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A Registered Cohort Study on Charcot-Marie-Tooth Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04010188
Recruitment Status : Recruiting
First Posted : July 8, 2019
Last Update Posted : November 13, 2020
Information provided by (Responsible Party):
Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University

Brief Summary:
The aim of the study is to analyze the natural history data data from Charcot-Marie-Tooth disease and related disorders in China, to assess the clinical, genetic, epigenetic features of patients with Charcot-Marie-Tooth disease, and to optimize clinical management.

Condition or disease
Charcot-Marie-Tooth Disease

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 20 Years
Official Title: A Registered Observational Cohort Study of Charcot-Marie-Tooth Disease
Actual Study Start Date : July 1, 2019
Estimated Primary Completion Date : December 31, 2039
Estimated Study Completion Date : December 31, 2049

Primary Outcome Measures :
  1. The change of Charcot-Marie-Tooth Neuropathy Score (CMTNS) during 6 month [ Time Frame: Up to 6 months ]
    Charcot-Marie-Tooth Neuropathy Score (CMTNS) was first proposed and validated by Shy et al (Neurology, 2005). to provide a reliable measure of impairment in Charcot-Marie-Tooth (CMT). The CMTNS is composed of 9 items evaluating different functions related to the disease: 5 of impairment ('Sensory Symptoms', 'Pin Sensibility', 'Vibration', 'Strength Arms' and 'Strength Legs'), 2 of activity limitations ('Motor Symptoms Arms' and 'Motor Symptoms Legs') and 2 electrophysiological measures. Each item is scored from 0 to 4 and the total sum of the item scores provides a global measure of disease severity, with higher scores indicating worsening function.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
CMT patients who are diagnosed in the First Affiliated Hospital of Fujian Medical University.

Inclusion Criteria:

  • Patients with the clinical diagnosis of Charcot-Marie-Tooth disease
  • Genetic diagnosis of patients with Charcot-Marie-Tooth disease
  • Unrelated healthy controls

Exclusion Criteria:

  • Decline to participate.
  • Other peripheral neuropathy caused by trauma, immunity and toxicosis.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04010188

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Contact: Ning Wang, MD, PhD 13805015340 ext 13805015340

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First Affiliated Hospital of Fujian Medical University Recruiting
Fuzhou, China
Contact: Jin He, MD   
Principal Investigator: Ning Wang, MD, PhD         
Sub-Investigator: Jin He, MD         
Sponsors and Collaborators
Ning Wang, MD., PhD.
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Responsible Party: Ning Wang, MD., PhD., Professor, First Affiliated Hospital of Fujian Medical University Identifier: NCT04010188    
Other Study ID Numbers: MRCTA,ECFAH of FMU [2019] 192
First Posted: July 8, 2019    Key Record Dates
Last Update Posted: November 13, 2020
Last Verified: November 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Stomatognathic Diseases
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn