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
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
Study locations (11)
Profound Research LLC
Carlsbad, California, 92011
University of California Irvine Medical Center
Irvine, California, 92868
University of Colorado Neuromuscular Division
Aurora, Colorado, 80045
SFM Clinical Research, LLC
Boca Raton, Florida, 33487
Neuromuscular Research Division | University of South Florida
Tampa, Florida, 33612
Augusta University, Neuroscience Center
Augusta, Georgia, 30912
NextGen Precision Health
Columbia, Missouri, 65212
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
The Ohio State University
Columbus, Ohio, 43221
University of Oregon
Portland, Oregon, 97239
Semmes Murphey Clinic
Memphis, Tennessee, 38120
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)