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

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Conditions

Charcot-Marie-Tooth Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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 2.

Official Title

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

Quick Facts

Study Start:2024-09-30
Study Completion:2025-11-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:COMPLETED

Study ID

NCT06482437

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.

Ages Eligible for Study:18 Years to 70 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female participants must be 18 to 70 years inclusive at the time of signing the ICF.
  2. * Diagnosis of CMT type 1 or 2 confirmed by genetic testing.
  3. * Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
  4. * Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  5. * Participant is capable of and has given signed informed consent
  1. * 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.
  2. * Participants with laboratory test result abnormalities at screening considered clinically significant by the Investigator.
  3. * 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.
  4. * Participants with history of poor compliance with relevant therapy in the opinion of the Investigator.
  5. * Female participants who plan to become pregnant during the study or are currently pregnant or breastfeeding.

Contacts and Locations

Study Locations (Sites)

University of Kansas Medical Center, Department of Neurology
Kansas City, Kansas, 66160
United States
Mass General Neurology
Boston, Massachusetts, 02114
United States
NextGen Precision Health
Columbia, Missouri, 65211
United States
Columbia University Medical Center
New York, New York, 10032
United States
University of Rochester Neuromuscular Disease Center
Rochester, New York, 14642
United States
OSU Department of Neurology Division of Neuromuscular Diseases
Columbus, Ohio, 43221
United States
National Neuromuscular research Institute, PLLC
Austin, Texas, 78759
United States
Providence Medical Research Center
Spokane, Washington, 99204
United States

Collaborators and Investigators

Sponsor: NMD Pharma A/S

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-30
Study Completion Date2025-11-04

Study Record Updates

Study Start Date2024-09-30
Study Completion Date2025-11-04

Terms related to this study

Additional Relevant MeSH Terms

  • Charcot-Marie-Tooth Disease