024
• Main ID: NCT04727970
• Date of registration: 19/01/2021
• Prospective Registration: Yes
• Primary sponsor: Cerecin
• Public title: Tricaprilin Infantile Spasms Pilot Study
• Scientific title: A Phase I Open-Label Pilot Study to Investigate the Feasibility, Safety, Tolerability and Efficacy of Daily Administration of Tricaprilin in Subjects with Infantile Spasms
• Date of first enrolment: November 1, 2021
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04727970
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Australia, Singapore

Contacts

• Name: Study Director
• Affiliation: Cerecin

Key inclusion & exclusion criteria

Inclusion Criteria: 1. Male and female infants ages 3 months to 24 months, inclusive, at the time of parent/legal guardian signing the informed consent 2. Clinical diagnosis of IS, confirmed by analysis of a 24-hour video-electroencephalogram (vEEG) recording, including at least one documented spasm 3. Continued infantile spasms despite adequate treatment with oral prednisolone (or adrenocorticotropic hormone [ACTH]) and vigabatrin 4. 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. 5. Subject is taking no more than 3 concomitant ASDs Exclusion Criteria: 1. Subject considered by the Investigator, for any reason, to be an unsuitable candidate to receive the investigational product 2. Significant and active pre-existing cardiovascular, renal, liver, infectious, or other systemic disease 3. Subject has clinically significant renal impairment 4. Clinically significant abnormality on ECG that, in the opinion of the Investigator, increases the safety risks of participating in the study 5. Known or suspected allergy to the investigational product 6. Known history of aspiration pneumonia within the past year 7. 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 8. Within 14 days of screening, subject has: 1. received therapy with felbamate, cannabinoids, ketogenic diet, or vagus nerve stimulation 2. received therapy with ACTH, prednisolone or other steroid 9. Pre-existing lethal or potentially lethal condition other than infantile spasms 10. Previous failure to respond to an appropriate trial (at least 2 weeks) of the ketogenic diet


Age minimum: 3 Months
Age maximum: 36 Months
Gender: All

Health Condition(s) or Problem(s) studied

Infantile Spasm

Intervention(s)

Drug: Tricaprilin

Primary Outcome(s)

To determine the safety and tolerability of daily administration of tricaprilin in subjects with Infantile Spasms (IS) [Time Frame: Up to end of study (Day 5 to 21; depending on subject)]

To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS) [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) [Time Frame: End of main phase treatment period to end of extension phase (1-year period)]

Secondary Outcome(s)

Change in spasm frequency based on caregiver spasm/seizure diary [Time Frame: Baseline (1-week period) to end of treatment period (1-week period)]

Change in spasm frequency based on 24-hour video-EEG [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 [Time Frame: End of main phase treatment period to end of extension phase (1-year period)]

Secondary ID(s)

AC-21-024

Source(s) of Monetary Support

Please refer to primary and secondary sponsors