NF type1 With Inoperable Plexiform Neurofibromas Clinical Trial
Official title:
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)
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
Patients must have received a clinical diagnosis of NF1 and have inoperable,
progressive/symptomatic PN, where inoperable is defined as PN that cannot be surgically
completely removed without risk of substantial morbidity.
The population are patients with NF1 who have inoperable, progressive/symptomatic PN aged ≥
2years with onset of disease before they were 18 years and who have demonstrated an ability
to swallow whole capsules, who have no further treatment options and are not eligible for
clinical trials.
There is no maximum duration for selumetinib treatment. Patients may continue to receive
selumetinib as long as they continue to show clinical benefit, as judged by the treating
physician, and in the absence of unacceptable toxicity.
Once patients have been discontinued from treatment, other available treatment options will
be at the discretion of the physician
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