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
Status: Completed
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: f
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• 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
Locations
United States
Kansas
University of Kansas Medical Center, Department of Neurology
Kansas City
Massachusetts
Mass General Neurology
Boston
Missouri
NextGen Precision Health
Columbia
New York
Columbia University Medical Center
New York
University of Rochester Neuromuscular Disease Center
Rochester
Ohio
OSU Department of Neurology Division of Neuromuscular Diseases
Columbus
Texas
National Neuromuscular research Institute, PLLC
Austin
Washington
Providence Medical Research Center
Spokane
Other Locations
Belgium
University Hospitals Leuven, Department of Neurology
Leuven
CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1
Liège
Denmark
Aarhus University Hospital
Aarhus
Rigshospitalet, Department of Neurology
Copenhagen
France
CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases
Marseille
Laboratoire d'Explorations Fonctionnelles, CHU Nantes
Nantes
Centre Hospitalier Universitaire De Nice
Nice
Bicêtre University Hospital
Paris
Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière
Paris
Spain
Hospital Universitari Vall d'Hebron
Barcelona
Hospital Universitari i Politecnic La Fe de Valencia
Valencia
Time Frame
Start Date: 2024-09-30
Completion Date: 2025-11-04
Participants
Target number of participants: 81
Treatments
Experimental: NMD670
Placebo_comparator: Placebo
Related Therapeutic Areas
Sponsors
Leads: NMD Pharma A/S