CMT clinical trials are specialized research efforts aimed at developing and testing new treatments for Charcot-Marie-Tooth disease, which affects the peripheral nerves. The trials focus on various aspects of treatment, from medications and gene therapy to physical interventions, and they are essential for advancing understanding and management of the disease.

These trials are not only pivotal for developing treatments but also provide CMT patients with access to cutting-edge therapies that could improve their quality of life. Use the clinical trial website to find out if you qualify to participate in currently available CMT therapy trials.

NMD-670: An oral drug that might enhance the signal between the nerve and the muscle
Phase 2A: Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability
Over 21 Days in Ambulatory Adult Patients
Recruiting: 80 patients with types 1 and types 2
18 Clinical Sites:
University of Kansas Medical Center
Mass General Neurology
NextGen Precision Health (University of Missouri)
Columbia University Medical Center
University of Rochester Neuromuscular Disease Center
Ohio State University Department of Neurology
National Neuromuscular Research Institute (Austin, TX)
Providence Medical Research Center (Spokane, WA)
2 sites in Belgium
2 sites in Demark
4 sites in France
2 sites in Spain

EN101: An injectable stem cell therapy thought to remyelinate the nerves and rebuild the muscles
Phase An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001
Recruiting: 12 patients with CMT1A and 1E
1 Clinical Sites: Samsung Medical Center, Seoul, Korea

ORY-4001: An oral medication that may reduce nerve degeneration cause by CMT
Phase: Planned
Recruiting: unknown
Clinical Sites: unknown

Attention patients and families affected by Charcot Marie Tooth Disease Type 2F.

Your participation is invaluable in advancing CMT research. Dr. Shy at the University of Iowa is conducting a study on CMT Subtype Biomarkers and Outcome Measures. The University of Iowa is one of three sites in the U.S. involved in this crucial project.

If you have a genetic diagnosis of CMT2F, you are invited to join them in determining the best methods to measure disease progression. For further details and to express your interest, please contact their study coordinator, Amanda, at [email protected]. Your involvement can make a significant impact.