Clinical trial studies new therapy in children and adults with neurofibromatosis-related GIST

Boy playing with cars

Travis Carpenter, 9, received treatment for neurofibromatosis type 1 (NF1) in a phase II clinical trial run by the NCI Pediatric Oncology Branch at the NCI Center for Cancer Research. Here, he's at the NIH Children's Inn, a residence for families with children participating in NIH clinical trials.
Photo by Daniel Sone

Gastrointestinal stromal tumors (GIST) are solid tumors that can develop anywhere in the gastrointestinal (GI) tract. Neurofibromatosis type 1 (NF1) is a genetic disorder that causes tumors to form on nerve tissue and is usually diagnosed in children and young adults. Patients with NF1 have an increased risk of developing GIST. There is currently no effective medical therapy for GIST that cannot be completely resected. Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch, is leading a study to see if NF1-related GIST responds to treatment with selumetinib, which blocks a signaling protein called MEK. Selumetinib has been shown to shrink another NF1-related tumor called plexiform neurofibroma. Selumetinib is taken as capsules by mouth twice daily. Investigators hope that selumetinib will block the growth of NF1-related GIST or shrink these tumors. identifier: NCT03109301
NCI Protocol ID: NCI-17-C-0084
Official Title: A Phase II Trial of the Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Patients With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)

The Center for Cancer Research is NCI’s internal cancer center, a publicly funded organization working to improve the lives of cancer patients by solving important, challenging and neglected problems in cancer research and patient care. Highly trained physician-scientists develop and carry out clinical trials to create the medicines of tomorrow treating patients at the world’s largest dedicated research hospital on the campus of the National Institutes of Health in Bethesda, Maryland.   

For more information on CCR clinical trials click here, and subscribe to have the latest CCR clinical trials sent directly to your inbox.

Summary Posted: Thu, 11/01/2018