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Clinical Trials in France / NCT02596191
Active, not recruiting Not applicable

Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers

NCT02596191 · tracked via the Priya Life Science France tracker
Sponsor
Assistance Publique Hopitaux De Marseille
Phase
Not applicable
Started
2016-06-06
Last updated
2025-06-27

Condition(s) studied

Charcot-Marie-Tooth Disease Type 1A

Investigational drug(s) / intervention(s)

Clinical evaluationelectrophysiological recordMuscle MRIblood samples analysis

Study summary

This is a 2-year follow-up study of a cohort of 60 CMT1A patients. The objective is to identify markers allowing to better understand the phenotypic variability observed on patients with CMT1A, to identify predictive markers of the disease's progression and to provide validated measurement tools that can be used as outcome measures in future clinical trials.

Eligibility

Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
Accepted
Inclusion Criteria: * Patients with CMT 1A disease * Diagnosis of CMT 1A confirmed by genotyping (duplication of the 17p11.2 region) Exclusion Criteria: * Patients suffering from co-morbidity at the origin of peripheral neuropathy (diabetes, hypothyroidism, renal insufficiency, drugs...) or muscle, articular, rheumatological disease * With HIV or cancer * With a significant progressive disease in the previous month * With a contra-indication for MRI * With a dislocation, fracture, or recent surgery (less than 6 months before inclusion) * with alcohol or psychoactive substances abuse * Treated by an anti-inflammatory drug over the past four weeks * Pregnant or breastfeeding women * Homeless patients

Primary outcome measure(s)

  • Change of functional scores — 3 months, 12 months and 24 months
    several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNDS)
  • Change of functional scores — 3 months, 12 months and 24 months
    several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNS)
  • Change of functional scores — 3 months, 12 months and 24 months
    several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNS2)
  • Change of functional scores at — 3 months, 12 months and 24 months
    several functional scores to evaluate the severity and the progression of the disease will be performed and compared (ONLS)

Trial sites (2)

FacilityCityRegionStatus
Assistance Publique Hôpitaux de Marseille Marseille France
CHU Gui de Chauliac, CHU MONTPELLIER Montpellier France

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Other trials for the same condition

Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease Recruiting
Clinical Trial Evaluating the Impact of an Intensive Rehabilitation Program Combined With Tendon Vibratory Stimulation on Functional Balance in Individuals With Charcot-Marie-Tooth Disease Type 1A Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02596191 on ClinicalTrials.gov ↗ ← All trials in France