An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable Progressive/Symptomatic Plexiform Neurofibromas (PN)

Study Overview

The purpose of this study is to provide compassionate access to selumetinib capsules to patients for the treatment of NF1.

Study Description

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.

Additional Information

Participants will not be paid for their participation.

  • IRB Number: 1708890674 (PHO-SHIH-ASTRA-SELUMETINI)
  • Research Study Identifier: TX7986
  • Principal Investigator: Michael Ferguson, MD

Recruitment Status


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