Open Actively Recruiting
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
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).
Key Inclusion Criteria:
- Participant has documented NF1 mutation or a diagnosis of neurofibromatosis type 1 (NF1) using National Institute of Health (NIH) Consensus Conference criteria inclusive of the presence of a plexiform neurofibroma (PN).
- Participant has a PN that is causing significant morbidity.
- Participant has a PN that cannot be completely surgically removed.
- Participant has a target tumor that is amenable to volumetric MRI analysis.
- Participant is willing to undergo a tumor biopsy pre and post treatment if ≥ 18 years of age.
- Participant has adequate organ and bone marrow function.
Key Exclusion Criteria:
- Participant has abnormal liver function or history of liver disease.
- Participant has lymphoma, leukemia or any malignancy within the past 5 years (except for resected basal/squamous skin carcinomas without metastases within 3 years).
- Participant has breast cancer within 10 years.
- Participant has active optic glioma or other low-grade glioma requiring treatment.
- Participant has abnormal QT interval corrected or other heart disease within 6 months.
- Participant has a history of retinal pathology, risk factors for retinal vein occlusion or has a history of glaucoma.
- Participant has known malabsorption syndrome or gastrointestinal conditions that would impair absorption of mirdametinib (PD-0325901).
- Participant has received NF1 PN-targeted therapy within 45 days.
- Participant previously received or is currently receiving therapy with mirdametinib (PD-0325901) or any other MEK1/2 inhibitor.
- Participant has received radiation therapy within 6 months or has received radiation to the orbit at any time.
- Participant is unable to undergo or tolerate MRI.
- Participant has active bacterial, fungal or viral infection.
- Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year.
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