Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
31-Phosphorus Magnetic Resonance Spectroscopy |
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 |
3 months |
|
Primary |
volumetric magnetic resonance imaging |
A decrease in the rate of caudate atrophy, using volumetric MRI |
6 months |
|
Secondary |
motor function after 6 months |
Decrease in the progression of the UHDRS United Huntington's Disease Rating Scale after 6 months of treatment |
6 months |
|
Secondary |
motor function after 12 months |
Decrease in the progression of the UHDRS United Huntington's Disease Rating Scale after 12 months of treatment |
12 months |
|
Secondary |
Patient autonomy after 6 months |
Stability of the Total Functional Capacity (TFC) after 6 months of treatment |
6 months |
|
Secondary |
Patient autonomy after 12 months |
Stability of the Total Functional Capacity (TFC) after 12 months of treatment |
12 months |
|
Secondary |
Sustained restoration of brain energy metabolism after 6 months |
Sustained restoration of brain energy metabolism using 31P-MRS after 6 months of treatment |
6 months |
|
Secondary |
Sustained restoration of brain energy metabolism after 12 months |
Sustained restoration of brain energy metabolism using 31P-MRS after 12 months of treatment |
12 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
12 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
12 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
12 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
12 months |
|
Secondary |
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 |
3 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
9 months |
|
Secondary |
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 |
12 months |
|
Secondary |
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). |
6 months |
|
Secondary |
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 |
6 months |
|
Secondary |
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 |
12 months |
|
Secondary |
Number of adverse events |
Safety of triheptanoin will be evaluated based on review of adverse events |
12 months |
|
Secondary |
clinical exam for Long term tolerance |
Long-term tolerance will be confirmed by clinical exam at study visits |
12 months |
|
Secondary |
home nurse visits for Long term tolerance |
Long-term tolerance will be confirmed by patient report during home nurse visits |
12 months |
|