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    Association details:
    Biomarker:NF1 mutation
    Cancer:CNS Tumor
    Drug:Koselugo (selumetinib) (MEK inhibitor)
    Direction:Sensitive
    Evidence:
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    Source:
    clinicaltrials.gov
    Title:

    Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

    Excerpt:
    ...- Patients be >= 18 years old at the time of enrollment and must have a documented germline neurofibromatosis 1 (NF1) mutation in a Clinical Laboratory Improvement Act (CLIA) certified laboratory or a diagnosis of NF1 based on clinical National Institutes of Health (NIH) consensus criteria; in addition to substantial cutaneous neurofibroma burden, at least one of the criteria below have to be present...
    Trial ID:
    NCT02839720
    Evidence Level:
    Sensitive: C3 – Early Trials
    Source:
    Lancet Oncol
    Title:

    Selumetinib in Children with BRAF-Aberrant or Neurofibromatosis type 1-Associated Recurrent, Refractory or Progressive Low-Grade Glioma: a Multi-Center Phase II Trial

    Excerpt:
    ...Patients with an optic pathway tumor enrolled to stratum 3...Stratum 3 included children with any neurofibromatosis type 1 (NF1)…On stratum 3, 10/25 (40%) patients achieved a PR with a median follow-up of 48.6 months (8.6–59.1; IQR=12.2) for the 17 subjects without progressions...
    DOI:
    10.10162FS1470-2045(19)30277-3
    Trial ID:
    NCT01089101