Status:
COMPLETED
Tricaprilin Infantile Spasms Pilot Study
Lead Sponsor:
Cerecin
Conditions:
Infantile Spasm
Eligibility:
All Genders
3-36 years
Phase:
PHASE1
Brief Summary
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
- Within 14 days of screening, subject has:
- received therapy with felbamate, cannabinoids, ketogenic diet, or vagus nerve stimulation
- received therapy with ACTH, prednisolone or other steroid
- Pre-existing lethal or potentially lethal condition other than infantile spasms
- Previous failure to respond to an appropriate trial (at least 2 weeks) of the ketogenic diet
Key Trial Info
Start Date :
November 1 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 4 2023
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT04727970
Start Date
November 1 2021
End Date
December 4 2023
Last Update
September 19 2024
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031
2
Queensland Children's Hospital
South Brisbane, Queensland, Australia, 4101
3
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
4
KK Women's and Children's Hospital
Singapore, Singapore, 229899