🏛️ Verified NIH Record • Updated 2026-07-07
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (NCT06151600)
🔵 Active, Not Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Elpida Therapeutics SPC
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Stanford University — San Francisco, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (3 Sites)
- Stanford University — San Francisco, California
- University of Iowa — Iowa City, Iowa
- University of Texas Southwestern — Dallas, Texas
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.