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

This study is enrolling participants by invitation only.
Sponsor:
Collaborators:
National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy Association
Information provided by (Responsible Party):
Michael E. Shy, MD, Wayne State University
ClinicalTrials.gov Identifier:
NCT01455623
First received: October 17, 2011
Last updated: October 19, 2011
Last verified: October 2011
History of Changes
  Purpose

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
Charcot Marie Tooth Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Wayne State University:

Primary Outcome Measures:
  • Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Develop a Disability Severity Index including items with high patient-physician concordance [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 200
Study Start Date: June 2011
Estimated Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Groups/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.

  Eligibility

Ages Eligible for Study:   8 Years and older
Genders Eligible for Study:   Both
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.
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01455623

Locations
United States, Michigan
Wayne State University
Detroit, Michigan, United States, 48201
Sponsors and Collaborators
Wayne State University
National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy Association
Investigators
Principal Investigator: Sindhu Ramchandren, MD Wayne State University
  More Information

No publications provided

Responsible Party: Michael E. Shy, MD, Professor, Wayne State University
ClinicalTrials.gov Identifier: NCT01455623     History of Changes
Other Study ID Numbers: INC-6604
Study First Received: October 17, 2011
Last Updated: October 19, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Wayne State University:
CMT

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

ClinicalTrials.gov processed this record on May 22, 2013