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

NCT ID: NCT06482437

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

81 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-30

Study Completion Date

2025-11-04

Brief Summary

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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.

Detailed Description

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Conditions

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Charcot-Marie-Tooth Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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NMD670

Group Type EXPERIMENTAL

NMD670

Intervention Type DRUG

Tablets taken twice daily for 21 days

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Tablets taken twice daily for 21 days

Interventions

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NMD670

Tablets taken twice daily for 21 days

Intervention Type DRUG

Placebo

Tablets taken twice daily for 21 days

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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NMD Pharma A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Kansas Medical Center, Department of Neurology

Kansas City, Kansas, United States

Site Status

Mass General Neurology

Boston, Massachusetts, United States

Site Status

NextGen Precision Health

Columbia, Missouri, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

University of Rochester Neuromuscular Disease Center

Rochester, New York, United States

Site Status

OSU Department of Neurology Division of Neuromuscular Diseases

Columbus, Ohio, United States

Site Status

National Neuromuscular research Institute, PLLC

Austin, Texas, United States

Site Status

Providence Medical Research Center

Spokane, Washington, United States

Site Status

University Hospitals Leuven, Department of Neurology

Leuven, , Belgium

Site Status

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

Liège, , Belgium

Site Status

Aarhus University Hospital

Aarhus, , Denmark

Site Status

Rigshospitalet, Department of Neurology

Copenhagen, , Denmark

Site Status

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

Marseille, , France

Site Status

Laboratoire d'Explorations Fonctionnelles, CHU Nantes

Nantes, , France

Site Status

Centre Hospitalier Universitaire De Nice

Nice, , France

Site Status

Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière

Paris, , France

Site Status

Bicêtre University Hospital

Paris, , France

Site Status

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

Site Status

Countries

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United States Belgium Denmark France Spain

Other Identifiers

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NMD670-02-0003

Identifier Type: -

Identifier Source: org_study_id