A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
NCT06151600 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 20
Last updated 2026-01-22
Summary
This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study will enroll 20 subjects of any age into a uniform protocol for follow-up and evaluations. Subject visits will occur every 12 months + 4 weeks for up to 2 years.
Conditions
Sponsors & Collaborators
-
Elpida Therapeutics SPC
lead INDUSTRY
Principal Investigators
-
Souad Messahel, Ph.D · Elpida Therapeutics SPC
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-07-29
- Primary Completion
- 2031-03-01
- Completion
- 2032-03-01
Countries
- United States
Study Locations
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