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Study on the Safety and Effectiveness of NMD670 for Adults with Charcot-Marie-Tooth Disease Types 1 and 2

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Charcot-Marie-Tooth Disease (CMT), specifically types 1 and 2. CMT is a group of inherited disorders that affect the peripheral nerves, which are responsible for movement and sensation in the limbs. The trial will evaluate a new treatment called NMD670, which is taken as a tablet. The purpose of the study is to assess how effective and safe NMD670 is for patients with CMT.

Participants in the study will be randomly assigned to receive either the NMD670 tablet or a placebo, which looks like the NMD670 tablet but does not contain the active ingredient. The study will last for 21 days, during which participants will take the tablets daily. Throughout the study, participants will undergo various assessments to monitor their progress and any changes in their condition.

The main focus of the study is to observe changes in the distance participants can walk over a set period, as well as the time it takes to complete certain walking tasks. These assessments will help determine the effectiveness of NMD670 in improving mobility and overall function in individuals with Charcot-Marie-Tooth Disease. The study aims to provide valuable insights into the potential benefits of NMD670 for people living with this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of Charcot-Marie-Tooth Disease Type 1 or 2, and physical capabilities without assistive devices.

    Participants must have a stable medication regimen for muscle cramps and pain, and meet specific body mass index and weight criteria.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either the NMD670 tablet or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  3. Step 3

    Treatment phase

    The treatment phase lasts for 21 days. Participants take the assigned tablet orally each day. The dosage and specific instructions are provided by the study team.

    Participants continue their usual physical or occupational therapy regimen if applicable, and maintain a stable dose of any current medications for muscle cramps or pain.

  4. Step 4

    Monitoring and assessments

    Throughout the study, participants undergo regular assessments to monitor the effects of the treatment. This includes measuring changes in walking distance and time using specific tests such as the 6-minute walk test (6MWT) and the timed up and go test (TUG).

    These assessments help evaluate the efficacy and safety of NMD670 compared to the placebo.

  5. Step 5

    Completion and follow-up

    At the end of the 21-day treatment period, a final assessment is conducted to gather data on the primary endpoints, which include changes in walking distance and time.

    Participants may be asked to attend follow-up visits to ensure their well-being and to collect additional data if necessary.

Who can join the trial?

9 criteria

  • Participants must be men or women aged between 18 and 70 years old at the time of signing the informed consent form.
  • Participants must have a diagnosis of **Charcot-Marie-Tooth Disease** (CMT) type 1 or 2, confirmed by genetic testing.
  • Participants must meet certain physical capabilities without using assistive devices like **ankle-foot orthoses** (AFOs), although orthopedic inserts are allowed. This includes:
    • Walking a distance within 1 standard deviation of the average for their age and sex in a test called the **6-Minute Walk Test** (6MWT).
    • Having a strength of 4 or less in both ankles when tested manually.
    • If participants are already receiving physical or occupational therapy or following a training plan for more than 30 days before screening, they should continue this treatment during the study. They cannot start new therapy or training within 30 days of screening or during the study.
    • Participants taking medication for muscle cramps or pain should be on a stable dose for at least 30 days before screening and maintain this dose throughout the study.
    • Participants must have a **body mass index** (BMI) between 18 and 35 kg/m² and weigh at least 40 kg.
    • Female participants who can have children and male participants with partners who can have children must agree to use a highly effective method of contraception during the study.
    • Participants must be able to give signed informed consent, meaning they understand and agree to participate in the study.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Charcot-Marie-Tooth Disease Type 1 or Type 2** cannot participate. This is a genetic condition that affects the nerves.
  • Patients who are not within the specified age range cannot participate. The age range includes children and adults.
  • Patients who are not part of the specified clinical trial groups cannot participate. This refers to specific categories or groups of patients that the study is focusing on.
  • Both **male** and **female** patients are eligible, so exclusion is not based on gender.
  • Patients who are considered part of a **vulnerable population** may be excluded. This term refers to groups of people who might be at a higher risk of harm or exploitation.
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Investigated drugs

NMD670 is a medication being studied for its potential to help patients with Charcot-Marie-Tooth Disease Type 1 and Type 2. This trial aims to evaluate how effective, safe, and tolerable this medication is when taken over a period of 21 days. The goal is to see if NMD670 can improve the condition of patients as assessed by healthcare professionals.

What is already known about the treatment

NMD670 – This medication is administered orally and is currently being studied in clinical trials for its effectiveness in treating Charcot-Marie-Tooth Disease Types 1 and 2. It is in the Phase 2a stage of clinical trials, where its safety, efficacy, and tolerability are being evaluated. The main therapeutic indication for NMD670 is to improve the condition of patients with these types of Charcot-Marie-Tooth Disease. At the molecular level, NMD670 works by targeting specific pathways involved in nerve function, aiming to enhance nerve conduction and muscle strength. It is classified pharmacologically as a potential treatment for neuromuscular disorders.

Investigated diseases

  • Charcot-Marie-Tooth Disease Type 1

    This is a genetic disorder that affects the peripheral nerves, which are responsible for transmitting signals between the brain and the rest of the body. It typically begins with muscle weakness and atrophy in the feet and legs, progressing to the hands and arms. People with this condition may experience difficulty walking, loss of sensation, and foot deformities. The disease progresses slowly, and symptoms can vary widely among individuals. Over time, it may lead to difficulties with balance and coordination.

  • Charcot-Marie-Tooth Disease Type 2

    This is another form of Charcot-Marie-Tooth disease, also affecting the peripheral nerves but with different underlying genetic causes. It is characterized by muscle weakness and atrophy, primarily in the lower legs and feet, and can also affect the hands. Unlike Type 1, Type 2 involves damage to the nerve axons rather than the myelin sheath. Symptoms often include difficulty with fine motor skills and walking. The progression is gradual, and the severity of symptoms can differ significantly among those affected.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-507892-23-00Protocol codeNMD670-02-0003Estimated enrolment80 patientsSponsorNMD Pharma A/S

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