Seeking Participants for a New CMT Clinical Trial: A Chance to Help Advance Treatment
If you’re living with Charcot-Marie-Tooth (CMT) disease, specifically CMT1 or CMT2 subtypes you may be eligible to participate in an exciting new clinical trial. NMD Pharma, a biotechnology company, has received approval from the U.S. Food and Drug Administration (FDA) to launch the SYNAPSE-CMT Phase 2 trial. This trial will evaluate the safety, tolerability, and potential effectiveness of an investigational medication, NMD670, which could improve muscle strength and endurance in individuals with CMT.
What’s the Goal of the Study?
NMD670 works by enhancing communication between nerves and muscles where transmission is impaired, a common issue for people living with CMT. The hope is that this treatment will improve your strength, endurance, and overall daily functioning. By joining the study, you’ll play a critical role in determining whether this therapy could help others with CMT in the future.
Who Can Join?
If you’re between the ages of 18-70 and have been diagnosed with CMT1 or CMT2 subtypes you may qualify. The study is open to patients who are ambulatory and able to complete basic walking tests without the assistance of ankle-foot orthoses (AFOs). During the study, you’ll either receive NMD670 or a placebo over a 21-day period, with regular assessments to monitor your progress.
Where Is the Study Taking Place?
The study is being conducted at several sites across the U.S. Two locations are currently accepting participants:
NextGen Precision Health, Missouri
Principal Investigator: W. David Arnold, MD
Contact: Neetha Gali | Email: [email protected] | Phone: (573) 882-3065
National Neuromuscular Research Institute, Texas
Principal Investigator: Yessar Hussain, MD
Contact: Stephanie Gonsoulin, RN, BSN | Email: [email protected] | Phone: (512) 920-0140 x210
Other locations, including those in New York, Boston, Kansas, Ohio, and Washington will be opening soon.
What Can You Expect?
As a participant, you’ll be closely monitored through a series of tests that measure your ability to walk/run, and get up from sitting. You’ll also receive all study-related medication, supplies, and evaluations at no cost, and reasonable travel expenses will be fully reimbursed.
*To be considered for the trial, there is a specific criteria around daily AFO use.
Patients using an AFO but who do not rely on it for regular activities, home activities, or who use it only in specific situations, such as during outdoor activities, traveling, or work-related tasks, are not considered daily AFO users.
Interested in Joining?
If you think you might qualify or would like to learn more, please complete the form.
By participating, you’ll not only be helping researchers learn more about potential treatments for CMT, but you’ll also be part of the broader community working toward better care for all those affected by CMT. Stay connected with us by joining GRIN to stay informed about the latest research and clinical trials.
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