Tricaprilin Infantile Spasms Pilot Study

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

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 Overview

• Status: Active, not recruiting
• Conditions: Infantile Spasm
• Intervention / Treatment: Drug: Tricaprilin

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Project Manager; Phone Number: +1 303 999 3700; Email: clinical@cerecin.com

Study Locations

• Australia
  • New South Wales
    • Randwick, New South Wales, Australia, 2031
      • Sydney Children's Hospital
  • Queensland
    • South Brisbane, Queensland, Australia, 4101
      • Queensland Children's Hospital
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Royal Children's Hospital Melbourne
• Singapore
  • Singapore, Singapore, 229899
    • KK Women's and Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 months to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tricaprilin; 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; Tricaprilin will be emulsified in infant formula/milk; Other Names: AC-OL-01

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in spasm frequency based on caregiver spasm/seizure diary	Number of clusters and mean cluster duration	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	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	End of main phase treatment period to end of extension phase (1-year period)

Collaborators and Investigators

• Sponsor: Cerecin
• Investigators: Study Director: Study Director, Cerecin

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2021
• Primary Completion (Estimated): January 1, 2025
• Study Completion (Estimated): January 1, 2025

Study Registration Dates

• First Submitted: January 19, 2021
• First Submitted That Met QC Criteria: January 25, 2021
• First Posted (Actual): January 27, 2021

Study Record Updates

• Last Update Posted (Actual): July 27, 2023
• Last Update Submitted That Met QC Criteria: July 25, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Neurologic Manifestations; Neuromuscular Manifestations; Epilepsy; Spasms, Infantile; Spasm
• Other Study ID Numbers: AC-21-024

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No