CMT Clinical Trials
What are CMT Clinical Trials?
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.
Active CMT Trials
Applied Therapeutics
AT-007: An oral drug that might block the conversion of glucose to sorbitol, reducing sorbitol levels in people with SORD deficiency
Phase 3: Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy to reduce sorbitol levels
Recruited: 56 patients with SORD-CMT type 4 (Recruiting complete)
10 Clinical Sites:
University of Colorado
University of Miami
University of Iowa
Mass General Neurology
Hassman Research Institute (NJ)
University of Rochester Neuromuscular Disease Center
University of Pennsylvania
1 site in Prague
1 site in Milan
1 site in London
NMD Pharma
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
EnCell
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
Augustine Therapeutics
AGT-100216: An oral medication that may reduce nerve degeneration cause by CMT
Phase: 1
Recruiting: unknown
Clinical Sites: unknown
Planned CMT Trials
Actio Biosciences
ABS-0871: Oral drug for CMT2C (TRPV4) small molecule thought to inhibit overactivated ion channel activity and restore vocal cord, respiratory, bladder and other functions.
Phase: Planned
Recruiting: unknown
Clinical Sites: unknown
Alesta
ALE2: A medication thought to help with some tRNA mutations of CMT
Phase: Planned
Recruiting: unknown
Clinical Sites: unknown
Oryzon Therapeutics
ORY-4001: An oral medication that may reduce nerve degeneration cause by CMT
Phase: Planned
Recruiting: unknown
Clinical Sites: unknown
Observational Studies
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
Outcome Measures and Biomarkers
Registered Cohort Study
Pediatric Scale
- Iowa City, Iowa, USA
- Philadelphia, Pennsylvania, USA
- Sydney, New South Wales, Australia
- Milan, Italy
IpSC Biobanking
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