Safety and Efficacy of NMD670 in Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease
- Registration Number
- NCT06482437
- Lead Sponsor
- NMD Pharma A/S
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 80
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - NMD670 NMD670 -
- Primary Outcome Measures
Name Time Method Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo Baseline to day 21 Change from baseline to day 21 in the time to complete 10 meter walk/run test for NMD670 vs placebo Baseline to day 21 Change from baseline to day 21 in the time to complete timed-up-and-go for NMD670 vs placebo Baseline to day 21
- Secondary Outcome Measures
Name Time Method Incidence of clinically significant ECG abnormalities Over 21 days of dosing Summarised per treatment
Incidence of Suicidal Ideation or Suicidal Behavior Over 21 days of dosing Summarised per treatment
Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo Baseline to day 21 CMT Functional Outcome Meausre is a 12-item scale. Scale goes from 0-100 and a higher score indicates worse symptomatology
Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo Baseline to day 21 Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo Baseline to day 21 The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology
Incidence of treatment emergent adverse events Over 21 days of dosing Summarised per treatment
Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo Baseline to day 21 The Overall Neuropathy Limitation Scale consists of an arm and a leg scale. Scale goes from 0-12 and a higher score indicates worse symptomatology
Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo Baseline to day 21 The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology
Incidence of serious treatment emergent adverse events Over 21 days of dosing Summarised per treatment
Incidence of clinically significant abnormalities on physical examinations Over 21 days of dosing Summarised per treatment
Incidence of clinically significant abnormalities on safety laboratory parameters Over 21 days of dosing Summarised per treatment
Incidence of clinically significant abnormalities on opthalmological examinations From screening (day -28 to day -1) until follow up (day 28)] Summarised per treatment
Incidence of clinically significant vital signs abnormalities Over 21 days of dosing Summarised per treatment
Trial Locations
- Locations (18)
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
🇧🇪Liege, 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
Bicêtre University Hospital
🇫🇷Paris, France
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain