Understanding Charcot-Marie-Tooth Disease through patient data
Global Registry for Inherited Neuropathies Natural History Study for Charcot Marie Tooth Disease
This study is collecting information from people with Charcot-Marie-Tooth Disease to better understand their experiences and help improve future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10000 (estimated) |
| Sex | All |
| Sponsor | Hereditary Neuropathy Foundation Government |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT05902351 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather and analyze patient-reported data, genetic reports, Electronic Health Records (EHRs), and clinical notes related to Charcot-Marie-Tooth Disease (CMT). By documenting the burden and diagnostic journey of individuals with CMT, the study seeks to enhance understanding of the disease's prevalence and characteristics. Participants will complete a Natural History Survey to contribute valuable insights that could inform future research and potential treatments.
Who should consider this trial
Good fit: Ideal candidates include individuals of all ages with a confirmed or suspected diagnosis of Charcot-Marie-Tooth Disease or other inherited neuropathies.
Not a fit: Patients who do not have Charcot-Marie-Tooth Disease or other inherited neuropathies will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide critical data that aids in the development of effective treatments for Charcot-Marie-Tooth Disease.
How similar studies have performed: While this study focuses on natural history, similar observational studies have successfully contributed to understanding other genetic conditions, indicating potential for valuable insights.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
New York, New York
- Hereditary Neuropathy Foundation — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Allison Moore — Hereditary Neuropathy Foundation
- Study coordinator: Allison Moore
- Email: allison@hnf-cure.org
- Phone: 212-722-8396
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.