Natural History Study for Charcot Marie Tooth Disease

Charcot-Marie-Tooth Disease Clinical Trial

Official title:

Global Registry for Inherited Neuropathies Natural History Study for Charcot Marie Tooth Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05902351
• Other study ID #: GRIN1001
• Status: Recruiting
• Start date: November 1, 2013
• Est. completion date: December 31, 2029

Study information

• Verified date: June 2023
• Source: Hereditary Neuropathy Foundation
• Contact: Allison Moore
• Phone: 212-722-8396
• Email: allison[@]hnf-cure.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden, diagnostic journey, and prevalence of disease that will aid scientists in their work toward finding a cure. Participants will be asked to complete a Natural History Survey.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10000
• Est. completion date: December 31, 2029
• Est. primary completion date: December 31, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

Patients will be made aware of the study by HNF and others (referenced above) and invited to participate. Once patients have reviewed and signed electronically the informed consent document, it is attached to their file. All affected individuals with CMT/IN are eligible to participate in GRIN with proper informed consent. Children, adolescents and adults with either a confirmed diagnosis or suspected to have CMT/IN are eligible with parent and/or guardian consent. Individuals that have been clinically diagnosed through family history and/or standard clinical testing (e.g. neuro exam, EMG, NCS) and/or genetically tested or suspected to have CMT/IN (note: many mutations have not been identified yet) are eligible. Exclusion Criteria: People that do not have Charcot-Marie-Tooth or other Inherited Neuropathies

Related Conditions & MeSH terms

• Charcot-Marie-Tooth
• Charcot-Marie-Tooth Disease
• Charcot-Marie-Tooth Disease Type 2A
• Charcot-Marie-Tooth Disease, Type IA
• Charcot-Marie-Tooth Disease, Type IB
• Deafness
• Disease
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes
• Refsum Disease
• Tooth Diseases

Locations

Country	Name	City	State
United States	Hereditary Neuropathy Foundation	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Hereditary Neuropathy Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Identify the type of CMT	Patient-Reported Outcomes depending on individual experience I.e. Genetic testing, clinical observation, EMG, family history.	156 weeks
Primary	Disease Symptoms	Patient-Reported Observations	156 weeks
Primary	Impact of symptoms on Activities of Daily Living	Patient-Reported Observations	156 weeks
Primary	Associated Comorbidities	Patient-Reported Observations	156 weeks

External Links

•   Landing page and entry for patients to participate in the Global Registry for Inherited Neuropathies