An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie

Status Completed

Clinical Trial 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.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Charcot Marie Tooth Disease (CMT)
Charcot-Marie-Tooth Disease
Congenital Abnormalities
Genetic Diseases, Inborn
Hereditary Sensory and Motor Neuropathy
Heredodegenerative Disorders, Nervous System
Nerve Compression Syndromes
Nervous System Diseases
Tooth Diseases

Clinical Trial Details

NCT number	NCT02429947
Study type	Observational
Source	University of South Florida
Contact
Status	Completed
Phase	N/A
Start date	July 2012
Completion date	June 2013

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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms