Development and Validation of a Disability Severity Index for CMT

NCT01455623 | Completed

• 1 other identifier: INC-6604
• Study Type: observational
• Target: 291
• Locations: 1 country
• Sites: 1

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.

Conditions

Charcot Marie Tooth Disease

Keywords

CMT

Outcome Measures

Primary Outcomes (1)

• Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT

  1 year

Secondary Outcomes (1)

• Develop a Disability Severity Index including items with high patient-physician concordance

  1 year

Study Arms (2)

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 Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Does not read or speak English.

Sponsors & Collaborators

• University of Iowa: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator
• Muscular Dystrophy Association: collaborator

Study Sites (1)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Sindhu Ramchandren, MD

  Wayne State University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 17, 2011
• First Posted: October 20, 2011
• Study Start: June 1, 2011
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: March 26, 2014

Record last verified: 2014-03

Locations

US (1)