Development and Validation of a Disability Severity Index for CMT

University of Iowa291 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

291

Conditions

Charcot Marie Tooth Disease

Eligibility

Sex: ALLMin age: 8 YearsHealthy volunteers:

Medical Language ↔ Plain English

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.

Locations (1)

University of Iowa

Iowa City, Iowa, United States

Outcomes

Primary Outcomes

Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT

Time frame: 1 year

Secondary Outcomes

Develop a Disability Severity Index including items with high patient-physician concordance

Time frame: 1 year

Data from ClinicalTrials.gov

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