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

What is the Purpose of this Study?

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).

What is the Condition Being Studied?

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

Who Can Participate in the Study?

-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.

Age Group
Adults, Children

What is Involved?

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)

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

Study Details

Full Title
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
Principal Investigator
Pediatric Hematology-Oncology Specialist
Protocol Number
IRB: PRO00103213
NCT: NCT03962543
Phase II
Enrollment Status
Open for Enrollment
Contact the Duke Recruitment Innovation Center