Status:
TERMINATED
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Lead Sponsor:
Neurogene Inc.
Conditions:
Charcot-Marie-Tooth Disease
Hereditary Motor and Sensory Neuropathy IV
Eligibility:
All Genders
Brief 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 ...
Detailed Description
Charcot-Marie-Tooth (CMT) diseases are the most common inherited motor and sensory neuropathies, composed of a group of pathologically and genetically distinct subtypes ranging from slowly to rapidly ...
Eligibility Criteria
Inclusion
- Subjects must have a prior confirmed molecular (genetic) diagnosis of CMT4J by clinical presentation and genetic testing.
Exclusion
Key Trial Info
Start Date :
July 18 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 15 2022
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT03810508
Start Date
July 18 2019
End Date
February 15 2022
Last Update
March 31 2022
Active Locations (2)
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1
University of Iowa
Iowa City, Iowa, United States, 52242
2
University of Texas Southwestern
Dallas, Texas, United States, 75390