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

• Register: ClinicalTrials.gov
• Last refreshed on: 7 September 2021
• Main ID: NCT02453061
• Date of registration: 12/05/2015
• Prospective Registration: Yes
• Primary sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Public title: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease TRIHEP3
• Scientific title: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
• Date of first enrolment: June 29, 2015
• Target sample size: 100
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02453061
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France, Netherlands

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Huntington Disease

Intervention(s)

Drug: Triheptanoin oil

Drug: Placebo

Primary Outcome(s)

volumetric magnetic resonance imaging [Time Frame: 6 months]

31-Phosphorus Magnetic Resonance Spectroscopy [Time Frame: 3 months]

Secondary Outcome(s)

patients' daily life [Time Frame: 6 months]

psychiatric symptoms after 6 months [Time Frame: 6 months]

Stroop test after 6 months [Time Frame: 6 months]

Sustained restoration of brain energy metabolism after 12 months [Time Frame: 12 months]

home nurse visits for Long term tolerance [Time Frame: 12 months]

Patient autonomy after 6 months [Time Frame: 6 months]

Patient autonomy after 12 months [Time Frame: 12 months]

Symbol Digit Modalities Test after 6 months [Time Frame: 6 months]

psychiatric symptoms after 3 months [Time Frame: 3 months]

quality of life questionnaire after 6 months [Time Frame: 6 months]

clinical exam for Long term tolerance [Time Frame: 12 months]

Digit span test after 12 months [Time Frame: 12 months]

motor function after 12 months [Time Frame: 12 months]

Trail making test after 12 months [Time Frame: 12 months]

motor function after 6 months [Time Frame: 6 months]

Number of adverse events [Time Frame: 12 months]

Sustained restoration of brain energy metabolism after 6 months [Time Frame: 6 months]

Digit span test after 6 months [Time Frame: 6 months]

psychiatric symptoms after 9 months [Time Frame: 9 months]

psychiatric symptoms after 12 months [Time Frame: 12 months]

Symbol Digit Modalities Test after 12 months [Time Frame: 12 months]

Stroop test after 12 months [Time Frame: 12 months]

quality of life questionnaire after 12 months [Time Frame: 12 months]

Trail making test after 6 months [Time Frame: 6 months]

Secondary ID(s)

C14-62

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ultragenyx Pharmaceutical Inc