WithdrawnPhase 2

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 range3 Years – 99 Years

SexALL

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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…

What they're measuring

Efficacy

Timeframe: End of treatment

Trial details

NCT IDNCT03109301

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2019-03-27