A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington’s Disease
Completed
ABOUT
SPONSOR
Institut National de la Santé Et de la Recherche Médicale, France
PARTICIPANTS
100
In this trial a compound called Triheptanoin is tested to see if it has a positive impact on HD patients functions in all areas but with a special emphasis on cognitive functions. Trhiheptanoion is artificially produced and it is a fat. It is almost tasteless and colorless and is already used as medical food to treat certain diseases related to the bodies energy production, socalled metabolic disorders. We know that the brain cells energy production is negatively affected in HD. 100 participants is included in the trial. 50 will get Triheptanoin each day for 12 months. The other 50 participants will for the first 6 months receive placebo. But for the following 6 months they will get Trheptanoin. Potential effect will be tested with MRS to get an image of whether the energy production has improved or at least not decreased. MRI images will also be taken to see any effect on brain volume. In addition the participants will be tested with a battery of motor and cognitive tests as well as questionnaires related to general function and psychiatric status.
Estimated Study Completion Date: 31 December, 2020