martes, 3 de octubre de 2017

A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood | Orphanet Journal of Rare Diseases | Full Text

A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood | Orphanet Journal of Rare Diseases | Full Text

Biomed Central



Orphanet Journal of Rare Diseases

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

  • Elodie HainqueEmail author,
  • 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 and
  • Emmanuel Roze
Contributed equally
Orphanet Journal of Rare Diseases201712:160
Received: 6 April 2017
Accepted: 25 September 2017
Published: 2 October 2017

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.

Keywords

Alternating hemiplegia of childhood Triheptanoin Crossover trial

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