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Healthy control children are needed to help participate in a study on Immunometabolic Dysregulation.
Glomerular disease is a major cause of chronic kidney disease associated with substantial morbidity; children and adults with glomerular disease face many threats to bone health.
16BT052; Unrelated And Partially Matched Related Donor PSCT w/ TCR αβ depletion for patients with BMF
The purpose of this research study is to test a new method of cell processing for stem cell transplants with an unrelated donor or partially matched related donor for patients with BMF who do not have a well matched related donor. The new method is called alpha/beta T cell and B cell depletion using a device called CliniMACs.¬† We want to see if this method can reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to establish a new immune system.
The overall goals of this study are to evaluate the side effects and benefits of CUDC-907 in patients with relapsed or treatment refractory solid tumor, brain tumor, or lymphoma, and to determine the best dose of CUDC-907 for children and adolescents.
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.
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).
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).
A Study to Evaluate the Safety andEfficacy of a Single Dose of CTX001 in Subjects With Severe Sickle Cell Disease
The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease.
The purpose of this study is to compare the genes of African American children and adults who have inflammatory bowel disease (IBD) with the genes of those who do not have IBD.
This study is testing the safety and effectiveness of adding a targeted therapy (ruxolitinib) to standard chemotherapy for children, adolescents, and young adults with high-risk acute lymphoblastic leukemia with CRLF2 and other JAK pathway mutations.