Development and Validation of a Disability Severity Index for CMT (6604)
It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate (11-20) or severe (21-36). These terms are arbitrary. This study is looking to base mild, moderate, and severe on what both people affected with CMT and those who provide for people with CMT consider appropriate.
Charcot Marie Tooth Disease
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease|
- Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Develop a Disability Severity Index including items with high patient-physician concordance [ Time Frame: 1 year ] [ Designated as safety issue: No ]
|Study Start Date:||June 2011|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Health Care Provider
A person working within the field of CMT.
Patient with CMT
Any person of any age self-identifying as having CMT and belonging to the Inherited Neuropathies Consortium Contact Registry hosted by the Rare Disease Clinical Research Network.
|United States, Iowa|
|University of Iowa|
|Iowa City, Iowa, United States, 52242|
|Principal Investigator:||Sindhu Ramchandren, MD||Wayne State University|