Center for Precision Medicine for High-Risk Pediatric Cancer Clinical Studies

This study involves taking a study drug called ALRN-6924. ALRN-6924 is an investigational cancer treatment that activates p53, which suppresses the growth of tumors by blocking proteins in the cell called MDM2 and MDMX that inhibit p53 from doing its job. The overall goals of this study are to evaluate the side effects and benefits of ALRN-6924 in patients with relapsed or treatment refractory solid tumor or brain tumor, and to determine the best dose of ALRN-6924 for children and adolescents. This study will also look at how the body breaks down ALRN-6924 by measuring the amount of ALRN-6924 in the blood over time after a dose. 

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. 

The purpose of this study is to find out what is the right dose of copanlisib in children and adolescents, and if your disease responds to this new drug.

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.