The SYNAPSE-CMT Clinical Trial for Charcot-Marie-Tooth Disease скачать в хорошем качестве

The SYNAPSE-CMT Clinical Trial for Charcot-Marie-Tooth Disease

Teresa Gidaro, Director of Clinical Development for NMD Pharma, discusses the SYNAPSE-CMT clinical trial testing NMD670 in the treatment of Charcot-Marie-Tooth disease (CMT). CMT is a type of peripheral neuropathy, a condition affecting the transmission of information between the central nervous system and the rest of the body. The condition is usually slowly progressive. Signs and symptoms include distal muscle weakness and atrophy, sensory loss, and slow nerve conduction velocity. It is often associated with pes cavus foot deformity and bilateral foot drop. There are currently no approved treatments for the condition. The SYNAPSE-CMT is a phase 2, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, and tolerability of NMD670 in ambulatory adults with CMT. NMD670 is a first-in-class neuromuscular transmission enhancer that targets selective inhibition of the skeletal muscle CIC-1 chloride ion channel. Primary endpoints of the study include change in baseline to day 21 in total distance walked during the 6 Minute Walk Test, time to complete the 10 Minute Walk/Run Test, and total time taken to perform the Timed Up and Go test in NMD670 versus placebo. The Synapse-CMT study will be conducted in 19 clinical sites across five countries.