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

NCT02429947 | Completed

• 2 other identifiers: INC66065U54NS064808-07
• Study Type: observational
• Target: 411
• Locations: 1 country
• Sites: 1

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.

Conditions

Charcot Marie Tooth Disease (CMT); Hereditary Sensory and Motor Neuropathy; Nerve Compression Syndromes; Tooth Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System

Keywords

Charcot Marie Tooth disease; CMT; HMSN; HMN; HSN

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Severity of impact of each identified symptom of CMT

  1 year

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with CMT, 18 years or older, who have joined the Inherited Neuropathies Consortium RDCRN Contact Registry.

You may qualify if:

• Patients with CMT that are 18 years or older and have joined the INC RDCRN Contact Registry.

You may not qualify if:

• Does not have CMT.
• Does not read or speak English.

Sponsors & Collaborators

• University of South Florida: lead
• University of Rochester: collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

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

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease; Hereditary Sensory and Motor Neuropathy; Nerve Compression Syndromes; Tooth Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System

Condition Hierarchy (Ancestors)

Nervous System Malformations; Nervous System Diseases; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Stomatognathic Diseases

Study Officials

• David Herrmann, MBBCh

  University of Rochester

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2015
• First Posted: April 29, 2015
• Study Start: July 1, 2012
• Primary Completion: June 1, 2013
• Study Completion: June 1, 2013
• Last Updated: June 14, 2017

Record last verified: 2017-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)