NCT07521657

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 55 (estimated)
• Sponsor: University of Alabama at Birmingham · Academic / Other
• Sex: All
• Age: 12 Years – 89 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a newly diagnosed GBM (Cohort 2); mirdametinib alone in other NF1-associated High-Grade Gliomas (Cohort 3).

Detailed description

Cohorts 1 and 2: Eligible participants will receive radiotherapy concurrently with twice daily mirdametinib. Mirdametinib will continue after completion of radiation in continuous 28-day cycles with treatment lasting up to 24 cycles. MR imaging to assess response will be performed 4 weeks after completion of radiation therapy, and every 2 months thereafter using Response Assessment in Neuro-Oncology (RANO) 2.0 enhancing criteria. Cohort 3: Eligible participants will receive twice-daily mirdametinib in 28-day cycles for 6 cycles. MR imaging to assess response by Response Assessment in Neuro-Oncology (RANO) 2.0 non-enhancing criteria will be performed every 3 months thereafter to determine radiographic response. Participants with ongoing benefit may remain on therapy for up to 24 cycles following evaluation of the primary endpoint as determined by the treating physician.

Conditions

• Neurofibromatosis 1 (NF1)

Interventions

Type	Name	Description
COMBINATION_PRODUCT	mirdametinib with radiation	Participants in Cohorts 1 and 2 will receive mirdametinib in combination with radiation therapy followed by mirdametinib alone.
DRUG	mirdametinib	Participants in Cohort 3 will receive mirdametinib alone.

Timeline

• Start date: 2026-08-01
• Primary completion: 2030-08-01
• Completion: 2031-08-01
• First posted: 2026-04-13
• Last updated: 2026-04-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study