Clinical trial NCT06516016: Multimodal Markers of Neurodegenerative Disorders at Presymptomatic Stages
Official title: Multimodal Markers of Neurodegenerative Disorders at Presymptomatic Stages
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Neurodegenerative Diseases
Lead sponsor: Paris Brain Institute (ICM)
Sponsor class: OTHER
First posted: 2024-07-23
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Maximum age (years): 95
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will focus on subjects potentially at high risk of developing a degenerative disease, compared to a control population of healthy volunteers and relatives with no evidence of genetic mutation under study, radiologically isolated syndrome (RIS), Idiopathic rapid eye-movement (REM) sleep behavior disorder (iRBD) and abnormal elevated levels of protein ptau217.

Brief summary: NeuroPrems is a prospective, monocentric, longitudinal, not relating to a medicinal product for human use, non-randomized, non-controlled research. The study mainly aims to identify longitudinal changes and events in multimodal markers of neurodegeneration and neuroinflammation during the presymptomatic phases of neurodegenerative diseases.


Eligibility criteria: 1. Inclusion Criteria :

   1. For all participants :

      * Male or female
      * Age ≥ 18 years
      * Signed Informed consent by the subject
      * Affiliated with a social security system or beneficiary of such a regime
      * Ability to undergo an MRI exam with Gadobutrol
   2. For presymptomatic participants only :

      * Absence of a diagnosis of neurodegenerative disease. And at least one of the criteria :
      * Known carrier or relative of a patient carrying a mutation in a causal or at-risk responsible for a neurodegenerative disease
      * Isolated REM sleep behavioral disorder
      * Radiological isolated syndrome
      * Abnormal brain protein aggregates
   3. For controls only :

      * Absence of symptoms or diagnosis of neurodegenerative disease (or criteria corresponding to at risk group)
2. Exclusion Criteria :

   * Clinical symptoms fulfilling the criteria of a neurodegenerative disease (see table in criteria section)
   * Refusal of blood draw or brain MRI
   * MRI contraindication (see criteria section)
   * Known allergy to gadoteric acid
   * Unwillingness to be informed in case of abnormal MRI (with a significant medical anomaly)
   * Pregnancy or breastfeeding. For women in fertile age a urine pregnancy test will be performed before the MRI.
   * Inability to understand information about the protocol
   * Person deprived of their liberty by judicial or administrative decision
   * Person under legal protection (legal guardianship, tutelage or maintenance of justice)
   * Person without any protection and unable to consent
   * Other medical, neurological, psychiatric, or social conditions that in the investigator's opinion are likely to interfere with study conduct.

Primary outcomes:
measure: Rate of change of clinical, neurophysiological, anatomical and molecular marker profiles of neurodegenerescence and neuroinflammation in presymptomatic individuals
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
Secondary outcomes:
measure: Intra-individual change in trajectory profiles determined by multimodal analysis
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
measure: Rate and mean time to symptomatic conversion/progression
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
measure: Mean time of onset in genetically presymptomatic individuals
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
measure: Rate of change for each study marker
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
measure: Rate of change in the self-administered questionnaires
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
measure: Rate of change in consumption of information issued from Consultations, hospitalizations and medication prescriptions
timeFrame: From Year -10 to Year 10 (annual update)
measure: Time to loss of autonomy
timeFrame: Baseline, Year 1, Year 2, Year 3, Year 4 and Year 5
Other outcomes:
Locations:
city: Paris
country: France
facility: Pitié Salpêtrière Hospital
Study officials:
References: