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A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood.

Université de la Sorbonne, UPMC Paris 06, UMR S 1127, Inserm U 1127, CNRS UMR 7225, Institut du Cerveau et de la Moëlle, F-75013, Paris, France. ehainque@gmail.com. Département de Neurologie, Groupe Hospitalier Pitié-Salpêtrière, AP-HP, 75013, Paris, France. ehainque@gmail.com. INSERM, Centre d'Investigation Clinique Neurosciences, CIC-1422, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France. ehainque@gmail.com. Service de Diététique, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France. EpiScience, London, UK. Centre Hospitalier Sud-Francilien, Université Paris Sud, Corbeil-Essonnes, Service de Neurologie et Unité Neurovasculaire, Corbeil-Essonnes, France. IFPPC, centre CAMKeys, Paris, France. Université de la Sorbonne, UPMC Paris 06, UMR S 1127, Inserm U 1127, CNRS UMR 7225, Institut du Cerveau et de la Moëlle, F-75013, Paris, France. Pharmacie, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France. INSERM, Centre d'Investigation Clinique Neurosciences, CIC-1422, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France. Service de Biochimie Métabolomique et protéomique, Hôpital Necker et Université Paris Descartes, AP-HP, Paris, France. Département de Neurologie, Groupe Hospitalier Pitié-Salpêtrière, AP-HP, 75013, Paris, France. Sorbonne Universités, UPMC Paris 06, CNRS UMR8256, Institut de Biologie Paris Seine, Adaptation Biologique et vieillissement, Paris, France. Département de Neurologie, Hôpital Saint-Antoine, AP-HP, Paris, France. Département de Génétique, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France. Groupe de Recherche Clinique Neurométabolique, Université Pierre et Marie Curie, Paris, France.

Orphanet journal of rare diseases. 2017;(1):160
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Abstract

BACKGROUND 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. METHODS 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. RESULTS 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. CONCLUSIONS Triheptanoin does not prevent paroxysmal events in Alternating hemiplegia of childhood. We show the feasibility of a randomized placebo-controlled trial in this setting. TRIAL REGISTRATION The study has been registered with clinicaltrials.gov ( NCT002408354 ) the 03/24/2015.

Methodological quality

Publication Type : Randomized Controlled Trial

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MeSH terms : Hemiplegia ; Triglycerides