A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Launched by NEUROGENE INC. · Jan 17, 2019
Trial Information
Current as of August 02, 2025
Terminated
Keywords
ClinConnect Summary
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 progressive disease.
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 b...
Gender
ALL
Eligibility criteria
- • Subjects must have a prior confirmed molecular (genetic) diagnosis of CMT4J by clinical presentation and genetic testing.
About Neurogene Inc.
Neurogene Inc. is a pioneering biotechnology company focused on developing innovative gene therapies for rare neurological disorders. With a commitment to addressing unmet medical needs, Neurogene leverages advanced gene delivery technologies to create transformative treatments aimed at improving the quality of life for patients and their families. The company’s research and clinical development efforts are driven by a team of experienced professionals dedicated to scientific excellence and patient-centered solutions. Through strategic collaborations and a robust pipeline of therapeutic candidates, Neurogene is at the forefront of advancing the field of neurology and gene therapy.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Iowa City, Iowa, United States
Dallas, Texas, United States
Patients applied
Trial Officials
Elise Beausoleil
Study Director
Neurogene Inc.
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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