Tricaprilin Infantile Spasms Pilot Study

NCT ID: NCT04727970

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-11-01
• Study Completion Date: 2023-12-04

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Tricaprilin

Intervention Type: DRUG

Tricaprilin will be emulsified in infant formula/milk

Interventions

Tricaprilin

Tricaprilin will be emulsified in infant formula/milk

Intervention Type: DRUG

Other Intervention Names

AC-OL-01

Eligibility 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

• Minimum Eligible Age: 3 Months
• Maximum Eligible Age: 36 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cerecin

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Cerecin

Locations

Sydney Children's Hospital

Randwick, New South Wales, Australia

Queensland Children's Hospital

South Brisbane, Queensland, Australia

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

KK Women's and Children's Hospital

Singapore, , Singapore

Countries

Australia; Singapore

Other Identifiers

AC-21-024

Identifier Type: -

Identifier Source: org_study_id