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 involves an investigational cellular therapy called Afamitresgene Autoleucel. The overall goal of this study is to find out how safe and effective this cellular therapy is in treating people that have been diagnosed with synovial sarcoma, malignant peripheral nerve sheath tumors (MPNST), neuroblastoma or osteosarcoma that has grown or returned after being treated.

This study enrolls patients with newly diagnosed differentiated thyroid cancer (papillary thyroid cancer or follicular thyroid cancer) that has spread to the lungs and has a genetic mutation called and NTRK fusion. The study involves taking a study drug called larotrectinib for approximately 6 months prior to receiving radioiodine therapy (RAI). The overall goal of this study is to find out if taking the study drug before RAI works better than receiving RAI alone (the usual standard of care treatment).

This study involves taking a study drug called lurbinectedin. The overall goal of this study is to see if different doses of lurbinectedin are safe and effective at treating children and young adults with recurrent or relapsed solid tumors, including Ewing Sarcoma.

In this study we are seeking to learn how different eye tests and devices can help understand vision in patients with NF1, including how vision loss might show up on different eye imaging devices that measure the nerves or the blood vessels of the eye. The study aims to understand the mechanisms of vision loss caused by neurofibromatosis type 1 (NF1)-associated optic pathway gliomas, or NF1-OPGs. Subjects eight years and older with NF1 are eligible to enroll.
Subjects may not benefit directly from participating in this study, but the knowledge gained will help doctors understand the potential benefit of using these tests and other procedures to improve their ability to identify the best tests to monitor visual function, and ultimately identify treatments to improve vision, in children and adults with NF1.

This study enrolls patients with refractory, progressive, or relapsed solid tumors. The purpose of this study is to find out whether a drug called PEEL-224 ("the study drug") is safe and effective when given as monotherapy (a drug given by itself) and as part of a combination therapy together with other chemotherapy drugs.

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 enrolls patients with newly diagnosed differentiated thyroid cancer (papillary thyroid cancer or follicular thyroid cancer) that has spread to the lungs and has a genetic mutation called RET fusion. The study involves taking a study drug called selpercatinib for approximately 6 months prior to receiving radioiodine therapy (RAI). The overall goal of this study is to find out if taking the study drug before RAI works better than receiving RAI alone (the usual standard of care treatment).

This study has two parts. Part A enrolls patients who have been diagnosed with a solid tumor, lymphoma or desmoid tumor that has either come back ("relapsed") or does not respond to therapy ("is refractory"). Part B enrolls patients with a relapsed or refractory Ewing sarcoma, desmoid tumor, osteosarcoma, liver tumor, Wilms tumor, or tumors with changes in a gene family known as the Wnt pathway. This study involves taking a study drug called tegavivint. The study will be testing different doses of the study drug to find the safest dose.