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Huntington DiseasePhase 2TRIHEP3NCT02453061

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

A Phase 2 study testing Triheptanoin oil in people with Huntington Disease, run by Institut National de la Santé Et de la Recherche Médicale, France at 2 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Huntington Disease
What is being tested?
Triheptanoin oil
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
100 participants
Where
2 sitesin 2 countries
Study dates
29 Jun 2015to 3 Dec 2019
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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

Trial locations and contacts

2 sites in 2 countries.

2 sites in 2 countries

France1 site

  • Département de Génétique

    Paris, 75013

Other trials in France

Netherlands1 site

  • Department of Neurology

    Leiden, 2300RC

Other trials in Netherlands

About this study

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.

Being tested
Triheptanoin oil, Placebo
Medical subject headings
Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Triheptanoin oilDrug

    Triheptanoin oil orally administered at 1g/kg/day

  • PlaceboDrug

    Safflower oil orally administered at 1g/kg/day

Study groups in NCT02453061
Study groupRoleWhat they receive
Placebo groupPlacebo comparatorSubjects will receive safflower oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12)
Receives: Drug: Placebo
Triheptanoin groupActive comparatorSubjects will receive triheptanoin oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12)
Receives: Drug: Triheptanoin oil

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. volumetric magnetic resonance imaging

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

    Measured: 6 months

Secondary outcomes (26)

  1. motor function after 6 months

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

    Measured: 6 months

  2. 31- Phosphorus Magnetic Resonance Spectroscopy

    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

    Measured: 3 months

  3. Caudal Atrophy

    Rate of caudate atrophy, using volumetric MRI

    Measured: 12 months

  4. motor function after 12 months

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

    Measured: 12 months

  5. Patient autonomy after 6 months

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

    Measured: 6 months

  6. Patient autonomy after 12 months

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

    Measured: 12 months

  7. Sustained restoration of brain energy metabolism after 6 months

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

    Measured: 6 months

  8. Sustained restoration of brain energy metabolism after 12 months

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

    Measured: 12 months

  9. Symbol Digit Modalities Test after 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

    Measured: 6 months

  10. Symbol Digit Modalities Test after 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

    Measured: 12 months

  11. Stroop test after 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

    Measured: 6 months

  12. Stroop test after 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

    Measured: 12 months

  13. Trail making test after 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

    Measured: 6 months

  14. Trail making test after 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

    Measured: 12 months

  15. Digit span test after 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

    Measured: 6 months

  16. Digit span test after 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

    Measured: 12 months

  17. psychiatric symptoms after 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

    Measured: 3 months

  18. psychiatric symptoms after 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

    Measured: 6 months

  19. psychiatric symptoms after 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

    Measured: 9 months

  20. psychiatric symptoms after 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

    Measured: 12 months

  21. patients' daily life

    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).

    Measured: 6 months

  22. quality of life questionnaire after 6 months

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

    Measured: 6 months

  23. quality of life questionnaire after 12 months

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

    Measured: 12 months

  24. Number of adverse events

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

    Measured: 12 months

  25. clinical exam for Long term tolerance

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

    Measured: 12 months

  26. home nurse visits for Long term tolerance

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

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 May 2015 (estimated)
  2. Study start29 Jun 2015
  3. Primary completion3 Dec 2019
  4. Study completion3 Dec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
100 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02453061
Sponsor's protocol ID
C14-62
Other IDs
2014-005112-42, 2014
Acronym
TRIHEP3
Status last verified
Jul 2024
First submitted
12 May 2015
Last updated
3 Jun 2026
Results posted
No
Data monitoring committee
No

Publications

  • Mochel F, Meneret A, Adanyeguh IM, Giron C, Hainque E, Luton MP, Atencio M, Debs R, Jacobs M, Veldkamp FCM, Barbier M, Coppen EM, van der Zwaan KF, Diallo A, Ottolenghi C, Vicaut E, Roos RA, Durr A. Effect of Triheptanoin on Caudate Atrophy and Motor Scores in Patients With Early-Stage Huntington Disease: A Phase II Study. Neurology. 2025 Jan 28;104(2):e210194. doi: 10.1212/WNL.0000000000210194. Epub 2024 Dec 30.

    PubMed 39804569

  • Rodrigues FB, Ferreira JJ, Wild EJ. Huntington's Disease Clinical Trials Corner: June 2019. J Huntingtons Dis. 2019;8(3):363-371. doi: 10.3233/JHD-199003.

    PubMed 31381524

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Triheptanoin oil in people with Huntington Disease, run by Institut National de la Santé Et de la Recherche Médicale, France at 2 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in France, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

volumetric magnetic resonance imaging (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02453061, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2026.

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