An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 (NCT03259633) | Clinical Trial Compass
APPROVED_FOR_MARKETINGNot Applicable
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
United States
Plain-language summary
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
Who can participate
Age range
2 Years – 130 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of neurofibromatosis type 1 (NF1) and have inoperable, progressive/symptomatic plexiform neurofibromas (PN)
. Presence of inoperable PN , defined as a PN that cannot be surgically completely removed without risk for substantial morbidity
. Patients aged ≥2 with onset of disease before they were 18 years and a BSA ≥ 0.55 m2 who are able to swallow whole capsules. (approx. length 15.4 mm, diameter 5.4 mm). A swallow test must be performed before requesting drug
. Normal cardiac function defined as normal ejection fraction (ECHO, MUGA or cardiac MRI) as per institutional normal and absence of prior heart disease
. Adequate blood pressure as defined in line with local practice.
. The patient has exhausted all available approved therapies as appropriate for NF1 with inoperable progressive/symptomatic PN
. Provision of a signed informed consent prior to any protocol specific procedures. Patients already receiving selumetinib through single patient access who enroll in this protocol must be reconsented and sign the consent form for this intermediate access protocol.
. For female patients of childbearing potential, have evidence of a post-menopausal status, or a negative urinary or serum pregnancy test.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Patients eligible for any ongoing clinical trials with selumetinib in the indication in question
. Ophthalmological conditions: Current or past history of retinal pigment epithelial detachment (RPED)/central serous retinopathy (CSR) or retinal vein occlusion Intraocular pressure (IOP) should not be \> 21 mmHg for adults or outside the range of normal for children or uncontrolled glaucoma (irrespective of IOP)
. Male or female patients of reproductive potential and, as judged by the investigator, are not employing an effective method of birth control.
. Female patients who are 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 (e.g. unstable or uncompensated respiratory, cardiac, hepatic, or renal disease, active infection (including hepatitis B, hepatitis C, HIV), active bleeding diatheses or renal transplant
. Have refractory nausea and vomiting, chronic gastrointestinal diseases (e.g., inflammatory bowel disease), or significant bowel resection that would adversely affect the absorption / bioavailability of the orally administered study medication