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

Recruitment & Eligibility

Status: Active, not recruiting

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

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.

Exclusion Criteria

Not provided

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Complete or partial response rate at the end of the Treatment Phase compared to baseline. Partial response is defined as a = 20% reduction in target tumor volume.

Secondary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events.;Duration of response (DOR) for participants who meet criteria for objective response rate.;Change from Baseline on quality of life (QOL) as measured by the Pediatric Quality of Life Inventory (PedsQL), Acute version.;Change from Baseline in pain as measured by the Numeric Rating Scale-11 (NRS-11).;Change from Baseline in pain as measured by the Pain Interference Index (PII).