An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable Progressive/Symptomatic Plexiform Neurofibromas (PN)
Study on Investigational Medication for Neurofibromatosis Type 1
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,neurofibromas,plexiform neurofibroma,PN
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Age: 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
This study investigates the use of an investigational medication for patients with neurofibromatosis type 1 (NF1), a genetic disorder that causes tumors to form on nerve tissue. The study focuses on patients with inoperable, progressive, or symptomatic plexiform neurofibromas (PN), which are complex tumors associated with NF1. These patients have no alternative therapeutic options available.
Participants will receive the investigational medication in an open-label, single-arm setting, meaning all participants will receive the same treatment without a placebo. The study is designed to provide access to this medication as long as patients continue to benefit from it. Approximately 100 patients in the US will be involved.
- Who can participate: Patients aged 2 and older with neurofibromatosis type 1 (NF1) who have exhausted all approved therapies can participate. Participants must be able to swallow whole capsules and have normal cardiac function and adequate blood pressure.
- Study details: Participants will take the investigational medication in capsule form. The study is open-label, meaning all participants receive the medication, and there is no placebo group.
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