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

NCT06482437 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: NMD670-02-0003
• Study Type: interventional
• Target: 81
• Locations: 5 countries
• Sites: 19

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 2.

Conditions

Charcot-Marie-Tooth Disease

Outcome Measures

Primary Outcomes (1)

• Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo

  Baseline to day 21

Secondary Outcomes (20)

• Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo

  Baseline to day 21

• Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo

  Baseline to day 21

• 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 Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo

  Baseline to day 21

• Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo

  Baseline to day 21

Study Arms (2)

NMD670

EXPERIMENTAL

Drug: NMD670

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

NMD670 DRUG

Tablets taken twice daily for 21 days

NMD670

Placebo DRUG

Tablets taken twice daily for 21 days

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

Sponsors & Collaborators

• NMD Pharma A/S: lead

Study Sites (19)

University of Kansas Medical Center, Department of Neurology

Kansas City, Kansas, 66160, United States

Location

Mass General Neurology

Boston, Massachusetts, 02114, United States

Location

NextGen Precision Health

Columbia, Missouri, 65211, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

University of Rochester Neuromuscular Disease Center

Rochester, New York, 14642, United States

Location

OSU Department of Neurology Division of Neuromuscular Diseases

Columbus, Ohio, 43221, United States

Location

National Neuromuscular research Institute, PLLC

Austin, Texas, 78759, United States

Location

Providence Medical Research Center

Spokane, Washington, 99204, United States

Location

University Hospitals Leuven, Department of Neurology

Leuven, 3000, Belgium

Location

CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1

Liège, 4000, Belgium

Location

Aarhus University Hospital

Aarhus, Denmark

Location

Rigshospitalet, Department of Neurology

Copenhagen, Denmark

Location

CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases

Marseille, 13005, France

Location

Laboratoire d'Explorations Fonctionnelles, CHU Nantes

Nantes, France

Location

Centre Hospitalier Universitaire De Nice

Nice, 06001, France

Location

Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière

Paris, 75651, France

Location

Bicêtre University Hospital

Paris, 94275, France

Location

Hospital Universitari Vall d'Hebron

Barcelona, 8035, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2024
• First Posted: July 1, 2024
• Study Start: September 30, 2024
• Primary Completion: October 28, 2025
• Study Completion: November 4, 2025
• Last Updated: November 12, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

ES (2); FR (5); BE (2); DK (2); US (8)