A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
NCT ID: NCT03810508
Last Updated: 2022-03-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
21 participants
OBSERVATIONAL
2019-07-18
2022-02-15
Brief Summary
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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.
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Detailed Description
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CMT4J is a rare subtype of CMT caused by mutations in the FIG4 gene. Pediatric-onset disease can often be characterized by rapid progression of muscle weakness and atrophy, culminating in loss of ambulation and respiratory compromise and premature death. Adult-onset CMT4J can present with a more variable disease course.
No prospective natural history study for CMT4J has been reported. This study aims to prospectively investigate the natural history of CMT4J, and concurrently to identify potential outcome measures that could be used in future clinical trials. No investigational product will be provided in the study.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
ALL
No
Sponsors
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Neurogene Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Elise Beausoleil
Role: STUDY_DIRECTOR
Neurogene Inc.
Locations
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University of Iowa
Iowa City, Iowa, United States
University of Texas Southwestern
Dallas, Texas, United States
Countries
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Other Identifiers
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CMT4J-100
Identifier Type: -
Identifier Source: org_study_id
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