An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable Progressive/Symptomatic Plexiform Neurofibromas (PN)
The purpose of this study is to provide compassionate access to selumetinib capsules to patients for the treatment of NF1.
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit . Approximately 100 patients in the US will be treated as part of this protocol.
- IRB Number: 1708890674 (PHO-SHIH-ASTRA-SELUMETINI)
- Research Study Identifier: TX7986
- Principal Investigator: Michael Ferguson, MD
Contact the research team to learn more about this study.
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