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

Description

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.

Conditions

Charcot-Marie-Tooth Disease

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Charcot-Marie-Tooth Disease
Intervention / Treatment

-

Contacts and Locations

Columbia

NextGen Precision Health, Columbia, Missouri, United States, 65211

Columbus

OSU Department of Neurology Division of Neuromuscular Diseases, Columbus, Ohio, United States, 43221

Austin

National Neuromuscular research Institute, PLLC, Austin, Texas, United States, 78759

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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.

Ages Eligible for Study

18 Years to 70 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

NMD Pharma A/S,

Study Record Dates

2026-03-16