Innovative Imaging and Cognitive BIOmarkers to Predict Huntington's Disease Progression

Sponsor
Assistance Publique - Hôpitaux de Paris
Study ID
NCT05808153
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Accepted

Interventions

  • radiotracer injection — RADIATION
    MRI with radiotracer injection

Study Details

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.

Key Dates

First listed
Apr 11, 2023
Start date
Mar 21, 2024
Status verified
Jan 2024
Primary completion
Apr 2, 2025
Completion
Feb 2, 2027

Study Design

Enrollment
80 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
OTHER

Arms

  • Experimental: Symptomatic (MH) and pre-symptomatic (preMH) patients
    * 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
  • Active Comparator: Age-matched controls (healthy volunteers)
    * TFC functional UHDRS score = 13 * TMS engine UHDRS rating \< 6

Primary Outcome Measure

Genetic markers [ Time Frame: Visit Month 0 ]

Central Contacts

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