An intermediate access protocol for selumetinib for treatment of neurofibromatosis type 1 with inoperable, progressive/symptomatic plexiform neurofibromas

Purpose

To provide access to selumetinib to people with neurofibromatosis type 1 (NF1) with plexiform neurofibromas that cannot be operated on.

Criteria

Patients ≥ 2 years old with disease before the age of 18 years old

Diagnosis of neurofibromatosis type 1 (NF1)

Plexiform neurofibromas that cannot be surgically completely removed

Details

There is no maximum duration for this study as long as it is deemed that a response is being obtained.

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Hematology Oncology Associates