A Registered Cohort Study on Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth Disease Clinical Trial

Official title:

A Registered Observational Cohort Study of Charcot-Marie-Tooth Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04010188
• Other study ID #: MRCTA,ECFAH of FMU [2019] 192
• Status: Recruiting
• Start date: July 1, 2019
• Est. completion date: December 31, 2049

Study information

• Verified date: January 2022
• Source: First Affiliated Hospital of Fujian Medical University
• Contact: Ning Wang, MD, PhD
• Phone: 13805015340
• Email: ningwang[@]fjmu.edu.cn
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: December 31, 2049
• Est. primary completion date: December 31, 2039
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Charcot-Marie-Tooth Disease
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes
• Tooth Diseases

Locations

Country	Name	City	State
China	First Affiliated Hospital of Fujian Medical University	Fuzhou

Sponsors (1)

Lead Sponsor	Collaborator
Ning Wang, MD., PhD.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The change of Charcot-Marie-Tooth Neuropathy Score (CMTNS) during 6 month	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.	Up to 6 months