Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease (TRIHEP2)

Institut National de la Santé Et de la Recherche Médicale, France

Status and phase: Completed; Phase 2

Conditions: Huntington Disease

Treatments: Drug: Triheptanoin 1g/kg/day

Study type: Interventional

Funder types: Other

Identifiers

NCT01882062

C12-62

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Triheptanoin 1g/kg/day

Experimental group

Description:

All patients received Triheptanoin oil at 1g/kg/day during 1 month

Treatment:

Drug: Triheptanoin 1g/kg/day