A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease (TRIHEP3)
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ClinicalTrials.gov Identifier: NCT02453061 |
Recruitment Status :
Completed
First Posted : May 25, 2015
Last Update Posted : August 25, 2021
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Condition or disease | Intervention/treatment | Phase |
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Huntington Disease | Drug: Triheptanoin oil Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 100 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease |
Actual Study Start Date : | June 29, 2015 |
Actual Primary Completion Date : | December 3, 2019 |
Actual Study Completion Date : | December 3, 2019 |
Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo group
Subjects 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)
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Drug: Placebo
Safflower oil orally administered at 1g/kg/day |
Active Comparator: Triheptanoin group
Subjects 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)
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Drug: Triheptanoin oil
Triheptanoin oil orally administered at 1g/kg/day |
- 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 ratio during visual stimulation and the mean of Pi/PCr ratio during rest and recovery - using 31P-MRS
- volumetric magnetic resonance imaging [ Time Frame: 6 months ]A decrease in the rate of caudate atrophy, using volumetric MRI
- 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
- 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
- Patient autonomy after 6 months [ Time Frame: 6 months ]Stability of the Total Functional Capacity (TFC) after 6 months of treatment
- Patient autonomy after 12 months [ Time Frame: 12 months ]Stability of the Total Functional Capacity (TFC) after 12 months of treatment
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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).
- 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
- 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
- Number of adverse events [ Time Frame: 12 months ]Safety of triheptanoin will be evaluated based on review of adverse events
- clinical exam for Long term tolerance [ Time Frame: 12 months ]Long-term tolerance will be confirmed by clinical exam at study visits
- home nurse visits for Long term tolerance [ Time Frame: 12 months ]Long-term tolerance will be confirmed by patient report during home nurse visits
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02453061
France | |
Département de Génétique | |
Paris, France, 75013 | |
Netherlands | |
Department of Neurology | |
Leiden, Netherlands, 2300RC |
Responsible Party: | Institut National de la Santé Et de la Recherche Médicale, France |
ClinicalTrials.gov Identifier: | NCT02453061 |
Other Study ID Numbers: |
C14-62 |
First Posted: | May 25, 2015 Key Record Dates |
Last Update Posted: | August 25, 2021 |
Last Verified: | August 2021 |
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Genetic Diseases, Inborn Cognition Disorders Neurocognitive Disorders Mental Disorders Dementia Chorea Dyskinesias |