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Department of Pediatrics Research Studies
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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.
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.
AALL1131; A Phase 3 Randomized Trial for HR B-ALL with Dasatinib Arm in Ph-like TKI Sensitive Mutations
The study compares 2 different experimental chemotherapy combinations with the usual chemotherapy combination given during post-Induction therapy.
The purpose of this registry is to study the long-term effects and safety of Abatacept(Orencia) as used for the treatment of Juvenile Idiopathic Arthritis(JIA). JIA patients that are currently taking Abatacept may be eligible to participate in this study.
The purpose of this study is to determine the highest safe dose of abemaciclib in patients with relapsed or treatment refractory solid tumors given in combination with the chemotherapy drugs, irinotecan and temozolomide (Part A) or in combination with just temozolomide (Part B).
The Alterra study is for patients in need of pulmonary valve replacement.
In this study we are using new biologic features, in addition to the standard risk factors, to help decide which subjects need treatment and what the best treatment is.
A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.
The goal of the study extension is to see whether the incretin hormone GIP increases insulin secretion in individuals with CF and normal glucose tolerance compared to individuals without CF and normal glucose tolerance.