NCT05808153 Innovative Imaging and Cognitive BIOmarkers to Predict Huntington's Disease Progression
| NCT ID | NCT05808153 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Huntington Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 80 participants |
| Start Date | 2024-03-21 |
| Primary Completion | 2025-04-02 |
Trial Parameters
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Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * For all participants: * Age ≥18 years and ≤65 years * Information and collection of written consent * Affiliation with a social security plan, beneficiary or beneficiary's right * Healthy controls * UHDRS functional score TFC = 13 * Motor UHDRS score TMS \< 6 With no known genetic disease and no direct relationship to an HD patient or family ancestors carrying the HD mutation (or knowing their genetic status with CAG \< 36). * Manifest carriers * Number of GACs ≥ 40 * CAP score ≥ 250 * 10 ≤ TFC ≤ 13 * TMS \>5 if TFC=13 * Diagnostic confidence level =4 * Age of onset of disease \> 20 years * Patients physically able to sign consent * Premanifest carriers * Number of GACs ≥ 40 * CAP score ≥250 * CFT = 13 * TMS \< 6 * Patients physically able to sign consent Exclusion Criteria: * Participant under guardianship or curatorship * Neurological or psychiatric disorder unrelated to HD * Intercurrent illness that may impact participant's performance * Chronic progressive ne