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

Tooth Diseases Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02429947
• Other study ID #: INC6606
• Secondary ID: 5U54NS064808-07I
• Status: Completed
• Phase: N/A
• First received: April 20, 2015
• Last updated: June 9, 2017
• Start date: July 2012
• Est. completion date: June 2013

Study information

• Verified date: June 2017
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 411
• Est. completion date: June 2013
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

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

Locations

Country	Name	City	State
United States	RDCRN Data Management and Coordinating Center , Epidemiology Center; University of South Florida	Tampa	Florida

Sponsors (3)

Lead Sponsor	Collaborator
University of South Florida	National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	1 year
Secondary	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.	1 year