Clinical trial NCT04727970: Tricaprilin Infantile Spasms Pilot Study
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Infantile Spasm
Lead sponsor: Cerecin
Sponsor class: INDUSTRY
First posted: 2021-01-27
Enrollment: 8
Sex: ALL
Minimum age (years): 3
Maximum age (years): 36
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 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.


Eligibility 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

Primary outcomes:
measure: 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)
measure: 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)
measure: 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)
Secondary outcomes:
measure: Change in spasm frequency based on caregiver spasm/seizure diary
timeFrame: Baseline (1-week period) to end of treatment period (1-week period)
measure: Change in spasm frequency based on 24-hour video-EEG
timeFrame: Baseline (1-week period) to end of treatment period (1-week period)
measure: Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary
timeFrame: End of main phase treatment period to end of extension phase (1-year period)
Other outcomes:
Locations:
city: Randwick
country: Australia
facility: Sydney Children's Hospital
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Parkville
country: Australia
facility: Royal Children's Hospital Melbourne
state: Victoria
city: Singapore
country: Singapore
facility: KK Women's and Children's Hospital
Study officials:
affiliation: Cerecin
name: Study Director
role: STUDY_DIRECTOR
References: