Tricaprilin Infantile Spasms Pilot Study

• ClinicalTrials.gov Identifier: NCT04727970
• Recruitment Status: Recruiting
• First Posted: January 27, 2021
• Last Update Posted: March 10, 2022
• Sponsor: Cerecin
• Information provided by (Responsible Party): Cerecin

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Infantile Spasm	Drug: Tricaprilin	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: November 1, 2021
• Estimated Primary Completion Date: March 2023
• Estimated Study Completion Date: March 2023

Arms and Interventions

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 Name: AC-OL-01

Outcome Measures

Primary Outcome Measures :

1. 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) ]
   Treatment emergent adverse events
2. 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) ]
   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.

Secondary Outcome Measures :

1. Change in spasm frequency based on caregiver spasm/seizure diary [ Time Frame: Baseline (1-week period) to end of treatment period (1-week period) ]
   Number of clusters and mean cluster duration
2. Change in spasm frequency based on 24-hour video-EEG [ Time Frame: Baseline (1-week period) to end of treatment period (1-week period) ]
   Number of clusters and mean cluster duration

Eligibility Criteria

• Ages Eligible for Study: 3 Months to 24 Months (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Contacts

Contact: Project Manager; +1 303 999 3700; clinical@cerecin.com

Locations

Australia, New South Wales

Sydney Children's Hospital; Recruiting

Randwick, New South Wales, Australia, 2031

Contact: Clinical Trials Project Manager (02) 9382 1111

Singapore

KK Women's and Children's Hospital; Not yet recruiting

Singapore, Singapore, 229899

Contact: Derrick Chan

Sponsors and Collaborators

Cerecin

Investigators

• Study Director: Study Director; Cerecin

More Information

• Responsible Party: Cerecin
• ClinicalTrials.gov Identifier: NCT04727970
• Other Study ID Numbers: AC-21-024
• First Posted: January 27, 2021
• Last Update Posted: March 10, 2022
• Last Verified: March 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• 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

Additional relevant MeSH terms:

Spasm; Spasms, Infantile; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Epileptic Syndromes