Close
Close

An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients

Descriptive Information
Brief Title † An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients
Official Title † An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients
Brief Summary The purpose of this study is to identify the issues that have greatest impact on QOL for patients with Charcot Marie Tooth (CMT) Disease. Patients who have -registered in the Inherited Neuropathies Consortium Contact Registry will be invited to participate.
Detailed Description The protocol consists of two anonymous surveys. The first large scale survey is designed to identify items that best reflect the issues and symptoms with high impact upon QOL in CMT. A second brief survey focuses on the frequency and impact of muscle cramps on QOL in adult CMT. The surveys will be distributed via an online link to all adult CMT patients self-registered with the RDCRN contact registry. Those who complete the first two surveys will be requested to complete only the second brief survey on muscle cramps again 3 weeks after completing it the first time. Those who complete the brief muscle cramps survey a second time will be requested to complete it again 5 weeks later to assess variability in occurrence of muscle cramps. The first large scale survey to identify items that best reflect the issues and symptoms with high impact upon QOL in CMT will be sent out in a second wave. This second wave will only contain the QOL survey and not the muscle cramp survey. The QOL survey will be distributed via an online link to three populations: adult CMT patients self-registered with the INC (RDCRN) Contact Registry who had not been registered at the time of the first enrollment period, adult patients who had been registered at the time of the first enrollment period and had received the survey, but had not returned it, and patients who were registered, but were under age 18 at the time of the first enrollment period and have turned 18 by the time of the second enrollment period.
Study Phase N/A
Study Type † Observational
Study Design †
Primary Outcome Measure † Percentage of participants affected by specific symptoms of CMT.
Secondary Outcome Measure † Severity of impact of each identified symptom of CMT
Condition † Charcot Marie Tooth Disease (CMT) Hereditary Sensory and Motor Neuropathy Nerve Compression Syndromes Tooth Diseases Congenital Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System
Intervention †
Study Arms / Comparison Groups INC Contact Registry Participants Adult CMT patients who have self-registered at the Inherited Neuropathies Consortium (INC) Contact Registry, a web-based contact registry developed and supported by the Data Management and Coordinating Center (DMCC) for the Rare Diseases Clinical Research Consortium (RDCRN), located at the University of South Florida.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status †
Estimated Enrollment † 411
Start Date † July 2012
Completion Date June 2013
Primary Completion Date June 2013
Eligibility Criteria † Inclusion Criteria: - Patients with CMT that are 18 years or older and have joined the INC RDCRN Contact Registry. Exclusion Criteria: - Does not have CMT. - Does not read or speak English.
Gender All
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT02429947
Organization ID INC6606
Secondary IDs †† 5U54NS064808-07, INC6606
Responsible Party Sponsor
Study Sponsor † University of South Florida
Collaborators †† University of Rochester
Investigators † Study Chair: David Herrmann, MBBCh, University of Rochester
Information Provided By
Verification Date June 2017
First Received Date † April 20, 2015
Last Updated Date June 9, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
Find a Clinical Trial
Related Videos
Free Newsletter