An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable Progressive/Symptomatic Plexiform Neurofibromas (PN)
Michael Ferguson, MD
Primary Investigator
Brief description of study
The purpose of this study is to provide compassionate access to selumetinib capsules to patients for the treatment of NF1.
Detailed description of study
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.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: neurofibromatosis, neurofibroma
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Age: Between 2 Years - 100 Years
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Gender: All
Inclusion Criteria
Diagnosis of neurofibromatosis type 1 (NF1)
Patients aged greater than or equal to 2 with onset of disease before they were 18 years
Able to swallow whole capsules
Normal cardiac function
Adequate blood pressure
The patient has exhausted all available approved therapies as appropriate for NF1
Exclusion Criteria
Prior treatment with MEK inhibitor, unless enrolling after receiving selumetinib through single patient access
Patient eligible for any ongoing clinical trials with selumetinib in the indication in question
Ophthalmological conditions
Breast-feeding
Have evidence of any other significant clinical disorder or laboratory finding that, as judged by the treating physician, makes it undesirable for the patient to participate in the study
Have any evidence of a severe or uncontrolled systemic disease, active bleeding diathesis or renal transplant
Have refractory nausea and vomiting, chronic gastrointestinal diseases, or significant bowel resection that would adversely affect the absorption/bioavailability of the orally administered study medication
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