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

Alternating Hemiplegia of Childhood Clinical Trial

— HEMIHEP  

Official title:

"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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02408354
• Other study ID #: C14-53
• Status: Completed
• Phase: Phase 2
• Start date: April 15, 2015
• Est. completion date: April 5, 2017

Study information

• Verified date: February 2020
• Source: Institut National de la Santé Et de la Recherche Médicale, France
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

The clinical spectrum of Alternating Hemiplegia of Childhood (AHC) is wide and characterized by the association of permanent and paroxysmal (palsy, dystonia, ocular, epileptic, dysautonomic events) neurological events, with onset in childhood. Most of AHC patients carry mutations in the ATP1A3 gene. This gene encodes the Na+/K+ ATPase witch is a transmembrane ion pump generating chemical and electrical gradient of sodium and potassium across the plasma membrane. Those paroxystic events in AHC patients with mutations in the ATP1A3 gene could be associated with a glucidic/energetic metabolism or intracerebral excitability disorder.

Triheptanoin is a triglyceride, whose derivatives pass the blood - brain barrier and enhance the Krebs cycle functions. Triheptanoin could therefore allow energy supply to the brain, which is essential for the functioning of the Na+/ K+ ATPase that consumes a significant amount of energy in the brain.

The investigators goal is to do a pilot study to test the effectiveness on paroxystic manifestations and the safety of triheptanoin in a small group of patients with Alternating Hemiplegia of Childhood secondary to ATP1A3 mutations.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: April 5, 2017
• Est. primary completion date: April 5, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years and older

Eligibility

Inclusion Criteria:

• 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)

Exclusion Criteria:

• 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

• Person subject to an exclusion period for another research

• Subjects with exclusion criteria required by french law

Related Conditions & MeSH terms

• Alternating Hemiplegia of Childhood
• Hemiplegia

Intervention

Drug:
• Triheptanoin
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.
• Placebo
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.

Locations

Country	Name	City	State
France	Groupe hospitalier Pitié Salpêtrière	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Composite score allying the number of neurological paroxystic events, their duration and severity.	visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28	7 months
Secondary	Clinical Global Impression Scales - Improvement	visit 2 at week 12, visit 4 at week 28	7 months
Secondary	The Short Form (36) Health Survey	visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28	7 months
Secondary	Brain 31phosphorus magnetic resonance spectroscopy	Ratio of Inorganic Phosphate (Pi) over Phosphocreatine during visual stimulation visit 2 at week 12, visit 4 at week 28	7 months
Secondary	Clinical Safety as measured by questionnaire	visit 2 at week 12, visit 4 at week 28	7 months
Secondary	Biological Safety as measured by acylcarnitine profile, organic acid dosage	visit 2 at week 12, visit 4 at week 28	7 months