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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04727970




Registration number
NCT04727970
Ethics application status
Date submitted
19/01/2021
Date registered
27/01/2021
Date last updated
27/07/2023

Titles & IDs
Public title
Tricaprilin Infantile Spasms Pilot Study
Scientific 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
Secondary ID [1] 0 0
AC-21-024
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Infantile Spasm 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tricaprilin

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.


Treatment: Drugs: Tricaprilin
Tricaprilin will be emulsified in infant formula/milk
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Timepoint [1] 0 0
Up to end of study (Day 5 to 21; depending on subject)
Primary outcome [2] 0 0
To determine the safety and tolerability of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Timepoint [2] 0 0
Up to end of study (Day 5 to 21; depending on subject)
Primary outcome [3] 0 0
Extension Phase (for Australian sites only): To determine the safety of daily long-term administration of tricaprilin in subjects with Infantile Spasms (IS)
Timepoint [3] 0 0
End of main phase treatment period to end of extension phase (1-year period)
Secondary outcome [1] 0 0
Change in spasm frequency based on caregiver spasm/seizure diary
Timepoint [1] 0 0
Baseline (1-week period) to end of treatment period (1-week period)
Secondary outcome [2] 0 0
Change in spasm frequency based on 24-hour video-EEG
Timepoint [2] 0 0
Baseline (1-week period) to end of treatment period (1-week period)
Secondary outcome [3] 0 0
Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary
Timepoint [3] 0 0
End of main phase treatment period to end of extension phase (1-year period)

Eligibility
Key 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
Minimum age
3 Months
Maximum age
36 Months
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2025
Actual
Sample size
Target
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital Melbourne - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
Singapore
State/province [1] 0 0
Singapore

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Cerecin
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04727970
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Cerecin
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04727970