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OpenTrials
Completed

NCT Number: NCT05794139

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UZ Leuven - Neurochirurgie Campus Gasthuisberg, Leuven, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants with a clinical diagnosis of Type 3 SMA.
  • Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids at screening during the 6-minute walk test.
  • Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])
  • Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
  • Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
  • Participant is male or female.
  • 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 giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.

Exclusion criteria

  • Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
  • Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
  • Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
  • Participants received treatment with an investigational medical product (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 SMA therapy.

Treatment and study plan

NMD670

Drug

Tablets

Placebo

Drug

Tablets

Primary outcomes

  1. Change from baseline in 6 minute walk test (6MWT) total distance versus placebo

    Time frame: Baseline to day 21

    Distance walked (meters)

Secondary outcomes

  1. Change from baseline in muscle strength versus placebo

    Time frame: Baseline to day 21

    Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)

  2. Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo

    Time frame: Baseline to day 21

    percentage change in distance walked in 6th minute compared to 1st minute

  3. Change from baseline in Revised Hammersmith Scale (RHS) versus placebo

    Time frame: Baseline to day 21

    Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms

  4. Change from baseline in jitter versus placebo

    Time frame: Baseline to day 21

    Jitter (micro seconds) assessed with single fiber EMG

  5. Change from baseline in blocking versus placebo

    Time frame: Baseline to day 21

    Blocking (%) assessed with single fiber EMG

  6. Incidence of treatment emergent adverse events

    Time frame: Over 21 days of dosing

    Summarised per treatment

  7. Incidence of serious treatment emergent adverse events

    Time frame: Over 21 days of dosing

    Summarised per treatment

  8. Incidence of clinically significant abnormalities on physical examinations

    Time frame: Over 21 days of dosing

    Summarised per treatment

  9. Incidence of clinically significant abnormalities on safety laboratory parameters

    Time frame: Over 21 days of dosing

    Summarised per treatment

  10. Incidence of clinically significant vital signs abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  11. Incidence of clinically significant ECG abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  12. Incidence of Suicidal Ideation or Suicidal Behavior

    Time frame: Over 21 days of dosing

    Summarised per treatment

  13. Incidence of clinically significant abnormalities on opthalmological examinations

    Time frame: Over 21 days of dosing

    Summarised per treatment

Sponsors and collaborators

Lead sponsor

NMD Pharma A/S

Industry

Registry information

Official study title

A Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy

Acronym: SYNAPSE-SMA

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Apr 3, 2023
Registry last updated
Jun 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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