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

This study has been completed.
Sponsor:
Collaborators:
Muscular Dystrophy Association
Information provided by (Responsible Party):
Michael Shy, University of Iowa
ClinicalTrials.gov Identifier:
NCT01455623
First received: October 17, 2011
Last updated: March 25, 2014
Last verified: March 2014

October 17, 2011
March 25, 2014
June 2011
March 2014   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01455623 on ClinicalTrials.gov Archive Site
Develop a Disability Severity Index including items with high patient-physician concordance [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Same as current
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Development and Validation of a Disability Severity Index for CMT
Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease

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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Observational
Observational Model: Cohort
Time Perspective: Prospective
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Non-Probability Sample

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.
Charcot Marie Tooth Disease
Not Provided
  • 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.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
291
March 2014
March 2014   (final data collection date for primary outcome measure)

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.
Both
8 Years and older
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01455623
INC-6604
No
Michael Shy, University of Iowa
University of Iowa
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Muscular Dystrophy Association
Principal Investigator: Sindhu Ramchandren, MD Wayne State University
University of Iowa
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP