Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common hereditary peripheral neuropathy, affecting approximately 26,000 patients in France. It presents as chronic and progressive sensorimotor deficits predominantly affecting the distal lower limbs, with onset typically in childhood. There is currently no specific pharmacological treatment; management remains symptomatic.
This research will:
In the long run, validated wearable sensors could improve patient follow-up, personalize rehabilitation, and support the design of clinical trials for CMT1A - including trials of the novel "Nano-Cur" treatment currently under development.
Age ≥ 18 years
Genetically confirmed diagnosis of CMT1A (PMP22 duplication on chromosomal analysis)
Followed at the National Reference Centre for Rare Peripheral Neuropathies (Service de Neurologie, CHU de Limoges) and/or having undergone gait analysis at the Quantified Movement Analysis Laboratory (Laboratoire d'AQM), Service de Médecine Physique et de Réadaptation, CHU de Limoges
Ability to walk independently (with or without walking aids)
Other associated neurological condition that could independently affect walking or motor activity
Inability to wear the sensor device (skin allergy, sensory intolerance)
Inability to comply with study procedures (cognitive impairment, no fixed domicile)
Participation in another interventional study during the same period
Adult patients diagnosed with genetically confirmed CMT1A (PMP22 duplication) who are followed at the National Reference Centre for Rare Peripheral Neuropathies (Service de Neurologie, CHU de Limoges) and/or who have undergone gait analysis at the Quantified Movement Analysis Laboratory (Laboratoire d'AQM), Service de Médecine Physique et de Réadaptation, CHU de Limoges.
Cohort
Other
Not listed
Pearson/Spearman r: daily step count (ActiGraph) vs. CMT-FOM total score
Correlation coefficient (r : -1 to +1) between mean daily step count (steps/day) over 7 days and CMT-FOM total score obtained at Day 1 hospital visit.
Pearson/Spearman r: daily step count (ActiGraph) vs. CMT-FOM total score
Correlation coefficient (r : -1 to +1) between mean daily step count (steps/day) over 7 days and CMT-FOM total score obtained at Day 1 hospital visit.
Pearson/Spearman r: daily activity counts (ActiGraph) vs. CMT-FOM total score
Correlation coefficient (r : -1 to +1) between mean daily activity counts (raw accelerometry counts/day) over 7 days and CMT-FOM total score.
Pearson/Spearman r: daily activity counts (ActiGraph) vs. CMT-FOM total score
Correlation coefficient (r : -1 to +1) between mean daily activity counts (raw accelerometry counts/day) over 7 days and CMT-FOM total score.
Intraclass Correlation Coefficient (ICC) of daily step count across 7 days
Test-retest reliability (ICC (0 to 1), two-way mixed, absolute agreement) of daily step count measured by ActiGraph LEAP across 7 home monitoring days.
Coefficient of Variation (CV, %) of daily activity counts across 7 days
Day-to-day variability expressed as CV (%) for raw accelerometry activity counts across 7 days.
Minimal Detectable Change (MDC) of daily step count
MDC95 of daily step count (steps/day), calculated from SEM and ICC, representing the smallest change exceeding measurement error.
Pearson r: daily step count vs. CMT Neuropathy Score (CMT-NS)
Correlation (r : -1 to +1) between mean daily steps/day and CMT-NS total score at Day 1.
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University Hospital, Limoges
Lead sponsor
Limoges University
Collaborator
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