Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

Completed · Phase 2 · Has a placebo group

Conditions studied: Charcot-Marie-Tooth Disease

In brief

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.

Key facts

Study ID

NCT06482437

Run by

NMD Pharma A/S

People needed

81

Starts

2024-09-30

Expected to finish

2025-11-04

Last updated by the study team

2025-11-12

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 laboratory test result abnormalities at screening considered clinically significant by the Investigator.
• 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 history of poor compliance with relevant therapy in the opinion of the Investigator.
• Female participants who plan to become pregnant during the study or are currently pregnant or breastfeeding.

Where it is running

• University of Kansas Medical Center, Department of Neurology — Kansas City, Kansas, United States
• Mass General Neurology — Boston, Massachusetts, United States
• NextGen Precision Health — Columbia, Missouri, United States
• Columbia University Medical Center — New York, New York, United States
• University of Rochester Neuromuscular Disease Center — Rochester, New York, United States
• OSU Department of Neurology Division of Neuromuscular Diseases — Columbus, Ohio, United States
• National Neuromuscular research Institute, PLLC — Austin, Texas, United States
• Providence Medical Research Center — Spokane, Washington, United States
• University Hospitals Leuven, Department of Neurology — Leuven, Belgium
• CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1 — Liège, Belgium
• Aarhus University Hospital — Aarhus, Denmark
• Rigshospitalet, Department of Neurology — Copenhagen, Denmark
• CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases — Marseille, France
• Laboratoire d'Explorations Fonctionnelles, CHU Nantes — Nantes, France
• Centre Hospitalier Universitaire De Nice — Nice, France
• Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière — Paris, France
• Bicêtre University Hospital — Paris, France
• Hospital Universitari Vall d'Hebron — Barcelona, Spain
• Hospital Universitari i Politecnic La Fe de Valencia — Valencia, Spain

Full record on ClinicalTrials.gov

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