CompletedPhase 2
Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
Key facts
- Who runs it
- NMD Pharma A/S
- Where
- Austin, Boston, Columbia, Columbus, Kansas City, New York, Rochester, and Spokane, United StatesSee the 8 sitesAlso in 4 other countries, 11 sites
- Trial code
- Status
- Completed
- Phase
- Phase 2
- People taking part
- 81 people enrolled
- Started
- 30 Sept 2024
- Ended
- 4 Nov 2025
- Type of study
- Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- NMD670
- Placebo
- Record updated
- 12 November 2025
Who can take part
- Age
- 18 years to 70 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 10 criteriaShow less
Who can take part
- Male or female participants must be 18 to 70 years inclusive at the time of signing the ICF
- Diagnosis of CMT type 1 or 2 confirmed by genetic testing
- Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
- Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- Participant is capable of and has given signed informed consent
Who cannot take part
- Participants who have received treatment with another IMP within 30 days (or 5 half-lives of the medication, whichever is longer) prior to day 1
- Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular diseases) and/or ability to complete the tests, in the opinion of the Investigator
- Participants with history of poor compliance with relevant therapy in the opinion of the Investigator
- Participants with laboratory test result abnormalities at screening considered clinically significant by the Investigator
- Female participants who plan to become pregnant during the study or are currently pregnant or breastfeeding
Where in United States
Also in 4 other countries, 11 sites
- National Neuromuscular research Institute, PLLCAustin, United StatesNo longer taking part
- Mass General NeurologyBoston, United StatesNo longer taking part
- NextGen Precision HealthColumbia, United StatesNo longer taking part
- OSU Department of Neurology Division of Neuromuscular DiseasesColumbus, United StatesNo longer taking part
- University of Kansas Medical Center, Department of NeurologyKansas City, United StatesNo longer taking part
- Columbia University Medical CenterNew York, United StatesNo longer taking part
- University of Rochester Neuromuscular Disease CenterRochester, United StatesNo longer taking part
- Providence Medical Research CenterSpokane, United StatesNo longer taking part
About the trial
What the trial wants to find out
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.
The 2 groups in the trial
- NMD670Treatment being tested
- PlaceboPlacebo, for comparison
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 Over 21 Days in Ambulatory Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
- Publications
- The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease.The Journal of neuroscience : the official journal of the Society for Neuroscience, 2025
- Record updated
- 12 November 2025