Mirdametinib in Infants and Toddlers With NF1-Associated Plexiform Neurofibromas

Phase 1, open-label trial of mirdametinib in plexiform neurofibroma (PN) (2 cohorts), sponsored by SpringWorks Therapeutics. Primary endpoints: Number of Participants With Treatment-emergent Adverse Events, Number of Participants With Clinically Significant Changes in Laboratory Parameters, Vital Signs, Physical Examination Findings and Electrocardiograms (ECGs), Echocardiogram (ECHO), and Ophthalmic… and 4 more. Planned start Dec 2026; status: not yet recruiting.