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

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

  • Clinical Trial Information

    Trial Contact: Spinelli, Jennifer; El-Shami, Jessica; Leffin, Melissa; Cevallos, Claudia

  • IRB No: 19.252.09

    Protocol Abbrev: MEK-NF-201

    Principal Investigator: Kalpana Vikram Patel, MD

    Phase: Drug: Phase II

    Age Group: Pediatric

    Secondary Protocol No: MEK-NF-201

    Treatment: Drug: PD-0325901 oral capsule

    Therapies Involved: Medication

    ClinicalTrials.gov ID: NCT03962543

  • Objective

    To evaluate the confirmed complete and
    partial response (PR) rate of PD-0325901
    using volumetric magnetic resonance imaging
    (MRI) analysis in participants with an
    inoperable NF1-associated PN that is
    progressing or causing significant morbidity.

  • Key Eligibility

    •  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.
    •  Participant can swallow capsules whole.