The Synapse-CMT logo Study

For Adults Living With Type 1 and Type 2 Charcot-Marie-Tooth Disease

What Is Charcot-Marie-Tooth?

Charcot-Marie-Tooth (CMT) is a group of inherited conditions that affect the nerves in the arms and legs. It can cause muscle weakness, trouble walking, balance problems, and fatigue. There are currently no approved medical treatments specifically for CMT. Researchers are looking for new ways to help improve how nerves and muscles communicate.

About NMD 670

NMD670 is an oral investigational medicine that aims to help muscles respond better to nerve signals in CMT.

Icon of arm flexing bicep muscle

It is taken as 2 tablets twice a day with water. It works differently from other treatments or recommendations that a doctor or therapist may provide for CMT, such as medications, braces, or physical therapy, and should be discussed with your doctor.

An image of a pill next to a glass of water, labeled with "2x day" to denote a twice-daily medication schedule.

This approach could reduce symptoms that you have regarding muscle weakness and fatigue, increasing muscle strength, power, and endurance throughout the day. NMD670 is not yet approved and is being studied to ensure safety and effectiveness.

About the CMT Clinical Trial

The purpose of this clinical trial is to study the safety and effects of NMD670 in adults with CMT.

What does participation include?
  • Taking oral tablets twice daily for about 8 weeks
  • At least 6 in-person study visits over about 8 weeks, lasting between 4-6 hours
  • Testing, which may include physical exams, lab work, heart tests, mobility assessments, and questionnaires
  • Study participants have a 50% chance of receiving NMD670 and a 50% chance of receiving placebo (a sugar pill) during the study
What else should I know?
  • Travel costs may be reimbursed, with possible compensation for time or lost wages
  • Participation is voluntary and at no cost
Who is eligible?
  • Adults aged 18–70
  • Those with a confirmed genetic diagnosis of CMT 1 or CMT 2 (not including CMT 1X)
  • Able to do physical tests without an ankle/foot brace (orthopedic inserts allowed)

Enrollment is Now Closed

Thank you to all 80 CMT patients who volunteered and participated in this clinical study!

Stay tuned for updates on upcoming clinical studies for Charcot-Marie-Tooth disease across a broad patient population.

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