Patients with symptomatic and pre-symptomatic Huntington's disease
Conditions
Brief summary
The primary endpoint will be effect size, assessed by the standardized mean difference (Cohen's d) for each biomarker between the values measured initially and their assessment at 2-year follow-up.
Detailed description
Analysis of patient profiles and progression trajectories, taking into account all available data at D0, M1, M1 bis, M12, M24 and M24 bis, will be based on socio-demographic characteristics, as well as initial clinical and paraclinical scores and their evolution over time. Clustering analyses will use statistical validation indices to determine the optimal number of clusters and provide information on cluster quality., Identification of the best biomarkers predicting an unfavorable disease course will be carried out using conventional regression and machine learning methods. Discrimination and calibration performances will be systematically evaluated and compared for each of the models constructed.
Interventions
DRUG[18F]MNI-659
Sponsors
Assistance Publique Hopitaux De Paris
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint will be effect size, assessed by the standardized mean difference (Cohen's d) for each biomarker between the values measured initially and their assessment at 2-year follow-up. | — |
Secondary
| Measure | Time frame |
|---|---|
| Analysis of patient profiles and progression trajectories, taking into account all available data at D0, M1, M1 bis, M12, M24 and M24 bis, will be based on socio-demographic characteristics, as well as initial clinical and paraclinical scores and their evolution over time. Clustering analyses will use statistical validation indices to determine the optimal number of clusters and provide information on cluster quality., Identification of the best biomarkers predicting an unfavorable disease course will be carried out using conventional regression and machine learning methods. Discrimination and calibration performances will be systematically evaluated and compared for each of the models constructed. | — |
Countries
France
Outcome results
None listed