Introduction
Methods
Trial design and participants
Inclusion criteria
Exclusion criteria
Interventions and outcomes
Baseline | One month review | Months 3,6,9,12,18 and 24 | 27 months | |
---|---|---|---|---|
Medical history, family history and physical examination | X | X | x | X |
Weight | X | X | x | X |
Vital signs (blood pressure and pulse/heart rate) | X | X | x | X |
Haematology | X | X | x | X |
Biochemistry | X | X | x | X |
12-Lead ECG | X | X | x | X |
Review of adverse events | X | X | x | X |
Blood sample for metabolomics | X | x | X | |
Review of concomitant medications | X | x | X | |
Magnetic resonance imaging (brain) | X | X | ||
Unified Huntington’s Disease Rating Scale | X | x | X | |
Mini mental State Examination | X | x | X | |
Neuropsychiatric Inventory | X | x | x | |
Trail making test | X | x | X | |
Verbal fluency | X | x | X | |
Tower of London (One Touch Stockings of Cambridge) | X | x | X | |
Extra-dimensional/intra-dimensional shifts | X | x | X |
Sample size and statistical methods
Imaging
Blood sampling and metabolomic analysis
Results
Recruitment
Baseline data
Sex | 11 male 5 female |
Age | Mean 53 (range 37–69) |
Weight | 80.2 kg (62.5–100) |
BMI | 27.5 (21.4–33.4) |
MMSE | 27.4 (24–30) |
Verbal fluency | 23.2 (6–53) |
UHDRS motor | 24.5 (7–46) |
UHDRS functional capacity | 9.62 (5–13) |
UHDRS functional assessment | 28.2 (25–35) |
UHDRS independence | 80 (70–100) |
Primary outcome measures
Secondary outcome measures
Variable | Units | Change per annum | Lower CI | Upper CI |
p value |
---|---|---|---|---|---|
UHDRS (motor) | Score | 3.5 | 0.8 | 6.3 | 0.009 |
OTS-latency to correct | Log s | − 0.12 | − 0.23 | − 0.01 | 0.014 |
UHDRS (total functional capacity) | Score | − 0.4 | − 0.8 | − 0.02 | 0.020 |
EDS errors | Number | − 0.2 | − 0.5 | 0.06 | 0.050 |
UHDRS (functional assessment). | Score | 0.7 | − 0.2 | 1.5 | 0.063 |
Trail B | Log s | 0.13 | − 0.06 | 0.32 | 0.081 |
UHDRS independence scale | Score | − 1.1 | − 2.9 | 0.6 | 0.100 |
Weight | kg | 0.6 | − 0.8 | 2.1 | 0.190 |
Verbal Fluency | Log value | − 0.04 | − 0.13 | 0.06 | 0.221 |
MMSE | Score | 0.1 | − 0.3 | 0.5 | 0.263 |
OTS problems solved on first choice | Log odds | 0.06 | − 0.18 | 0.31 | 0.300 |
Systolic blood pressure | mmHg | − 0.95 | − 5.09 | 3.16 | 0.318 |
Trail A | Log s | 0.01 | − 0.11 | 0.13 | 0.429 |
Pre ED errors | Log odds | 0.05 | − 0.70 | 0.78 | 0.455 |
Region | Percent change |
p value |
---|---|---|
Left lateral ventricle | 7.2 | <0.001 |
Right lateral ventricle | 6.6 | <0.001 |
Brain volume | − 1.3 | 0.001 |
Brain volume exc. ventricle | − 1.8 | 0.001 |
Left putamen | − 4.4 | 0.002 |
Right inferior lateral ventricle | 8.1 | 0.002 |
Brain stem | − 0.9 | 0.003 |
Left cerebellum cortex | − 0.9 | 0.003 |
Pons | − 0.8 | 0.003 |
Left inferior lateral ventricle | 9.6 | 0.005 |
Right cerebellum cortex | − 1.0 | 0.009 |
Total grey matter volume | − 1.5 | 0.016 |
Cerebral white matter volume | − 2.2 | 0.019 |
Subcort grey matter volume | − 1.2 | 0.02 |
Cerebral cortex volume | − 1.8 | 0.038 |
Midbrain | − 0.4 | 0.174 |
Right thalamus proper | − 1.4 | 0.195 |
Right putamen | − 2.1 | 0.282 |
Left amygdala | − 0.7 | 0.296 |
Left thalamus proper | − 0.9 | 0.328 |
Right amygdala | − 0.6 | 0.487 |
Medulla | 0.4 | 0.549 |
Right hippocampus | − 0.2 | 0.618 |
Left caudate | 0.8 | 0.621 |
Left accumbens area | − 1.2 | 0.75 |
Right caudate | 1.0 | 0.797 |
Right accumbens area | − 0.4 | 0.905 |
Left hippocampus | 0.0 | 0.957 |