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

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.

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Key information

Conditions

Charcot Marie Tooth Disease (CMT) Charcot-Marie-Tooth Disease Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Nerve Compression Syndromes Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies Stomatognathic Diseases Tooth Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

RDCRN Data Management and Coordinating Center , Epidemiology Center; University of South Florida

Tampa, Florida, 33612, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

Percentage of participants affected by specific symptoms of CMT.

Time frame: 1 year

The percentage of subjects within the sample who have reported experience with the symptom will be calculated.

Secondary outcomes

Severity of impact of each identified symptom of CMT

Time frame: 1 year

The symptoms will be rank ordered with symptoms that affect life severely receiving the highest rank.

Sponsors and collaborators

Lead sponsor

University of South Florida

Other

Collaborators

National Institute of Neurological Disorders and Stroke (NINDS)
University of Rochester

Registry information

Important dates

Study start: 2012
Primary completion: 2013
Study completion: 2013
First posted: Apr 29, 2015
Registry last updated: Jun 14, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed