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RecruitingInterventionalPhase 2

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

NCT ID: NCT06414954Sponsor: NMD Pharma A/SLast updated: 2026-04-13

Summary

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.

Arms & interventions

  • DrugNMD670

    Tablets taken twice a day for 21 days

  • DrugPlacebo

    Tablets taken twice a day for 21 days

Outcome measures

Primary

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

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

    Time frame: Baseline to day 21

Secondary

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

    Time frame: Baseline to day 21

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

    Time frame: Baseline to day 21

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

    Time frame: Baseline to day 21

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

    Time frame: Baseline to day 21

  • Incidence of treatment emergent adverse event

    Time frame: Over 21 days of dosing

  • Incidence of serious treatment emergent adverse events

    Time frame: Over 21 days of dosing

  • Incidence of clinically significant abnormalities on physical examinations

    Time frame: Over 21 days of dosing

  • Incidence of clinically significant abnormalities on safety laboratory parameters

    Time frame: Over 21 days of dosing

  • Incidence of clinically significant vital signs abnormalities

    Time frame: Over 21 days of dosing

  • Incidence of clinically significant ECG abnormalities

    Time frame: Over 21 days of dosing

  • Incidence of Suicidal Ideation or Suicidal Behavior

    Time frame: Over 21 days of dosing

  • Incidence of clinically significant abnormalities on opthalmological examinations

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

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No
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

Study locations (11)

Profound Research LLC

Carlsbad, California, 92011

Withdrawn

University of California Irvine Medical Center

Irvine, California, 92868

Recruiting
Siddhant Partap · Contact
Ali Amer Habib · Principal Investigator

University of Colorado Neuromuscular Division

Aurora, Colorado, 80045

Recruiting
Alexis Shepardson · Contact
Stacy Dixon, MD · Principal Investigator

SFM Clinical Research, LLC

Boca Raton, Florida, 33487

Recruiting
Gabrielle DeMaria · Contact
Marc Feinberg, MD · Principal Investigator

Neuromuscular Research Division | University of South Florida

Tampa, Florida, 33612

Recruiting
Jessica Shaw · Contact
Tuan H Vu · Principal Investigator

Augusta University, Neuroscience Center

Augusta, Georgia, 30912

Recruiting
Michael Rivner · Principal Investigator

NextGen Precision Health

Columbia, Missouri, 65212

Recruiting
David Arnold · Principal Investigator

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599

Recruiting
James Howard · Principal Investigator

The Ohio State University

Columbus, Ohio, 43221

Recruiting
Lischele Watkins,, MACPR · Contact

University of Oregon

Portland, Oregon, 97239

Recruiting
Katie Lewis · Contact
Nizar Chahin · Principal Investigator

Semmes Murphey Clinic

Memphis, Tennessee, 38120

Recruiting
Courtney Berryman · Contact
Tulio Bertorini · Principal Investigator

References

  • Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.(PubMed)