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A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

NCT03810508 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 21

Last updated 2022-03-31

No results posted yet for this study

Summary

Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory compromise and premature death with no available treatment.

The purpose of this study is to investigate the clinical characteristics and natural clinical progression of symptoms in individuals with CMT4J. This natural history study is important to better understand disease course to be able to determine clinically meaningful outcome measures for use in future clinical trials.

Conditions

  • Charcot-Marie-Tooth Disease
  • Hereditary Motor and Sensory Neuropathy IV

Sponsors & Collaborators

  • Neurogene Inc.

    lead INDUSTRY

Principal Investigators

  • Elise Beausoleil · Neurogene Inc.

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-18
Primary Completion
2022-02-15
Completion
2022-02-15

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03810508 on ClinicalTrials.gov