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

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ClinicalTrials.gov Identifier: NCT01455623
Recruitment Status : Completed
First Posted : October 20, 2011
Last Update Posted : March 26, 2014
Information provided by (Responsible Party):

Study Description
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.

Condition or disease
Charcot Marie Tooth Disease

Study Design

Study Type : Observational
Actual Enrollment : 291 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
Study Start Date : June 2011
Primary Completion Date : March 2014
Study Completion Date : March 2014

Groups and Cohorts

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.

Outcome Measures

Primary Outcome Measures :
  1. Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Develop a Disability Severity Index including items with high patient-physician concordance [ Time Frame: 1 year ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population

Two cohorts:

  1. Patients self identified as having CMT and have chosen to be a part of the Inherited Neuropathies Consortium Contact Registry, housed by the Rare Disease Clinical Research Network.
  2. Healthcare Providers working in the field of CMT.

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01455623

United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
Sponsors and Collaborators
University of Iowa
National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy Association
Principal Investigator: Sindhu Ramchandren, MD Wayne State University
More Information

Responsible Party: Michael Shy, Professor, University of Iowa
ClinicalTrials.gov Identifier: NCT01455623     History of Changes
Other Study ID Numbers: INC-6604
First Posted: October 20, 2011    Key Record Dates
Last Update Posted: March 26, 2014
Last Verified: March 2014

Keywords provided by Michael Shy, University of Iowa:

Additional relevant MeSH terms:
Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Stomatognathic Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Nervous System Malformations
Congenital Abnormalities