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Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers
Sponsor: Assistance Publique Hopitaux De Marseille
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.
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
80
Start Date
2016-06-06
Completion Date
2027-12-05
Last Updated
2025-06-27
Healthy Volunteers
Yes
Conditions
Interventions
Clinical evaluation
electrophysiological record
Muscle MRI
blood samples analysis
Locations (2)
Assistance Publique Hôpitaux de Marseille
Marseille, France
CHU Gui de Chauliac, CHU MONTPELLIER
Montpellier, France