Development and Validation of a Disability Severity Index for CMT

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.

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Key information

Conditions

Charcot Marie Tooth Disease Charcot-Marie-Tooth Disease Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies

Age range

8 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Iowa

Iowa City, Iowa, 52242, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Sponsors and collaborators

Lead sponsor

University of Iowa

Other

Collaborators

Muscular Dystrophy Association
National Institute of Neurological Disorders and Stroke (NINDS)

Registry information

Official study title

Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease

Acronym: 6604

Important dates

Study start: 2011
Primary completion: 2014
Study completion: 2014
First posted: Oct 20, 2011
Registry last updated: Mar 26, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed