Clinical Studies
6604: Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
Status: Closed to Accrual
Study Summary
Please Note: The Rare Diseases Clinical Research Network will make every effort to enroll all the patients we can, but we cannot make any guarantees that we will be able to enroll everyone in a particular study who wants to participate.
Background
CMT affects people’s feet and legs, and often people will need to use ambulation aids, such as orthotics, braces, or wheelchairs, in order to get around. While researchers have looked at some of these aids as a way to measure how severe a person’s CMT is, people with CMT have not been asked what ambulation aids they consider to be severe. In this study, we are asking both patients with CMT and researchers who study CMT to rate the severity of disability, depending on type of ambulation aid used, in their opinion.
The research questions are:
- How do patients rate the severity of disability, depending on type of ambulation aid used?
- How do researchers rate the severity of disability, depending on type of ambulation aid used?
- How do the patient and researchers’ opinions compare?
About this Study
This is a one-time study of about 200 individuals with either CMT or researchers of CMT. Twenty-five people will be randomly selected to take the survey a second time two to four weeks following the first survey. Patients with CMT will be sent the survey electronically if they have signed up for the Inherited Neuropathies Consortium Contact Registry through the Rare Disease Clinical Research Network homepage. Researchers will be given the survey in paper form at the CMT Consortium meeting in Potomac, MD. Patients between the ages of 8 and 100 years and researchers of all ages are eligible for this survey. For the patients, we are requesting the opinions of those affected with CMT and not parents or guardians.
Targeted Enrollment
To be eligible to participate, you must:
- Be an individual with CMT who has joined the INC RDCRN Contact Registry.
- Be a researcher of CMT attending the 4th International CMT Consortium meeting in Potomac, MD.
You are not eligible to participate if:
- You do not read or speak English.
Join the Contact Registry for: Charcot Marie Tooth disease (CMT)
