Center for Precision Medicine for High-Risk Pediatric Cancer Clinical Studies
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Tegavivint for the Treatment of Solid Tumors, Lymphomas & Desmoids
Uproleselan in Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or Mixed Phenotype Acute Leukemia (MPAL)
GD2CART for Osteosarcoma or Neuroblastoma
Selpercatinib in Solid or CNS tumors with RET-Alterations
The purpose of this study is to test the safety and efficacy of the investigational drug called Selpercatinib in children whose cancers have a specific genetic change (RET-fusion or RET-mutation). Selpercatinib has been FDA-approved for adults and pediatric patients 12 years of age or older for certain types of lung and thyroid cancers. Selpercatinib is administered in capsule form, liquid suspension, or may also be administered by a feeding tube for subjects who are unable to swallow.
Repotrectinib for ALK, ROS1, or NTRK1-3 Alterations
The purpose of this study is to test the safety and efficacy of the investigational drug called repotrectinib in children whose cancers have specific genetic changes (in the ALK, ROS1, or NTRK1-3 genes). This is the first study using this drug in children under 12 years of age. Repotrectinib is administered in capsule form or liquid suspension.
Palbociclib, Irinotecan, and Temozolomide in Ewing Sarcoma
The purpose of the Phase 2 portion of this study is to evaluate the safety and efficacy of the study drug called palbociclib when given in combination with two other drugs (temozolomide and irinotecan) in patients with Ewing sarcoma and when given in combination with topotecan and cyclophosphamide in patients with neuroblastoma.
Lutathera in Somatostatin Neuroendocrine Tumors
This study involves taking a study drug called Lutathera. The overall goal of this study is to see if Lutathera is safe in adolescent subjects with either a gastroenteropancreatic neuroendocrine tumor (GEP-NET), or a pheochromocytoma and/or paraganglioma (PPGL). This study will also look at the levels of radioactivity after Lutathera administration.
BA3011 for advanced solid tumors
This study involves taking a study drug called BA3011. The overall goal of this study is to see if BA3011 is a possible treatment for patients with sarcoma.