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.

Clinicaltrials.gov identifier: NCT03041701
NCI Protocol ID: NCI-17-C-0084
Official Title: A Phase I/II Trial of the Insulin-Like Growth Factor 1 Receptor (IGF-1R) Antibody AMG479 (Ganitumab) in Combination With the Src Family Kinase (SFK) Inhibitor Dasatinib in Patients With Embryonal and Alveolar Rhabdomyosarcoma

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Summary Posted: 11/2018