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

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

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

Brief 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.

Detailed Summary:
Sponsor: Assistance Publique Hopitaux De Marseille

Current Primary Outcome: Change of functional scores at 3 different times [ Time Frame: 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, CMTNDS, CMTNS2, ONLS)


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Assistance Publique Hopitaux De Marseille

Dates:
Date Received: October 30, 2015
Date Started: November 2015
Date Completion: November 2019
Last Updated: November 3, 2015
Last Verified: November 2015