Testing a new medication for inherited nerve damage disease

This is an early-stage study testing a new medication called EDK060 for people with Charcot-Marie-Tooth Disease Type 1A (CMT1A), an inherited condition affecting the nerves that control muscle movement and sensation. Researchers want to understand whether this medication is safe and how the body processes it. About half of participants will receive the medication at different dose levels, and half will receive a placebo (inactive treatment), so researchers can compare how the two groups respond.

CMT1A is caused by a genetic duplication that leads to progressive nerve damage, causing weakness and numbness in the feet and hands. There are very few treatment options available today, so this study aims to explore whether this medication could offer a new way to help slow or manage the condition.