Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease

Phase 2CompletedNCT01882062

Institut National de la Santé Et de la Recherche Médicale, France10 enrolled

Overview

The purpose of this project is to study the efficacy of an anaplerotic treatment on brain energy profile evolution at an early stage of the Huntington disease.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Huntington Disease

Interventions

Triheptanoin 1g/kg/dayDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 5 \< UHDRS \< 50 * Age \> 18 years * Ability to undergo MR scanning * Covered by french social security Exclusion Criteria: * Evidence of psychiatric disorder * Attendant neurological disorder * Contraindications to MRI (claustrophobia, metallic or material implants) * Severe head injury * Unable to understand the protocol * Pregnancy * Failure to give informed consent * Subjects with exclusion criteria required by french law (e.g. subjects who require a legally authorized representative to obtain consent) * Unwillingness to be informed in case of abnormal MRI

Locations (1)

Brain and Spine Institute

Paris, France

Outcomes

Primary Outcomes

Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr

The Pi/PCr Ratio is a measure of brain metabolism and it is an index of mitochondrial oxidative regulation. A 6-cm 31P transmit/receive surface coil (RAPID Biomedical GmbH, Rimpar, Germany) was used to collect free induction decays for 4 minutes at rest, 8 minutes during visual activation with 6-Hz red/black checkerboard flashes, and 8 minutes after stimulation. Subjects were able to focus on the flashes with a nonmagnetic mirror mounted above their eyes while all lights in the room were turned off. The Pi/PCr ratio was then calculated to determine brain response to cortical activation.

Time frame: visit 1 (baseline), visit 2 (after 1 month of treatment)

Data from ClinicalTrials.gov

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