Actively Recruiting

All Genders
ID05902351

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

Led by Hereditary Neuropathy Foundation · Updated on 2024-10-01

10000

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are gathering detailed information about Charcot-Marie-Tooth disease (CMT) and other inherited neuropathies through a Natural History Study. This study collects patient-reported data, genetic reports, electronic health records, and clinical notes to better understand the disease's impact, the diagnosis process, and how common it is. The goal is to support scientists in their efforts to find a cure for these conditions. Participants will be asked to complete a Natural History Survey that helps record their experience with CMT. There are no specific treatment groups or interventions in this observational study. Instead, the study focuses on collecting information from affected individuals of all ages, including children, adolescents, and adults, with confirmed or suspected diagnoses. Throughout the study, researchers will review data related to the type of CMT, symptoms experienced, how symptoms affect daily activities, and any related health conditions over a period of 156 weeks. Participation involves sharing survey responses and allowing access to medical records, supporting long-term data collection to improve understanding of inherited neuropathies.

CONDITIONS

Brief Title

Natural History Study for Charcot Marie Tooth Disease

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have a confirmed or suspected diagnosis of Charcot-Marie-Tooth disease or other inherited neuropathies.
  • Children, adolescents, and adults are eligible with appropriate consent.
  • Diagnosis can be based on family history, clinical tests such as neuro exam, EMG, NCS, or genetic testing.
  • Participants must provide electronic informed consent to join the study.
Not Eligible

You will not qualify if you...

  • Individuals without Charcot-Marie-Tooth disease or other inherited neuropathies are not eligible to participate.

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Surveillance

Duration - Up to 156 weeks

Participants are observed to monitor the natural history of Charcot-Marie-Tooth disease and other inherited neuropathies.

Periodic visits for assessments

Trial Site Locations

Total: 1 location

1

Hereditary Neuropathy Foundation

New York, New York, United States, 10128

Actively Recruiting

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Research Team

A

Allison Moore

J

Joy Aldrich

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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