Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)
Stopped: slow accrual
0Started 2017-04-07
Plain-language summary
Background:
Gastrointestinal stromal tumors (GIST) can cause serious medical problems. The only known treatment is surgery. But completely removing a GIST tumor with surgery is often not possible. Researchers want to see if a new drug, selumetinib, can help treat these tumors.
Objective:
To find out if selumetinib shrinks or slows the growth of GIST tumors and to see its side effects.
Eligibility:
People ages 3 and over who have one or more GIST tumors and may have neurofibromatosis type I (also called NF1). Their NF1 GIST has shown some growth or cannot be completely removed with surgery.
Design:
Participants will be screened with heart and eye tests and scans.
Participants will be told what foods and medicines they cannot take during the study. Participants will keep a diary of the medicine they take during the study.
Participants will take selumetinib capsules twice daily on an empty stomach for 28 days in a row. This is 1 cycle.
During the cycles, participants will have study visits. These may include:
Medical history
Physical exam
Blood and urine tests
Heart tests
Scans of their tumors
Eye exam
Positron emission tomography scan. They will be get radioactive glucose an IV line. They will lie quietly in a darkened room for 50-60 minutes then have the scan.
Participants will answer questions about how they are feeling.
Participants can stay in the study until they have bad side effects or their tumor grows.
After finishing treatment, participants will be watched for side effects for 30 days.
Who can participate
Age range
3 Years – 99 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.
* ELIGIBILITY CRITERIA:
* Age: greater than or equal to 3 years of age, BSA greater than or equal to 0.55 m\^2, and able to swallow intact capsules.
* Diagnosis: must have either a clinical diagnosis of NF1 or a germline NF1 mutation, or in patients without the NF1 syndrome, demonstrate an NF1 mutation in the GIST verified in a CLIA certified laboratory. In patients without the NF1 syndrome, confirmation of the NF1 mutation in the GIST is required for enrollment.
* a) For a clinical diagnosis of NF1 patients must have at least two of the diagnostic criteria for NF1 listed below
* Six or more cafe-au-lait macules (greater or equal to 0.5cm in prepubertal subjects or greater than or equal to 1.5 cm in post pubertal subjects)
* Freckling in axilla or groin
* A neurofibroma or plexiform neurofibroma
* Optic glioma
* Two or more Lisch nodules
* A distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
* A first-degree relative with NF1
* Patients must have a histologically or cytologically confirmed measurable GIST without PDGFRA or KIT mutations. GIST may be newly diagnosed or recurrent provided that it meets criteria for progressive or metastatic disease. Metastatic disease refers to disease outside the GI tract, not simply a multifocal primary tumor. Testing performed by the Laboratory of Pathology, NCI, unless previously conducted by a CLIA/CAP external laboratory; analysis will include evaluation of…
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.