An observational study in Charcot Marie Tooth Disease (CMT), Hereditary Sensory and Motor Neuropathy and Nerve Compression Syndromes, sponsored by University of South Florida. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2017-06-14.
Sponsored by University of South Florida · Observational
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.
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.
130 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 38 are open to participants now.
This study's enrollment of 411 is above the median of 92 across 52 observational studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →University of South Florida is the lead sponsor of 338 studies on the registry; 63 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 4 (24%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with CMT, 18 years or older, who have joined the Inherited Neuropathies Consortium RDCRN Contact Registry.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
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.
The percentage of subjects within the sample who have reported experience with the symptom will be calculated.
Time frame: 1 year
Severity of impact of each identified symptom of CMT
The symptoms will be rank ordered with symptoms that affect life severely receiving the highest rank.
Time frame: 1 year
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Jun 2017. You cannot join it, but the record below documents what was studied.
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