Infantile SpasmPhase 1NCT04727970
Tricaprilin Infantile Spasms Pilot Study
A Phase 1 study testing Tricaprilin in people with Infantile Spasm, run by Cerecin at 4 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 3 to 36 months
- Conditions the study is for
- Infantile Spasm
- What is being tested?
- Tricaprilin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 1 Nov 2021to 4 Dec 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Australia3 sites
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Sydney Children's Hospital
Randwick, New South Wales, 2031
Queensland Children's Hospital
South Brisbane, Queensland, 4101
About this 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
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
- Being tested
- Tricaprilin
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TricaprilinDrug
Also called AC-OL-01
Tricaprilin will be emulsified in infant formula/milk
| Study group | Role | What they receive |
|---|---|---|
| Tricaprilin | Experimental | 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. Receives: Drug: Tricaprilin |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety of daily administration of tricaprilin in subjects with Infantile Spasms (IS)
Treatment emergent adverse events
Measured: Up to end of study (Day 5 to 21; depending on subject)
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.
Measured: Up to end of study (Day 5 to 21; depending on subject)
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
Measured: End of main phase treatment period to end of extension phase (1-year period)
Secondary outcomes (3)
Change in spasm frequency based on caregiver spasm/seizure diary
Number of clusters and mean cluster duration
Measured: Baseline (1-week period) to end of treatment period (1-week period)
Change in spasm frequency based on 24-hour video-EEG
Number of clusters and mean cluster duration
Measured: Baseline (1-week period) to end of treatment period (1-week period)
Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary
Number of clusters and mean cluster duration
Measured: End of main phase treatment period to end of extension phase (1-year period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2021
- Study start1 Nov 2021
- Primary completion4 Dec 2023
- Study completion4 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile SpasmStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cerecin
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Cerecin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04727970
- Sponsor's protocol ID
- AC-21-024
- Status last verified
- Apr 2024
- First submitted
- 19 Jan 2021
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04727970
- Acronym / sponsor ID
- AC-21-024
- Conditions
- Infantile Spasm
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- CerecinIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2021 (actual)
- Primary completion
- 4 Dec 2023 (actual)
- Study completion
- 4 Dec 2023 (actual)
- First posted
- 27 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tricaprilin | Experimental | Drug: TricaprilinTricaprilin 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. |
Who may qualify
- Ages
- 3 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TricaprilinAlso: AC-OL-01Tricaprilin will be emulsified in infant formula/milk | Drug | Tricaprilin |
Primary outcome
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
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.
Extension Phase (for Australian sites only): To determine the safety of daily long-term administration of tricaprilin in subjects with Infantile Spasms (IS)
Time frame: End of main phase treatment period to end of extension phase (1-year period)
Treatment emergent adverse events
3 secondary outcomes
Change in spasm frequency based on caregiver spasm/seizure diary
Time frame: Baseline (1-week period) to end of treatment period (1-week period)
Change in spasm frequency based on 24-hour video-EEG
Time frame: Baseline (1-week period) to end of treatment period (1-week period)
Extension Phase (for Australian sites only): Change in spasm frequency based on caregiver spasm/seizure diary
Time frame: End of main phase treatment period to end of extension phase (1-year period)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Cerecin
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tricaprilin in people with Infantile Spasm, run by Cerecin at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia, Singapore. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.