A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington’s Disease




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



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

Ages Eligible for Study:

18 Years and older (Adult, Older Adult)

Sexes Eligible for Study:


Accepts Healthy Volunteers:


Inclusion Criteria

  • Positive genetic test with CAG repeat length ≥39 in HTT gene
  • At least 18 years of age
  • Signature of informed consent
  • Covered by social security
  • UHDRS score between 5 and 40
  • Ability to undergo MRI scanning
  • BMI between 18 and 30

Exclusion Criteria

  • Hypersensitivity to triheptanoin or to one of its excipients
  • Additional major comorbidities
  • History of severe head injury
  • Participation in another therapeutic trial (3 month exclusion period)
  • For women of childbearing age, the absence of two forms of effective contraception (with the exception of those who are abstinent)
  • Pregnancy or breastfeeding
  • Inability to understand information about the protocol
  • Persons deprived of their liberty by judicial or administrative decision
  • Adult subject under legal protection or unable to consent
  • Treatment with tetrabenazine



Département de Génétique

Address:Paris, France, 75013

Active, Not Recruiting


Department of Neurology

Address: Leiden, Netherlands, 2300RC

Active, Not Recruiting