Evidence map›Paper›PMID 28969699›Full record

Trial reportOrphanet journal of rare diseases2017

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

Elodie Hainque, Samantha Caillet, Sandrine Leroy, Constance Flamand-Roze, Isaac Adanyeguh, Fanny Charbonnier-Beaupel, Maryvonne Retail, Benjamin Le Toullec, Mariana Atencio, Sophie Rivaud-Péchoux and 12 more

2 registry-linked trialsOpen access · goldAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.2field-weighted citation impact, top 21% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02408354 phase2completednot on this map

"Etude Pilote, Comparative, Monocentrique, randomisée, en Cross Over, en Double Aveugle, Contre Placebo, Testant l'efficacité de l'Huile triheptanoïne Dans Les Hémiplégies Alternantes de l'Enfant" HEMIHEP

TypeinterventionalSponsorInstitut National de la Santé Et de la Recherche Médicale, FranceRan2015 to 2017Enrolled10ConditionsAlternating Hemiplegia of ChildhoodArmsTriheptanoin, Placebo
NCT04513002 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation

TypeinterventionalSponsorThe University of QueenslandRan2022 to 2023Enrolled30ConditionsAtaxia TelangiectasiaArmsTriheptanoin
3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 27 citations in OpenAlex.

  1. No effect of triheptanoin on exercise performance in McArdle disease.Annals of clinical and translational neurology · 2019
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

22 authors at 8 institutions in 1 country.

Elodie HainqueUniversité 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.
Samantha CailletService de Diététique, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France.
Sandrine LeroyEpiScience, London, UK.
Constance Flamand-RozeCentre Hospitalier Sud-Francilien, Université Paris Sud, Corbeil-Essonnes, Service de Neurologie et Unité Neurovasculaire, Corbeil-Essonnes, France.
Isaac AdanyeguhUniversité 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.
Fanny Charbonnier-BeaupelPharmacie, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France.
Maryvonne RetailINSERM, Centre d'Investigation Clinique Neurosciences, CIC-1422, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France.
Benjamin Le ToullecINSERM, Centre d'Investigation Clinique Neurosciences, CIC-1422, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France.
Mariana AtencioUniversité 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.
Sophie Rivaud-PéchouxUniversité 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.
Vanessa BrochardINSERM, Centre d'Investigation Clinique Neurosciences, CIC-1422, Hôpital Pitié-Salpêtrière, AP-HP, Paris, France.
Florence HabarouService de Biochimie Métabolomique et protéomique, Hôpital Necker et Université Paris Descartes, AP-HP, Paris, France.
Chris OttolenghiService de Biochimie Métabolomique et protéomique, Hôpital Necker et Université Paris Descartes, AP-HP, Paris, France.
Florence CormierUniversité 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.
Aurélie MéneretUniversité 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.
Marta RuizUniversité 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.
Mohamed DoulazmiSorbonne Universités, UPMC Paris 06, CNRS UMR8256, Institut de Biologie Paris Seine, Adaptation Biologique et vieillissement, Paris, France.
Anne RoubergueDépartement de Neurologie, Hôpital Saint-Antoine, AP-HP, Paris, France.
Jean-Christophe CorvolUniversité 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.
Marie VidailhetUniversité 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.
Fanny MochelUniversité 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.
Emmanuel RozeUniversité 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.
Institut du Cerveau · FRInserm · FRAssistance Publique – Hôpitaux de Paris · FRSorbonne Université · FRCentre National de la Recherche Scientifique · FRPitié-Salpêtrière Hospital · FRCentre de Neurophysique Physiologie et Pathologie · FRUniversité Paris Cité · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBased 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.

methodsWe 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.

resultsIn 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.

conclusionsTriheptanoin does not prevent paroxysmal events in Alternating hemiplegia of childhood. We show the feasibility of a randomized placebo-controlled trial in this setting.

trial registrationThe study has been registered with clinicaltrials.gov ( NCT002408354 ) the 03/24/2015.

Indexed as

AdolescentCross-Over StudiesDietary SupplementsDouble-Blind MethodFemaleHemiplegiaHumansMaleTriglyceridesYoung AdultTriglyceridestriheptanoinAlternating hemiplegia of childhoodCrossover trialTriheptanoin

Identifiers

PMID28969699
PMCPMC5625655
OpenAlexW2761617604

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.