Title |
A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood
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Published in |
Orphanet Journal of Rare Diseases, October 2017
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DOI | 10.1186/s13023-017-0713-2 |
Pubmed ID | |
Authors |
Elodie Hainque, Samantha Caillet, Sandrine Leroy, Constance Flamand-Roze, Isaac Adanyeguh, Fanny Charbonnier-Beaupel, Maryvonne Retail, Benjamin Le Toullec, Mariana Atencio, Sophie Rivaud-Péchoux, Vanessa Brochard, Florence Habarou, Chris Ottolenghi, Florence Cormier, Aurélie Méneret, Marta Ruiz, Mohamed Doulazmi, Anne Roubergue, Jean-Christophe Corvol, Marie Vidailhet, Fanny Mochel, Emmanuel Roze |
Abstract |
Based on the hypothesis of a brain energy deficit, we investigated the safety and efficacy of triheptanoin on paroxysmal episodes in patients with alternating hemiplegia of childhood due to ATP1A3 mutations. We conducted a randomized, double-blind, placebo-controlled crossover study of triheptanoin, at a target dose corresponding to 30% of daily calorie intake, in ten patients with alternating hemiplegia of childhood due to ATP1A3 mutations. Each treatment period consisted of a 12-week fixed-dose phase, separated by a 4-week washout period. The primary outcome was the total number of paroxysmal events. Secondary outcomes included the number of paroxysmal motor-epileptic events; a composite score taking into account the number, severity and duration of paroxysmal events; interictal neurological manifestations; the clinical global impression-improvement scale (CGI-I); and safety parameters. The paired non-parametric Wilcoxon test was used to analyze treatment effects. In an intention-to-treat analysis, triheptanoin failed to reduce the total number of paroxysmal events (p = 0.646), including motor-epileptic events (p = 0.585), or the composite score (p = 0.059). CGI-I score did not differ between triheptanoin and placebo periods. Triheptanoin was well tolerated. Triheptanoin does not prevent paroxysmal events in Alternating hemiplegia of childhood. We show the feasibility of a randomized placebo-controlled trial in this setting. The study has been registered with clinicaltrials.gov ( NCT002408354 ) the 03/24/2015. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 45 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 7 | 16% |
Student > Master | 6 | 13% |
Other | 3 | 7% |
Researcher | 3 | 7% |
Student > Postgraduate | 2 | 4% |
Other | 5 | 11% |
Unknown | 19 | 42% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 10 | 22% |
Nursing and Health Professions | 5 | 11% |
Neuroscience | 2 | 4% |
Biochemistry, Genetics and Molecular Biology | 2 | 4% |
Pharmacology, Toxicology and Pharmaceutical Science | 1 | 2% |
Other | 2 | 4% |
Unknown | 23 | 51% |