Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood (HEMIHEP)
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|ClinicalTrials.gov Identifier: NCT02408354|
Recruitment Status : Unknown
Verified March 2017 by Institut National de la Santé Et de la Recherche Médicale, France.
Recruitment status was: Active, not recruiting
First Posted : April 3, 2015
Last Update Posted : March 7, 2017
|Condition or disease||Intervention/treatment||Phase|
|Alternating Hemiplegia of Childhood||Drug: Triheptanoin Drug: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Study Start Date :||March 2015|
|Estimated Primary Completion Date :||March 2017|
|Estimated Study Completion Date :||December 2017|
Active Comparator: Triheptanoin
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.
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 Comparator: Placebo
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.
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.
- Number of neurologic paroxystic events report in patient diary [ Time Frame: 7 months ]visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
- Composite score allying the number of neurological paroxystic events, their duration and severity. [ Time Frame: 7 months ]visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
- Clinical Global Impression Scales - Improvement [ Time Frame: 7 months ]visit 2 at week 12, visit 4 at week 28
- The Short Form (36) Health Survey [ Time Frame: 7 months ]visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
- Brain 31phosphorus magnetic resonance spectroscopy [ Time Frame: 7 months ]Ratio of Inorganic Phosphate (Pi) over Phosphocreatine during visual stimulation visit 2 at week 12, visit 4 at week 28
- Clinical Safety as measured by questionnaire [ Time Frame: 7 months ]visit 2 at week 12, visit 4 at week 28
- Biological Safety as measured by acylcarnitine profile, organic acid dosage [ Time Frame: 7 months ]visit 2 at week 12, visit 4 at week 28
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02408354
|Groupe hospitalier Pitié Salpêtrière|
|Paris, France, 75013|
|Principal Investigator:||Emmanuel Flamand-Roze, MD, PhD||INSERM UMRS 975, 47 bd de l'hôpital - 75651 Paris Cedex 13|