Tricaprilin Infantile Spasms Pilot Study

Infantile Spasm Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04727970
• Other study ID #: AC-21-024
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: November 1, 2021
• Est. completion date: January 2025

Study information

• Verified date: July 2023
• Source: Cerecin
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 8
• Est. completion date: January 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months to 36 Months

Eligibility

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

Related Conditions & MeSH terms

• Infantile Spasm
• Spasm
• Spasms, Infantile

Intervention

Drug:
• Tricaprilin
Tricaprilin will be emulsified in infant formula/milk

Locations

Country	Name	City	State
Australia	Royal Children's Hospital Melbourne	Parkville	Victoria
Australia	Sydney Children's Hospital	Randwick	New South Wales
Australia	Queensland Children's Hospital	South Brisbane	Queensland
Singapore	KK Women's and Children's Hospital	Singapore

Sponsors (1)

Lead Sponsor	Collaborator
Cerecin

Countries where clinical trial is conducted

Australia,  Singapore, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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	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)
Secondary	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)
Secondary	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)