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Tricaprilin Infantile Spasms Pilot Study

Primary Purpose

Infantile Spasm

Status

Active

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

Tricaprilin

About this trial

This is an interventional treatment trial for Infantile Spasm

Eligibility Criteria

3 Months - 36 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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:
1. received therapy with felbamate, cannabinoids, ketogenic diet, or vagus nerve stimulation
2. 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

Sites / Locations

Sydney Children's Hospital
Queensland Children's Hospital
Royal Children's Hospital Melbourne
KK Women's and Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Tricaprilin

Arm Description

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.

Outcomes

Primary Outcome Measures

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

Treatment emergent adverse events

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.

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

Secondary Outcome Measures

Change in spasm frequency based on caregiver spasm/seizure diary

Number of clusters and mean cluster duration

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

Number of clusters and mean cluster duration

Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary

Number of clusters and mean cluster duration

Full Information

NCT ID

NCT04727970

First Posted

January 19, 2021

Last Updated

July 25, 2023

Sponsor

Cerecin

1. Study Identification

Unique Protocol Identification Number

NCT04727970

Brief Title

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

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

November 1, 2021 (Actual)

Primary Completion Date

January 2025 (Anticipated)

Study Completion Date

January 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cerecin

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

5. 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. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Infantile Spasm

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Tricaprilin

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Tricaprilin

Other Intervention Name(s)

AC-OL-01

Intervention Description

Tricaprilin will be emulsified in infant formula/milk

Primary Outcome Measure Information:

Title

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

Description

Treatment emergent adverse events

Time Frame

Up to end of study (Day 5 to 21; depending on subject)

Title

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

Description

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.

Time Frame

Up to end of study (Day 5 to 21; depending on subject)

Title

Extension Phase (for Australian sites only): To determine the safety of daily long-term administration of tricaprilin in subjects with Infantile Spasms (IS)

Description

Treatment emergent adverse events

Time Frame

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

Secondary Outcome Measure Information:

Title

Change in spasm frequency based on caregiver spasm/seizure diary

Description

Number of clusters and mean cluster duration

Time Frame

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

Title

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

Description

Number of clusters and mean cluster duration

Time Frame

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

Title

Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary

Description

Number of clusters and mean cluster duration

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Months

Maximum Age & Unit of Time

36 Months

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Cerecin

Official's Role

Study Director

Facility Information:

Facility Name

Sydney Children's Hospital

City

Randwick

State/Province

New South Wales

ZIP/Postal Code

2031

Country

Australia

Facility Name

Queensland Children's Hospital

City

South Brisbane

State/Province

Queensland

ZIP/Postal Code

4101

Country

Australia

Facility Name

Royal Children's Hospital Melbourne

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3052

Country

Australia

Facility Name

KK Women's and Children's Hospital

City

Singapore

ZIP/Postal Code

229899

Country

Singapore

12. IPD Sharing Statement

Plan to Share IPD

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Tricaprilin Infantile Spasms Pilot Study

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