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

Trial ID or NCT#

NCT03962543

Status

not recruiting iconNOT RECRUITING

Purpose

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). Eligible participants may continue in a long-term follow-up phase.

Official 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

Eligibility Criteria

Ages Eligible for Study: Older than 2 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * 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.
    1. Key
Exclusion Criteria:
  1. * 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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Investigator(s)

Cynthia Campen

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Contact

Erica Velasco, SC

View on ClinicalTrials.gov