Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease (TRIHEP2)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01882062|
Recruitment Status : Completed
First Posted : June 20, 2013
Results First Posted : March 24, 2016
Last Update Posted : March 24, 2016
|Condition or disease||Intervention/treatment||Phase|
|Huntington Disease||Drug: Triheptanoin 1g/kg/day||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Study Start Date :||May 2013|
|Actual Primary Completion Date :||July 2013|
|Actual Study Completion Date :||July 2013|
Experimental: Triheptanoin 1g/kg/day
All patients received Triheptanoin oil at 1g/kg/day during 1 month
Drug: Triheptanoin 1g/kg/day
- Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr [ Time Frame: visit 1 (baseline), visit 2 (after 1 month of treatment) ]
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.
- Correlation Between Primary Outcome Measure and Clinical Parameters [ Time Frame: visit 1 (baseline), visit 2 (after 1 month of treatment) ]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).
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): NCT01882062
|Brain and Spine Institute|
|Paris, France, 75013|