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

NMD Pharma A/S81 enrolled

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 2.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Charcot-Marie-Tooth Disease

Interventions

NMD670DRUG

Tablets taken twice daily for 21 days

PlaceboDRUG

Tablets taken twice daily for 21 days

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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.

Locations (19)

University of Kansas Medical Center, Department of Neurology

Kansas City, Kansas, United States

Outcomes

Primary Outcomes

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

Time frame: Baseline to day 21

Secondary Outcomes

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

CMT Functional Outcome Meausre is a 12-item scale. Scale goes from 0-100 and a higher score indicates worse symptomatology

Time frame: Baseline to day 21

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

Time frame: Baseline to day 21

Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo

Time frame: Baseline to day 21

Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo

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

Time frame: Baseline to day 21

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

The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology

Time frame: Baseline to day 21

Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo

The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology

Time frame: Baseline to day 21

Data from ClinicalTrials.gov

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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

Liège, Belgium

...and 9 more locations

Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo

Time frame: Baseline to day 21

Incidence of treatment emergent adverse events

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of serious treatment emergent adverse events

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of clinically significant abnormalities on physical examinations

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of clinically significant abnormalities on safety laboratory parameters

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of clinically significant vital signs abnormalities

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of clinically significant ECG abnormalities

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of Suicidal Ideation or Suicidal Behavior

Summarised per treatment

Time frame: Over 21 days of dosing

Incidence of clinically significant abnormalities on opthalmological examinations

Summarised per treatment

Time frame: From screening (day -28 to day -1) until follow up (day 28)]

Proportion of participants with clinically meaningful change from baseline in CMT-FOM total score and individual items for NMD670 vs placebo

Time frame: Baseline to day 21

Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo

Time frame: Baseline to day 21

Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo

Time frame: Baseline to day 21

Proportion of participants with clinically meaningful change from baseline in CMT-HI total score and individual domains for NMD670 vs placebo

Time frame: Baseline to day 21

Proportion of participants with clinically meaningful change from baseline in SF-36 total score and individual domains for NMD670 vs placebo

Time frame: Baseline to day 21