Tricaprilin Phase 2 Pilot Study in Migraine

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

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.

Study Overview

• Status: Completed
• Conditions: Migraine
• Intervention / Treatment: Drug: Tricaprilin; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 83
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • Adelaide, Australia
    • Calvary Adelaide Hospital
  • Blacktown, Australia
    • Paratus Clinical Research Western Sydney
  • Brisbane, Australia
    • Paratus Clinical Research Brisbane
  • Camberwell, Australia
    • Emeritus Research
  • Canberra, Australia
    • Paratus Clinical Research Canberra
  • Heidelberg, Australia
    • Austin Health Hospital
  • Melbourne, Australia
    • Alfred Health Hospital
  • Southport, Australia
    • Gold Coast University Hospital
  • New South Wales
    • Kanwal, New South Wales, Australia, 2259
      • Paratus Clinical Central Coast
    • Sydney, New South Wales, Australia, 2010
      • Holdsworth House

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participant must be 18 to 70 years of age inclusive, at the time of signing the informed consent.
2. 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.
3. Participants must have a certain number of migraine headaches days per month, as confirmed by the baseline measurement period.
4. 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.
5. Participants must have failed (no therapeutic response) 1-4 migraine prophylactic treatments.
6. 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.
7. From Visit 2 to Visit 3 (baseline measurement period), the participant has sufficient compliance (at least 80%) with daily eDiary headache entries.

Exclusion Criteria:

1. 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.
2. 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.
3. 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.
4. 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.
5. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: AC-SD-03; Tricaprilin SD formulation, twice daily. Administered orally	Drug: Tricaprilin; 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); Other Names: AC-SD-03
Placebo Comparator: AC-SD-03P; Placebo formulation, twice daily. Administered orally	Drug: Placebo; Powder formulation will be mixed with 240 mL water and shaken until fully dispersed.; Other Names: AC-SD-03P

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in the number of headache days (migraine and non-migraine headache) during treatment months 1, 2 and 3 (ΔHDMth1, ΔHDMth2 and ΔHDMth3)	Headache diary parameters	4, 8, 12 weeks
Change from baseline in MIDAS score at the end of Month 1, 2 and 3	Migraine Disability Assessment Scale (MIDAS) score. Total score range between 0-28 (higher score indicates more severe disability)	4, 8, 12 weeks

Collaborators and Investigators

• Sponsor: Cerecin
• Investigators: Study Director: Study Director, Cerecin

Study record dates

Study Major Dates

• Study Start (Actual): December 17, 2020
• Primary Completion (Actual): February 7, 2022
• Study Completion (Actual): February 7, 2022

Study Registration Dates

• First Submitted: June 11, 2020
• First Submitted That Met QC Criteria: June 16, 2020
• First Posted (Actual): June 18, 2020

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 7, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Headache Disorders, Primary; Headache Disorders; Migraine Disorders
• Other Study ID Numbers: AC-20-022

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No