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

Primary Purpose

Huntington Disease

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Triheptanoin 1g/kg/day
Sponsored by
Institut National de la Santé Et de la Recherche Médicale, France
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Brain and Spine Institute

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Triheptanoin 1g/kg/day

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Correlation Between Primary Outcome Measure and Clinical Parameters
Correlating an improvement of brain energy profile with clinical parameters in Huntington patients such as the Unified Huntington's disease rating scale (UHDRS) and total functional capacity score (TFC).

Full Information

First Posted
June 18, 2013
Last Updated
February 24, 2016
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France
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1. Study Identification

Unique Protocol Identification Number
NCT01882062
Brief Title
Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
Acronym
TRIHEP2
Study Type
Interventional

2. Study Status

Record Verification Date
December 2013
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
July 2013 (Actual)
Study Completion Date
July 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Institut National de la Santé Et de la Recherche Médicale, France

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Triheptanoin 1g/kg/day
Arm Type
Experimental
Arm Description
All patients received Triheptanoin oil at 1g/kg/day during 1 month
Intervention Type
Drug
Intervention Name(s)
Triheptanoin 1g/kg/day
Primary Outcome Measure Information:
Title
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Description
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)
Secondary Outcome Measure Information:
Title
Correlation Between Primary Outcome Measure and Clinical Parameters
Description
Correlating an improvement of brain energy profile with clinical parameters in Huntington patients such as the Unified Huntington's disease rating scale (UHDRS) and total functional capacity score (TFC).
Time Frame
visit 1 (baseline), visit 2 (after 1 month of treatment)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Brain and Spine Institute
City
Paris
ZIP/Postal Code
75013
Country
France

12. IPD Sharing Statement

Citations:
PubMed Identifier
25568297
Citation
Adanyeguh IM, Rinaldi D, Henry PG, Caillet S, Valabregue R, Durr A, Mochel F. Triheptanoin improves brain energy metabolism in patients with Huntington disease. Neurology. 2015 Feb 3;84(5):490-5. doi: 10.1212/WNL.0000000000001214. Epub 2015 Jan 7.
Results Reference
result
Links:
URL
http://www.ncbi.nlm.nih.gov/pubmed/25568297
Description
Related Info

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

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