Related Experiment Video
Updated: Jun 18, 2025
3D-Neuronavigation In Vivo Through a Patient's Brain During a Spontaneous Migraine Headache
Published on: June 2, 2014
Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind,
Lilian Chow1, Julia Presanis1, Nikki McIntyre1
1Cerecin Australia Pty Ltd, Australia.
Background:
Increasing evidence indicates a metabolic etiology for migraines, with ketosis potentially rectifying metabolic and clinical features. We conducted a pilot study to evaluate CER-0001, a ketogenic agent, for migraine prevention without dietary changes.
Methods:
This was a 2-part, double-blind, randomised, placebo-controlled study conducted in Australia. Adults with at least a 1-year history of migraine and ≥ 1 prior preventive treatment failure were randomised to either oral CER-0001 (up to 30 g twice a day) or placebo for 12 weeks. The primary endpoint was Month 3 change in Migraine Headache Days from baseline.
Results:
Part 1 results are presented. 81 participants were randomised and dosed (n = 40 CER-0001, n = 41 placebo), and 61 participants had evaluable efficacy data. No statistically significant difference was observed in the primary endpoint (LSMean difference 0.92 days; p = 0.586). During Month 2, a mean improvement of -2.8 days was observed for CER-0001 (p = 0.056). Withdrawal rates were 45.0% and 53.7% (CER-0001; placebo). The proportion of participants reporting at least one treatment-emergent adverse event was similar between arms (90.0% CER-0001, 82.9% placebo), mostly gastrointestinal (85.0% CER-0001, 70.7% placebo).
Conclusion:
Results suggest positive directional promise over 2-3 months for CER-0001. A new formulation will be used for larger, fully powered phase 2/3 studies.
Trial Registration:
This study is registered at ClinicalTrials.gov (NCT04437199).
Insights
This pilot study explored CER-0001, a ketogenic agent, for migraine prevention. While not statistically significant, early results showed promise for reducing migraine headache days.
Area of Science:
- Neurology
- Metabolic Disorders
Background:
- Migraine may have a metabolic origin, with ketosis showing potential to improve symptoms.
- CER-0001 is a ketogenic agent investigated for migraine prevention.
- This pilot study aimed to assess CER-0001 without requiring dietary changes.
Purpose of the Study:
- To evaluate the efficacy of CER-0001 in preventing migraines.
- To assess the safety and tolerability of CER-0001 in migraine patients.
Main Methods:
- A 2-part, double-blind, randomized, placebo-controlled study.
- Adults with a history of migraine and prior treatment failure were randomized.
- Participants received oral CER-0001 or placebo for 12 weeks, with the primary endpoint being the change in migraine headache days.
Main Results:
- 81 participants were randomized; 61 had evaluable efficacy data.
- No statistically significant difference in the primary endpoint was observed between CER-0001 and placebo.
- A trend towards improvement in migraine headache days was noted for CER-0001 during Month 2 (p=0.056).
- Adverse events were primarily gastrointestinal and similar between groups.
Conclusions:
- Results indicate potential positive directional effects of CER-0001 over 2-3 months.
- Further investigation in larger, powered phase 2/3 studies is warranted.
- A new formulation of CER-0001 will be utilized for future trials.
