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

AGT-100216: An oral medication that may reduce nerve degeneration cause by CMT
Phase: 1
Recruiting: unknown
Clinical Sites: unknown

ALE2: A medication thought to help with some tRNA mutations of CMT
Phase: Planned
Recruiting: unknown
Clinical Sites: unknown

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

Observational Trials are trials that are assessing people with CMT and how their symptoms progress. They’re very important to help researchers and scientists understand the natural course of the disease and how disruption of the progression can be measured.

Natural History 

• New York, New York, USA
• Iowa City, Iowa, USA

Outcome Measures and Biomarkers

• Göttingen, Lower Saxony, Germany
• Marseille, France
• Montpellier, France

Registered Cohort Study

• London, United Kingdom

Pediatric Scale

• Iowa City, Iowa, USA
• Philadelphia, Pennsylvania, USA
• Sydney, New South Wales, Australia
• Milan, Italy

IpSC Biobanking

• New York, New York, USA

Genetics of Modifiers in CMT1A and New Genes for Types 2 

• Los Angeles, California, USA
• Palo Alto, California, USA
• Aurora, Colorado, USA
• Hartford, Connecticut, USA
• Miami, Florida, USA
• Orlando, Florida, USA
• Iowa City, Iowa, USA
• Baltimore, Maryland, USA
• Boston, Massachusetts, USA
• Maple Grove, Minnesota, USA
• Rochester, New York, USA
• Philadelphia, Pennsylvania, USA
• Sydney, New South Wales, Australia
• Milan, Italy
• London, United Kingdom

Genetics of Charcot-Marie-Tooth

• Beijing, China

NT-3 Levels and Function in Individuals With CMT

• Columbus, Ohio, USA