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NCT Number: NCT02453061

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

In the study the investigators plan to include 100 early affected HD patients (5 ≤ UHDRS ≤ 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a control oil (n = 50) at 1g/kg of body weight per day for 6 months followed by an open label phase using triheptanoin for another 6 months. Efficacy of triheptanoin will be evaluated by measurements of caudate volume using volumetric magnetic resonance imaging. Clinical improvement will be evaluated by brain energy metabolism quantification as evaluated by the ratio of inorganic phosphate/phosphocreatine, during visual stimulation, using 31P-MRS, UHDRS, TFC, and PBA-S scores as well as performance on the neuropsychological battery. Patient quality of life will be evaluated with the SF-36 questionnaire before and after treatment; biological tolerance and compliance will be evaluated by routine biochemical blood tests, plasma and urine measurements of triheptanoin oil derivatives and patient report.

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Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Département de Génétique, Paris, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Triheptanoin oil

Drug

Triheptanoin oil orally administered at 1g/kg/day

Placebo

Drug

Safflower oil orally administered at 1g/kg/day

Primary outcomes

  1. volumetric magnetic resonance imaging

    Time frame: 6 months

    A decrease in the rate of caudate atrophy, using volumetric MRI

Secondary outcomes

  1. motor function after 6 months

    Time frame: 6 months

    Decrease in the progression of the UHDRS United Huntington's Disease Rating Scale after 6 months of treatment

  2. 31- Phosphorus Magnetic Resonance Spectroscopy

    Time frame: 3 months

    An increase in the index of brain energy restoration - as defined by the difference between Pi/PCr ration during visual stimulation and the mean of the Pi/PCr ratio during rest and recovery - using 31P-MRS

  3. Caudal Atrophy

    Time frame: 12 months

    Rate of caudate atrophy, using volumetric MRI

  4. motor function after 12 months

    Time frame: 12 months

    Decrease in the progression of the UHDRS United Huntington's Disease Rating Scale after 12 months of treatment

  5. Patient autonomy after 6 months

    Time frame: 6 months

    Stability of the Total Functional Capacity (TFC) after 6 months of treatment

  6. Patient autonomy after 12 months

    Time frame: 12 months

    Stability of the Total Functional Capacity (TFC) after 12 months of treatment

  7. Sustained restoration of brain energy metabolism after 6 months

    Time frame: 6 months

    Sustained restoration of brain energy metabolism using 31P-MRS after 6 months of treatment

  8. Sustained restoration of brain energy metabolism after 12 months

    Time frame: 12 months

    Sustained restoration of brain energy metabolism using 31P-MRS after 12 months of treatment

  9. Symbol Digit Modalities Test after 6 months

    Time frame: 6 months

    The benefit of triheptanoin on cognitive function will be evaluated after 6 months using a neuropsychological battery including the SDMT (Symbol Digit Modalities Test), a test of visuomotor coordination

  10. Symbol Digit Modalities Test after 12 months

    Time frame: 12 months

    The benefit of triheptanoin on cognitive function will be evaluated after 12 months using a neuropsychological battery including the SDMT (Symbol Digit Modalities Test), a test of visuomotor coordination

  11. Stroop test after 6 months

    Time frame: 6 months

    The benefit of triheptanoin on cognitive function will be evaluated after 6 months using a neuropsychological battery including the Stroop test, a test evaluating concentration and capacity for inhibition

  12. Stroop test after 12 months

    Time frame: 12 months

    The benefit of triheptanoin on cognitive function will be evaluated after 12 months using a neuropsychological battery including the Stroop test, a test evaluating concentration and capacity for inhibition

  13. Trail making test after 6 months

    Time frame: 6 months

    The benefit of triheptanoin on cognitive function will be evaluated after 6 months using a neuropsychological battery including the Trail making test, a test to evaluate mental flexibility

  14. Trail making test after 12 months

    Time frame: 12 months

    The benefit of triheptanoin on cognitive function will be evaluated after 12 months using a neuropsychological battery including the Trail making test, a test to evaluate mental flexibility

  15. Digit span test after 6 months

    Time frame: 6 months

    The benefit of triheptanoin on cognitive function will be evaluated after 6 months using a neuropsychological battery including the Digit span test, a test evaluating attention and working memory

  16. Digit span test after 12 months

    Time frame: 12 months

    The benefit of triheptanoin on cognitive function will be evaluated after 12 months using a neuropsychological battery including the Digit span test, a test evaluating attention and working memory

  17. psychiatric symptoms after 3 months

    Time frame: 3 months

    The effect of triheptanoin on psychiatric symptoms will be evaluated after 3 months with the PBA-S, an evaluation of problem behaviors associated with HD

  18. psychiatric symptoms after 6 months

    Time frame: 6 months

    The effect of triheptanoin on psychiatric symptoms will be evaluated after 6 months with the PBA-S, an evaluation of problem behaviors associated with HD

  19. psychiatric symptoms after 9 months

    Time frame: 9 months

    The effect of triheptanoin on psychiatric symptoms will be evaluated after 9 months with the PBA-S, an evaluation of problem behaviors associated with HD

  20. psychiatric symptoms after 12 months

    Time frame: 12 months

    The effect of triheptanoin on psychiatric symptoms will be evaluated after 12 months with the PBA-S, an evaluation of problem behaviors associated with HD

  21. patients' daily life

    Time frame: 6 months

    The global impact of triheptanoin on patients' daily life will be evaluated at the end of the blinded treatment period using qualitative research methods. To statistically test whether experts classify treated and not treated patients better than could be expected by chance, a permutation test based on a modified version of Fisher's Lady tasting tea procedure will be used (Fischer 1935).

  22. quality of life questionnaire after 6 months

    Time frame: 6 months

    A standardized quality of life questionnaire, the Short Form-36 Health Survey, will be used after 6 months

  23. quality of life questionnaire after 12 months

    Time frame: 12 months

    A standardized quality of life questionnaire, the Short Form-36 Health Survey, will be used after 12 months

  24. Number of adverse events

    Time frame: 12 months

    Safety of triheptanoin will be evaluated based on review of adverse events

  25. clinical exam for Long term tolerance

    Time frame: 12 months

    Long-term tolerance will be confirmed by clinical exam at study visits

  26. home nurse visits for Long term tolerance

    Time frame: 12 months

    Long-term tolerance will be confirmed by patient report during home nurse visits

Sponsors and collaborators

Lead sponsor

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

Other Gov

Collaborators

  • Ultragenyx Pharmaceutical Inc

Registry information

Acronym: TRIHEP3

Important dates

Study start
2015
Primary completion
2019
Study completion
2019
First posted
May 25, 2015
Registry last updated
Jun 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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