Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study.
J Neurol Sci
; 464: 123147, 2024 Sep 15.
Article
em En
| MEDLINE
| ID: mdl-39094432
ABSTRACT
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).Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transtornos de Enxaqueca
Limite:
Adult
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
J Neurol Sci
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Austrália
País de publicação:
Holanda