NCT03962543

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 114 (actual)
• Sponsor: SpringWorks Therapeutics, Inc. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.

Detailed description

Neurofibromas are non-malignant peripheral nerve sheath tumors, which are classified as plexiform neurofibromas (PNs) if they extend longitudinally along a nerve and involve multiple fascicles. PNs are a major cause of morbidity and disfigurement in individuals with NF1, and as the tumor growth progresses, can cause a multitude of clinical problems including pain and impaired physical function. PNs have the potential to undergo malignant transformation to Malignant Peripheral Nerve Sheet Tumors (MPNST). Mirdametinib (PD-0325901) is an orally delivered, highly selective small-molecule inhibitor of the dual specificity kinases, MEK1 and MEK2 (MAPK/ERK Kinase) which prevents the phosphorylation and subsequent activation of mitogen-activated protein kinase (MAPK). Previous studies of mirdametinib (PD-0325901) demonstrated PN shrinkage and sustained inhibition of pERK. Reduced tumor volume indicated that cell proliferation or cell death may be altered in PNs with administration of mirdametinib (PD-0325901).

Conditions

• Plexiform Neurofibroma
• Neurofibromatosis Type 1 (NF1)

Interventions

Type	Name	Description
DRUG	Mirdametinib (PD-0325901) oral capsule or dispersible tablet	Mirdametinib (PD-0325901) capsule or dispersible tablet

Timeline

• Start date: 2019-09-29
• Primary completion: 2023-09-20
• Completion: 2028-12-22
• First posted: 2019-05-24
• Last updated: 2026-04-08
• Results posted: 2025-08-07

Locations

50 sites across 1 country: United States

Regulatory

• FDA-regulated drug study