An interventional study of radiotracer injection in Huntington Disease, sponsored by Assistance Publique - Hôpitaux de Paris. Recruiting at 1 site in France. Open to participants aged 18 Years to 65 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2024-05-07.
Sponsored by Assistance Publique - Hôpitaux de Paris · Not applicable, Interventional, and Other
Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort data, 2) predict disease entry in carriers of the HD mutation (during the prodromal phase or in patients considered asymptomatic: pre-HD patients), and 3) measure a significant evolution of the state of pre-HD patients over a time window compatible with the realization of clinical trials (about 2/3 years). Moreover, the markers of HD do not allow a fine stratification of the patients.
Hypothesis/Objective Our objectives are 1) to evaluate the sensitivity of new markers and assessment tools for symptomatic (HD) and presymptomatic (pre-HD) patients, 2) to define a model of disease progression, and 3) to establish an enrichment strategy to improve patient selection for future therapeutic trials.
Method We will evaluate newly developed cognitive tests, multimodal imaging techniques, biological markers and use innovative statistical approaches.
We will follow 60 patients with the mutation responsible for MH (40 presymptomatic pre-MH patients, 20 symptomatic MH patients) and 20 healthy volunteers (controls) over a 24-month period.
285 studies on the registry are indexed under Huntington Disease; 49 are open to participants now.
This study's planned enrollment of 80 is above the median of 40 across 203 interventional studies indexed under Huntington Disease.
Browse Huntington Disease studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
* Number of GAC ≥ 40 * GAP score ≥ 250 * 10 ≤ TFC ≤ 13 * TMS \>5 if TFC=13 * Diagnostic confidence level =4 * Age onset of the disease \> 20 years * Patients in physical capacity to sign the consent
Radiation: radiotracer injection
* TFC functional UHDRS score = 13 * TMS engine UHDRS rating \< 6
Radiation: radiotracer injection
MRI with radiotracer injection
Genetic markers
Time frame: Visit Month 0
cognitive tests
Cognitive scores - Neurological scores- Psychiatric scores
Time frame: Visits Month 0, Month 1, Month 12, Month 24
biological markers
Neuroinflammation markers in blood - Neurodegeneration markers in blood
Time frame: Visits Month 0, Month 1, Month 12, Month 24
multimodal imaging techniques
MRI
Time frame: Visits Month 0, Month 12, Month 24
multimodal imaging techniques
PET/MRI
Time frame: Visits Month 0, Month 24
Plan to share: Undecided
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Assistance Publique - Hôpitaux de Paris