The Charcot-Marie-Tooth Association (CMTA) congratulates CMTA-STAR Alliance Partner NMD Pharma A/S (NMD Pharma) on initiating its Phase II SYNAPSE-CMT trial. This significant milestone reflects a shared commitment to advancing therapeutic options for individuals living with CMT. The trial’s first patient was dosed under the direction of Yessar M. Hussain, MD, CMTA Centers of Excellence clinic director at the Austin Neuromuscular Center in Austin, Texas.
NMD Pharma’s experimental drug, NMD670, is designed to improve communication between nerves and muscles to address skeletal muscle weakness. The SYNAPSE-CMT trial will evaluate the safety, tolerability, and efficacy (how well the treatment works) of NMD670 over 21 days in 80 adult patients with genetically confirmed CMT types 1 and 2.
Shared Vision for Progress
“This milestone reflects the remarkable progress being achieved through the CMTA-STAR Alliance,” said CMTA CEO Sue Bruhn, PhD. “We are excited about NMD Pharma’s dedication to developing potential treatments that can work broadly and address multiple types of CMT. Their work offers real hope to those living with CMT, and we have been honored to support their commitment to patient-focused drug development through focus groups that leveraged our Patients as Partners in Research platform.”
Focus on the CMT Community
The SYNAPSE-CMT trial will measure key functional outcomes, including the six-minute walk test (6MWT) and the timed-up-and-go test (TUG), which evaluates how quickly participants can stand from a seated position and walk a short distance. These measures are designed to assess the impact of NMD670 on mobility and strength.
The study is currently enrolling patients with both CMT Types 1 and 2 across clinical sites in the U.S. and Europe, with additional locations to be announced through March 2025.
Dr. Hussain stated: “Patients with CMT are very excited about entering into this study. This is the only clinical study with a clinically feasible treatment to address the muscle weakness and fatigue with a pharmaceutical therapy at the moment. The fact that this study enrolls patients with all types of CMT 1 and 2 is also encouraging and motivating to the community. I am happy to be involved in this exciting clinical study.”
Register with Patients as Partners in Research Today
CMTA will soon contact our Patients as Partners in Research who match the study criteria for NMD Pharma’s SYNAPSE-CMT trial. CMTA will contact only those who consent to receive research opportunity notifications.
Not yet registered with Patients as Partners in Research but wish to participate in the SYNAPSE-CMT trial? Click the button below to register today! Registering today ensures you are the first in line when NMD Pharma announces trial locations. Registering also ensures you are updated on all the latest news and developments in CMT research. Registration is free and secure, and all who have CMT are invited to register.
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To learn more about the SYNAPSE-CMT trial visit ClinicalTrials.gov
Published On: November 20, 2024