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NCT Number: NCT06414954

Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis

This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.

Recruiting

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UZ Leuven, 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

  • Participant must be a male or female being 18 or more, at the time of signing the informed consent
  • Diagnosis of MG, MGFA class II, III or IV
  • Documented positive AChR or MuSK antibody test.
  • Participant must be able to swallow tablets
  • 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

  • Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study
  • Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
  • Participants that received treatment with an investigational medical product 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 MG therapy
  • Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding

Treatment and study plan

NMD670

Drug

Tablets taken twice a day for 21 days

Placebo

Drug

Tablets taken twice a day for 21 days

Primary outcomes

  1. Change from baseline to day 21 in QMG total score for NMD670 vs placebo

    Time frame: Baseline to day 21

    Scale goes from 0-39 and higher score indicates worse symptomatology

Secondary outcomes

  1. Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo

    Time frame: Baseline to day 21

    Scale goes from 0-24 and higher score indicates worse symptomatology

  2. Change from baseline to day 21 in MGC total score for NMD670 vs placebo

    Time frame: Baseline to day 21

    Scale goes from 0-50 and higher score indicate worse symptomatology

  3. Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo

    Time frame: Baseline to day 21

    Scale goes from 0-30 and higher score indicate worse quality of life

  4. Change from baseline to day 21 in Neuro-QoL Fatigue Short Form

    Time frame: Baseline to day 21

    Scale goes from 8-40 and higher score indicate worse symptomalogy

  5. Incidence of treatment emergent adverse event

    Time frame: Over 21 days of dosing

    Summarised per treatment

  6. Incidence of serious treatment emergent adverse events

    Time frame: Over 21 days of dosing

    Summarised per treatment

  7. Incidence of clinically significant abnormalities on physical examinations

    Time frame: Over 21 days of dosing

    Summarised per treatment

  8. Incidence of clinically significant abnormalities on safety laboratory parameters

    Time frame: Over 21 days of dosing

    Summarised per treatment

  9. Incidence of clinically significant vital signs abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  10. Incidence of clinically significant ECG abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  11. Incidence of Suicidal Ideation or Suicidal Behavior

    Time frame: Over 21 days of dosing

    Summarised per treatment

  12. Incidence of clinically significant abnormalities on opthalmological examinations

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

    Summarised per treatment

Study contacts

Contact information is provided by the study sponsor or research team.

NMD Pharma A/S

CONTACT

[email protected]

[email protected]

Sponsors and collaborators

Lead sponsor

NMD Pharma A/S

Industry

Registry information

Official study title

A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 Over 21 Days in Adult Patients With AChR/MuSK-Ab+ Myasthenia Gravis

Acronym: SYNAPSE-MG

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 16, 2024
Registry last updated
Jul 1, 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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