An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients
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|ClinicalTrials.gov Identifier: NCT02429947|
Recruitment Status : Completed
First Posted : April 29, 2015
Last Update Posted : June 14, 2017
|Condition or disease|
|Charcot Marie Tooth Disease (CMT) Hereditary Sensory and Motor Neuropathy Nerve Compression Syndromes Tooth Diseases Congenital Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System|
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 Type :||Observational|
|Actual Enrollment :||411 participants|
|Official Title:||An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients|
|Study Start Date :||July 2012|
|Actual Primary Completion Date :||June 2013|
|Actual Study Completion Date :||June 2013|
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.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02429947
|United States, Florida|
|RDCRN Data Management and Coordinating Center , Epidemiology Center; University of South Florida|
|Tampa, Florida, United States, 33612|
|Study Chair:||David Herrmann, MBBCh||University of Rochester|