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Development and Validation of a Disability Severity Index for CMT

Descriptive Information
Brief Title † Development and Validation of a Disability Severity Index for CMT
Official Title † Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
Brief Summary 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.
Detailed Description
Study Phase N/A
Study Type † Observational
Study Design † Observational Model: Cohort, Time Perspective: Prospective
Primary Outcome Measure † Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT
Secondary Outcome Measure † Develop a Disability Severity Index including items with high patient-physician concordance
Condition † Charcot Marie Tooth Disease
Intervention †
Study Arms / Comparison Groups 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.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status †
Estimated Enrollment † 291
Start Date † June 2011
Completion Date March 2014
Primary Completion Date March 2014
Eligibility Criteria † Inclusion Criteria: - patients with CMT ages 8-100, - health care professionals who take care of patients with CMT. - Eligible patients will be patients that have joined the INC RDCRN Contact Registry. These will be patients or guardians with known or probable inherited neuropathies. - Eligible health care professionals who participate in the care of patients with inherited neuropathies (physicians, genetic counselors, physical and occupational therapists etc.) attending the 4th International CMT Consortium to be held in Potomac Maryland June 29-July 1, 2011. Exclusion Criteria: - Does not read or speak English.
Gender Both
Ages 8 Years - N/A
Accepts Healthy Volunteers Accepts Healthy Volunteers
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT01455623
Organization ID INC-6604
Secondary IDs ††
Responsible Party Principal Investigator
Study Sponsor † University of Iowa
Collaborators †† National Institute of Neurological Disorders and Stroke (NINDS)
Investigators † Principal Investigator: Sindhu Ramchandren, MD, Wayne State University
Information Provided By
Verification Date March 2014
First Received Date † October 17, 2011
Last Updated Date March 25, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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