Charcot-Marie-Tooth DiseaseNCT04010188
A Registered Cohort Study on Charcot-Marie-Tooth Disease
An observational study of Charcot-Marie-Tooth Disease, run by Ning Wang, MD., PhD. at 1 site in China.
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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Charcot-Marie-Tooth Disease
- How many people
- 500 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2019to 31 Dec 2049
- How long will I take part?
- 20 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: CMT patients who are diagnosed in the First Affiliated Hospital of Fujian Medical University.
Who may be able to join
- Patients with the clinical diagnosis of Charcot-Marie-Tooth disease
- Genetic diagnosis of patients with Charcot-Marie-Tooth disease
- Unrelated healthy controls
Who cannot join
- Decline to participate.
- Other peripheral neuropathy caused by trauma, immunity and toxicosis.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Fujian Medical University
Fuzhou
Jin He, MD, hejinfimu@hotmail.com
Ning Wang, MD, PhD
About this study
Official title: A Registered Observational Cohort Study of Charcot-Marie-Tooth Disease
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.
- Conditions
- Charcot-Marie-Tooth Disease
- Medical subject headings
- Charcot-Marie-Tooth Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2019
- Study start1 Jul 2019
- Primary completion31 Dec 2039 (estimated)
- Study completion31 Dec 2049 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
- Target duration
- 20 Years
Sponsor and investigators
- Lead sponsor
- Ning Wang, MD., PhD.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04010188
- Sponsor's protocol ID
- MRCTA,ECFAH of FMU [2019] 192
- Status last verified
- Jan 2022
- First submitted
- 4 Jul 2019
- Last updated
- 11 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Registered Observational Cohort Study of Charcot-Marie-Tooth Disease
- ClinicalTrials.gov ID
- NCT04010188
- Acronym / sponsor ID
- MRCTA,ECFAH of FMU [2019] 192
- Conditions
- Charcot-Marie-Tooth Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Ning Wang, MD., PhD.University, hospital or other
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- 31 Dec 2039 (estimated)
- Study completion
- 31 Dec 2049 (estimated)
- First posted
- 8 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 20 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- CMT patients who are diagnosed in the First Affiliated Hospital of Fujian Medical University.
Key inclusion criteria (3 listed)
- Patients with the clinical diagnosis of Charcot-Marie-Tooth disease
- Genetic diagnosis of patients with Charcot-Marie-Tooth disease
- Unrelated healthy controls
Key exclusion criteria (2 listed)
- Decline to participate.
- Other peripheral neuropathy caused by trauma, immunity and toxicosis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Charcot-Marie-Tooth Disease, run by Ning Wang, MD., PhD. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ning Wang, MD, PhD (13805015340 ext. 13805015340, ningwang@fjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of Charcot-Marie-Tooth Neuropathy Score (CMTNS) during 6 month (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jan 2022.