Tricaprilin Infantile Spasms Pilot Study

NCT04727970 | Completed Phase 1

• 1 other identifier: AC-21-024
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 4

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.

Conditions

Infantile Spasm

Outcome Measures

Primary Outcomes (3)

• To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS)

  Treatment emergent adverse events

  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.

  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

  End of main phase treatment period to end of extension phase (1-year period)

Secondary Outcomes (3)

• Change in spasm frequency based on caregiver spasm/seizure diary

  Baseline (1-week period) to end of treatment period (1-week period)

• Change in spasm frequency based on 24-hour video-EEG

  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

  End of main phase treatment period to end of extension phase (1-year period)

Study Arms (1)

Tricaprilin

EXPERIMENTAL

Tricaprilin will be administered for 5-21 days. The total daily dose (individual per subject up to a maximum of 10g/kg/day) will be split into 4 doses administered orally, approximately every 6 hours.

Drug: Tricaprilin

Interventions

Tricaprilin DRUG

Tricaprilin will be emulsified in infant formula/milk

Also known as: AC-OL-01

Tricaprilin

Eligibility Criteria

• Age: 3 Months - 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cerecin: lead

Study Sites (4)

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

Location

Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia

Location

Royal Children's Hospital Melbourne

Parkville, Victoria, 3052, Australia

Location

KK Women's and Children's Hospital

Singapore, 229899, Singapore

Location

MeSH Terms

Conditions

Spasms, Infantile

Interventions

tricaprylin

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Study Officials

• Study Director

  Cerecin

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2021
• First Posted: January 27, 2021
• Study Start: November 1, 2021
• Primary Completion: December 4, 2023
• Study Completion: December 4, 2023
• Last Updated: September 19, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); SG (1)