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Clinical Trials Search at Vanderbilt-Ingram Cancer Center

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).
Phase II
Mol. targeted/Immunotherapy/Biologics
Moots, Paul
Vanderbilt University


2 Years
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.

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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