This study enrolls patients that have been diagnosed with acute leukemia associated with a KMT2A (MLL) gene rearrangement (referred to as KMT2Arearranged, or KMT2A-R). The KMT2A-R leukemia has come back after treatment (relapsed) or is not responding to treatment (it is refractory). This study involves taking a study drug called SNDX-5613. The study is divided into two phases: one in which the study drug is given in combination with chemotherapy and a phase where the study drug is given alone. The purpose of the study is to test the safety of the study drug with chemotherapy, and how well the treatment regimen works for infants and young children with relapsed or refractory KMT2A-R leukemia.

This study has several parts and involves taking a study drug called alpelisib. Some participants enrolled into Stage 2 will receive a placebo for the first part of the study (24 weeks) and will then receive the study drug. The purpose of this study is to find out if the study drug is safe and effective (can help) people with a confirmed diagnosis of lymphatic malformation (LyM) and a documented mutation in the PIK3CA gene

The purpose of this study is to find out if alpelisib (BYL719; the "study drug"), is safe and effective (can help) people who have PIK3CA-related overgrowth spectrum (PROS) disease.

CHOP, in collaboration with Onyx Therapeutics, Inc., is conducting a study for children and young adults up to 21 years old, with relapsed or refractory acute lymphoblastic leukemia. This clinical trial is adding the study drug, Carfilzomib, to a standard relapse chemotherapy regimen. For more information about this study, including specific eligibility criteria, please visit clinicaltrials.gov and search for "NCT02303821". To hear more about this and other available trails or get any questions answered please contact our Cancer Intake Specialist by phone at 267-426-0762 or email Oncointake@email.chop.edu.

This study enrolls patients that have been diagnosed with osteosarcoma or neuroblastoma that has come back (relapsed) or has not responded to standard treatments (refractory). This study involves an investigational cellular therapy called GD2CART. The study will be testing different doses of the study drug to find the safest dose.

This study involves taking a study drug called asciminib. The overall goal of the study is to find out how safe and effective the study drug is in treating people that have been diagnosed with Chronic Myeloid Leukemia (CML) and have not responded to treatment.

The purpose of this study is to see if a new investigational topical (applied to the skin) formulation of sirolimus, PTX-022 (the study drug), is safe and effective in treating participants with microcystic Lymphatic Malformations.

This study involves taking a study drug called SAR443579. The overall goal of this study is to find out if the study drug is safe and effective at treating adults and children with relapsed or refractory acute myeloid leukemia (R/R AML), high risk myelodysplastic syndrome (HR-MDS), or B-cell acute lymphoblastic leukemia (B-ALL). This is a first-in-human research study.

This study is for people with growing abnormal lymph vessels called lymphatic malformations (LMs). This study has 3 parts and involves taking a study drug called TARA-002. The purpose of this study is to find out if the study drug is safe and effective (can help) people with LMs. Participants receive the study drug by injection and will complete other research assessments throughout the study.

This study enrolls patients that have been diagnosed with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or mixed phenotype acute leukemia (MPAL) that has either come back (relapsed) or does not respond to therapy (refractory). This study involves taking a drug called uproleselan that is not yet approved for use in children or adults. The purpose of the study is to find a safe dose of the study drug in children that can be given with standard chemotherapy without causing severe side effects.