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

March 25, 2014 updated by: Michael Shy, University of Iowa

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

291

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
Develop a Disability Severity Index including items with high patient-physician concordance
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Iowa

Collaborators

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

Investigators

  • Principal Investigator: Sindhu Ramchandren, MD, Wayne State University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2011

Primary Completion (Actual)

March 1, 2014

Study Completion (Actual)

March 1, 2014

Study Registration Dates

First Submitted

October 17, 2011

First Submitted That Met QC Criteria

October 19, 2011

First Posted (Estimate)

October 20, 2011

Study Record Updates

Last Update Posted (Estimate)

March 26, 2014

Last Update Submitted That Met QC Criteria

March 25, 2014

Last Verified

March 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INC-6604

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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