Tricaprilin Phase 2 Pilot Study in Migraine

Study Overview
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Brief Summary
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This is a randomised, double-blind, placebo-controlled, parallel-group, multi-centre study of tricaprilin as AC-SD-03 compared with placebo for the reduction of migraine in participants with frequent migraine.

Official Title
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A 12-Week, Double-blind, Randomised, Placebo-controlled, Parallel Group Study to Investigate the Efficacy and Safety of Daily Administration of Tricaprilin as AC-SD-03 in Participants With Migraine

Conditions
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Migraine

Intervention / Treatment
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* Drug: Tricaprilin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant must be 18 to 70 years of age inclusive, at the time of signing the informed consent. Participants who have frequent (episodic or chronic) migraine with or without aura, for at least 1 year, according to the International Classification of Headache Disorders version 3 beta (ICHD 3-beta), age at time of onset must be <50 years. Participants must have a certain number of migraine headaches days per month, as confirmed by the baseline measurement period. Use of one allowed migraine prophylactic is permitted if the participant has been on a stable dose for at least 2 months prior to the screening. Participants must have failed (no therapeutic response) 1-4 migraine prophylactic treatments. The participant is able to tolerate a 12.5g dose of AC-SD-03 (containing 5g of tricaprilin) as per sentinel dose challenge at the Baseline Visit. From Visit 2 to Visit 3 (baseline measurement period), the participant has sufficient compliance (at least 80%) with daily eDiary headache entries. Exclusion Criteria: In the opinion of the Investigator, has presence or history of an advanced, severe, progressive, or unstable disease of any type that could interfere with efficacy and safety assessments, or put the participant at risk. Use of barbiturates (and/or butalbital-containing analgesics) or opioids (and/or opioid-containing analgesics) for migraine acute treatment ≥ 4 days per month on average and/or in the last month prior to Screening Visit. Use in the last 3 months prior to Screening Visit of CGRP agents, Botox injections, TENS, cranial nerve blocks, trigger-point injections, acupuncture, CBD-containing products, infusion therapy. Current use, or use within 3 months of Visit 2 (baseline), of Axona® or other MCT-containing products (such as coconut oil). Use of MCT-containing products is not allowed at any time during trial participation. Current use, or use within 3 months prior to Screening, of a ketogenic diet, low-carb diet, intermittent fasting (including the 5:2 diet). Ketogenic diets, low-carb diet, intermittent fasting (including the 5:2 diet) are not allowed during the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: AC-SD-03<br>Tricaprilin SD formulation, twice daily. Administered orally | Drug: Tricaprilin<br>* Powder formulation will be mixed with 240 mL water and shaken until fully dispersed. Each dosing unit of 12.5 g of AC-SD-03 contains 5 g of the active ingredients (tricaprilin)<br>* Other names: AC-SD-03;|
| Placebo Comparator: AC-SD-03P<br>Placebo formulation, twice daily. Administered orally | Drug: Placebo<br>* Powder formulation will be mixed with 240 mL water and shaken until fully dispersed.<br>* Other names: AC-SD-03P;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the number of migraine headache days during Month 3 (ΔMDMth3) | Number of migraine headache days using headache diary parameters | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the number of migraine headache days during Month 1 (ΔMDMth1) and Month 2 (ΔMDMth2) of treatment and overall, from Months 1-3 | Number of migraine headache days using headache diary parameters | 4, 8, 12 weeks | 
| The proportion of participants with a 50% reduction from baseline in number of migraine headache days in treatment months 1, 2, and 3 | Response rate defined as a reduction in number of migraine headache days | 4, 8, 12 weeks | 
| Change from baseline in monthly acute migraine medicine use during treatment months 1, 2 and 3 | Acute migraine medicine use during treatment | 4, 8, 12 weeks | 
| Change from baseline Headache Impact Test (HIT-6) score at the end of Months 1, 2 and 3 | Headache Impact Test (HIT-6) score. Total score range between 36-78 (higher score indicates a worse impact) | 4, 8, 12 weeks |