Development and Validation of a Disability Severity Index for CMT (6604)

University of Iowa

Status: Completed

Conditions: Charcot Marie Tooth Disease

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT01455623

INC-6604

Details and patient eligibility

About

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.

Enrollment: 291 patients

Sex: All

Ages: 8+ years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

291 participants in 2 patient groups

Health Care Provider

Description:

A person working within the field of CMT.

Patient with CMT

Description:

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.