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Study of NMD670 tablets to improve muscle strength in adults with Type 3 spinal muscular atrophy who can walk

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What is this trial about?

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

This study focuses on people with Type 3 spinal muscular atrophy, a genetic condition that causes muscle weakness and affects movement ability. The research evaluates a new medication called NMD670, which works by affecting muscle channels to improve communication between nerves and muscles. The study aims to determine if this medication can help improve muscle strength and function in people who can walk.

The medication is given as tablets that participants take by mouth twice daily for 21 days. During the study, some participants will receive NMD670 tablets while others will receive placebo tablets. After a break period, participants who first received the study medication will switch to placebo, and those who started with placebo will receive NMD670. This type of study design helps researchers better understand how well the medication works.

The main focus is on measuring how far participants can walk in 6 minutes before and after taking the medication. The study also looks at other aspects of muscle function and strength, as well as the safety of the medication. Throughout the study, participants' health will be monitored through various medical tests and examinations to ensure their safety and evaluate how well the treatment is working.

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 treatment period

    You will receive either NMD670 tablets or placebo tablets for 21 days

    If assigned to NMD670, you will take 400 mg twice daily by mouth

    The tablets should be taken at regular times each day

  2. Step 2

    Measurements and tests - first period

    Your walking ability will be measured using the 6-minute walk test

    Your muscle strength will be tested using special measuring equipment

    Medical staff will perform nerve and muscle tests

    Regular health checks will include physical examinations, blood tests, heart tests (ECG), and vital signs monitoring

  3. Step 3

    Switch to alternative treatment

    After the first 21-day period, you will switch to the other treatment option

    If you received NMD670 first, you will now receive placebo, or vice versa

  4. Step 4

    Second treatment period

    The second 21-day treatment period will follow the same pattern as the first

    You will take the medication twice daily by mouth

    The same tests and measurements will be repeated during this period

  5. Step 5

    Final evaluations

    Your walking ability and muscle strength will be measured again

    Final health checks will be performed

    The study team will assess all collected data to evaluate the medication's effectiveness

Who can join the trial?

12 criteria

  • Age between 18 and 75 years at the time of signing consent
  • Must have Type 3 spinal muscular atrophy (SMA) confirmed by clinical diagnosis
  • Must be able to walk at least 50 meters without assistance during a 6-minute walk test
  • Must have genetic confirmation of diagnosis through testing of the SMN1 gene (a gene important for muscle function)
  • Must have 2 to 5 copies of the SMN2 gene (another gene important for muscle function)
  • Must have a specific score (less than 80%) on a muscle function test called MFM-32 dimension 1
  • Must show certain muscle response measurements during nerve testing (7% or greater CMAP amplitude decrease)
  • Body Mass Index (BMI) must be between 19 and 35 (BMI is a measure of body weight in relation to height)
  • Both men and women may participate if they meet contraceptive requirements:
    • Men must use effective contraception and not donate sperm during the study
    • Women must either be unable to bear children or use effective contraception during the study
    • Women must not be pregnant or breastfeeding
    • Must be able to understand and sign informed consent documents

Who cannot join the trial?

12 criteria

  • Age below 18 or above 65 years
  • Not having a confirmed diagnosis of Type 3 Spinal Muscular Atrophy (SMA)
  • Currently participating in other clinical trials
  • Pregnancy or breastfeeding
  • Having significant medical conditions that could interfere with the study, such as:
    • Severe heart problems
    • Severe kidney or liver disease
    • Uncontrolled high blood pressure
    • Taking medications that could interact with the study drug
    • History of allergic reactions to similar medications
    • Unable to follow study procedures or attend scheduled visits
    • Having had major surgery within 3 months before the study
    • Having a history of substance abuse within the past year
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Investigated drugs

NMD670 is an investigational medication being studied for the treatment of spinal muscular atrophy (SMA). This medication is being tested to see if it can help improve muscle strength and function in people who are able to walk and have Type 3 SMA. The medication is taken twice daily and is being compared to a placebo to determine how well it works and how safe it is for patients.

What is already known about the treatment

NMD670 – An investigational drug being studied in clinical trials for the treatment of Type 3 spinal muscular atrophy (SMA) in ambulatory adult patients. The medication is administered orally twice daily at a dose of 400 mg and is currently being evaluated in a Phase 2 clinical trial to assess its effectiveness in improving muscle strength and function. While the exact molecular mechanism is not publicly disclosed in the trial data, this experimental compound is being tested as a potential therapeutic option for patients who can still walk independently despite having SMA, a genetic condition affecting muscle strength and movement.

Investigated diseases

Type 3 Spinal Muscular Atrophy - A genetic neuromuscular disorder that affects motor neurons in the spinal cord, leading to muscle weakness and atrophy. This form of SMA typically appears after 18 months of age, allowing children to achieve the ability to walk independently. The condition causes progressive weakness in leg, hip, shoulder, and respiratory muscles. People with Type 3 SMA experience difficulties with walking, climbing stairs, and rising from sitting positions, though the progression rate varies among individuals. The muscle weakness is typically symmetrical and more severe in legs than arms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-516321-31-00Protocol codeNMD670-02-0001Estimated enrolment93 patientsSponsorNMD Pharma A/S

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