Evaluating NMD670 for Charcot-Marie-Tooth Disease in Adults
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
This study is testing a new treatment called NMD670 to see if it helps adults with Charcot-Marie-Tooth disease feel better compared to a placebo.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 80 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | NMD Pharma A/S Industry-sponsored |
| Locations | 18 sites (Kansas City, Kansas and 17 other locations) |
| Trial ID | NCT06482437 on ClinicalTrials.gov |
What this trial studies
This Phase 2a study investigates the safety and efficacy of NMD670 compared to a placebo in ambulatory adult patients diagnosed with Charcot-Marie-Tooth disease types 1 and 2. Participants will receive the treatment twice daily for 21 days, with the aim of assessing tolerability and clinical outcomes. The study includes a range of eligibility criteria to ensure that participants are suitable for the intervention and can provide informed consent.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 70 with a confirmed diagnosis of Charcot-Marie-Tooth disease type 1 or 2.
Not a fit: Patients with other significant neuromuscular diseases or those with clinically significant laboratory abnormalities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve the quality of life and functional abilities of patients with Charcot-Marie-Tooth disease.
How similar studies have performed: While this approach is being evaluated in this specific context, similar studies have shown promise in treating neuromuscular diseases, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * 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 this trial is running
Kansas City, Kansas and 17 other locations
- University of Kansas Medical Center, Department of Neurology — Kansas City, Kansas, United States (Recruiting)
- Mass General Neurology — Boston, Massachusetts, United States (Recruiting)
- NextGen Precision Health — Columbia, Missouri, United States (Recruiting)
- Columbia University Medical Center — New York, New York, United States (Recruiting)
- University of Rochester Neuromuscular Disease Center — Rochester, New York, United States (Recruiting)
- OSU Department of Neurology Division of Neuromuscular Diseases — Columbus, Ohio, United States (Recruiting)
- National Neuromuscular research Institute, PLLC — Austin, Texas, United States (Recruiting)
- Providence Medical Research Center — Spokane, Washington, United States (Recruiting)
- University Hospitals Leuven, Department of Neurology — Leuven, Belgium (Recruiting)
- CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1 — Liege, Belgium (Recruiting)
- Aarhus University Hospital — Aarhus, Denmark (Recruiting)
- Rigshospitalet, Department of Neurology — Copenhagen, Denmark (Recruiting)
- CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases — Marseille, France (Recruiting)
- Laboratoire d'Explorations Fonctionnelles, CHU Nantes — Nantes, France (Recruiting)
- Centre Hospitalier Universitaire De Nice — Nice, France (Recruiting)
- Bicêtre University Hospital — Paris, France (Recruiting)
- Hospital Universitari Vall d'Hebron — Barcelona, Spain (Recruiting)
- Hospital Universitari i Politecnic La Fe de Valencia — Valencia, Spain (Recruiting)
Study contacts
- Study coordinator: contact@nmdpharma.com
- Email: contact@nmdpharma.com
- Phone: contact@nmdpharma.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.