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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
Sponsor:
Collaborators:
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
University of South Florida

Study Description
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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.

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

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 Design
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Study Type : Observational
Actual Enrollment : 411 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
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

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
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.


Outcome Measures
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Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with CMT, 18 years or older, who have joined the Inherited Neuropathies Consortium RDCRN Contact Registry.
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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United States, Florida
RDCRN Data Management and Coordinating Center , Epidemiology Center; University of South Florida
Tampa, Florida, United States, 33612
Sponsors and Collaborators
University of South Florida
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
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Study Chair: David Herrmann, MBBCh University of Rochester
More Information
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Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT02429947    
Other Study ID Numbers: INC6606
5U54NS064808-07 ( U.S. NIH Grant/Contract )
INC6606 ( Other Identifier: Inherited Neuropathies Consortium )
First Posted: April 29, 2015    Key Record Dates
Last Update Posted: June 14, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by University of South Florida:
Charcot Marie Tooth disease
CMT
HMSN
HMN
HSN
Additional relevant MeSH terms:
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Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Congenital Abnormalities
 Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Stomatognathic Diseases
Nervous System Malformations
Neurodegenerative Diseases
Polyneuropathies