MEK-NF-201 (Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas) - Clinical Trial

We are doing this study to learn more about the study drug (mirdametinib, PD-0325901) and how it treats symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs).

Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs)

-Adults and children 2+
-Have a clinical diagnosis of NF1
- Have a target PN that is inoperable
-Has never received therapy with PD-0325901 or any other MEK1/2 inhibitor

For more information about who can participate in this study, please contact the study team.

If you choose to be in the study, you, or your child, will have a screening visit that will include:
- A blood draw
-Urine test
-Vision Test
-Physical exam
-Electrocardiogram (ECG)-measures your heart
-Echocardiogram (ECHO)-looks at your heart
-Magnetic resonance imaging (MRI)
-Questionnaires

Once you join the study you will:
-Take the study drug (Mirdametinib, PD-0325901) by mouth twice a day for up to 2 years (3 weeks on, 1 week off cycles)
-Come to the clinic once a month for 7 months, then every other month for the rest of the time they are in the study