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出境医 / 临床实验 / A Registered Cohort Study on Charcot-Marie-Tooth Disease

A Registered Cohort Study on Charcot-Marie-Tooth Disease

Study Description
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

Study Design
Layout table for study information
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
Arms and Interventions
Outcome Measures
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.


Eligibility Criteria
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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.
Criteria

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.
Contacts and Locations

Contacts
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Contact: Ning Wang, MD, PhD 13805015340 ext 13805015340 ningwang@fjmu.edu.cn

Locations
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China
First Affiliated Hospital of Fujian Medical University Recruiting
Fuzhou, China
Contact: Jin He, MD       hejinfimu@hotmail.com   
Principal Investigator: Ning Wang, MD, PhD         
Sub-Investigator: Jin He, MD         
Sponsors and Collaborators
Ning Wang, MD., PhD.
Tracking Information
First Submitted Date July 4, 2019
First Posted Date July 8, 2019
Last Update Posted Date November 13, 2020
Actual Study Start Date July 1, 2019
Estimated Primary Completion Date December 31, 2039   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 6, 2019) 		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.
Original Primary Outcome Measures
 (submitted: July 4, 2019) 		Charcot-Marie-Tooth Neuropathy Score (CMTNS) [ Time Frame: Up to 6 months ]
CMTNS is a validated measure of length-dependent axonal and demyelinating CMT disability.
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Registered Cohort Study on Charcot-Marie-Tooth Disease
Official Title A Registered Observational Cohort Study of Charcot-Marie-Tooth Disease
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.
Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 20 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population CMT patients who are diagnosed in the First Affiliated Hospital of Fujian Medical University.
Condition Charcot-Marie-Tooth Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 4, 2019) 		500
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 31, 2049
Estimated Primary Completion Date December 31, 2039   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers Yes
Contacts
Contact: Ning Wang, MD, PhD 13805015340 ext 13805015340 ningwang@fjmu.edu.cn
Listed Location Countries China
Removed Location Countries  
 
Administrative Information
NCT Number NCT04010188
Other Study ID Numbers MRCTA,ECFAH of FMU [2019] 192
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University
Study Sponsor Ning Wang, MD., PhD.
Collaborators Not Provided
Investigators Not Provided
PRS Account First Affiliated Hospital of Fujian Medical University
Verification Date November 2020

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