Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood

NCT02408354 | Completed Phase 2

• 1 other identifier: C14-53
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this project is to study the efficacy of triheptanoin oil in patients with Alternating Hemiplegia of Childhood (AHC) due to ATP1A3 gene mutation.

Conditions

Alternating Hemiplegia of Childhood

Outcome Measures

Primary Outcomes (1)

• Number of neurologic paroxystic events report in patient diary

  visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28

  7 months

Secondary Outcomes (6)

• Composite score allying the number of neurological paroxystic events, their duration and severity.

  7 months

• Clinical Global Impression Scales - Improvement

  7 months

• The Short Form (36) Health Survey

  7 months

• Brain 31phosphorus magnetic resonance spectroscopy

  7 months

• Clinical Safety as measured by questionnaire

  7 months

Study Arms (2)

Triheptanoin

ACTIVE COMPARATOR

Triheptanoin/ Placebo Randomized to receive active Triheptanoin first for 12 weeks. At cross-over, participants will receive placebo for 12 weeks. Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between triheptanoine and placebo phases.

Drug: Triheptanoin

Placebo

PLACEBO COMPARATOR

Placebo / Triheptanoin Randomized to receive active Placebo first for 12 weeks. At cross-over, participants will receive Triheptanoin for 12 weeks. Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between placebo and triheptanoin phases.

Drug: Placebo

Interventions

Placebo DRUG

Placebo is a oily liquid, intended for oral (PO) administration. Participants will be given approximately 1g/kg of Placebo divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose.

Placebo

Triheptanoin DRUG

Triheptanoin is a triglyceride composed of three heptanoate (C7 fatty acid) esters. Triheptanoin is manufactured by chemical synthesis from glycerol and heptanoic acid. Triheptanoin is a liquid, intended for oral (PO) administration. Participants will be given approximately 1g/kg of Triheptanoin divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose.

Also known as: UX007, glycerol triheptanoate

Triheptanoin

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• AHC with mutation in ATP1A3 gene
• Age ≥ 15 years and 3 months
• ≥ 6 neurological paroxystic events during the last 3 months prior to the beginning of the study
• No specific diet
• Covered by french social security
• Patients who freely agree to participate in this study and understand the nature, risks and benefits of this study and give their written informed consent. (In addition to the requirement for the consent of parents or the legal representative, adolescents can provide additional informed consent to participate in clinical trials)

You may not qualify if:

• Age \< 15 years and 3 months
• Evidence of psychiatric disorder
• Comorbid medical condition that would render them unsuitable for the study, e.g. HIV, diabetes
• Pregnant or parturient or lactating women
• Absence of double effective contraception at the women old enough to procreate
• Unwillingness to be informed in case of abnormal MRI
• Absence of signed informed consent
• No covered by french social security
• Persons deprived of their liberty by judicial or administrative decision

Sponsors & Collaborators

• Institut National de la Santé Et de la Recherche Médicale, France: lead

Study Sites (1)

Groupe hospitalier Pitié Salpêtrière

Paris, 75013, France

Location

Related Publications (1)

• Hainque E, Caillet S, Leroy S, Flamand-Roze C, Adanyeguh I, Charbonnier-Beaupel F, Retail M, Le Toullec B, Atencio M, Rivaud-Pechoux S, Brochard V, Habarou F, Ottolenghi C, Cormier F, Meneret A, Ruiz M, Doulazmi M, Roubergue A, Corvol JC, Vidailhet M, Mochel F, Roze E. A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood. Orphanet J Rare Dis. 2017 Oct 2;12(1):160. doi: 10.1186/s13023-017-0713-2.

  PMID: 28969699 RESULT

MeSH Terms

Conditions

Alternating hemiplegia of childhood

Interventions

triheptanoin

Study Officials

• Emmanuel Flamand-Roze, MD, PhD

  INSERM UMRS 975, 47 bd de l'hôpital - 75651 Paris Cedex 13

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2015
• First Posted: April 3, 2015
• Study Start: March 25, 2015
• Primary Completion: April 5, 2017
• Study Completion: April 5, 2017
• Last Updated: December 10, 2025

Record last verified: 2025-02

Locations

FR (1)