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A Study to Evaluate the Safety and Efficacy 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 research study is to understand the impact of transplant on the quality of life of the entire family including sibling donors, other siblings and parents.
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.
AALL1521; Ruxolitinib and Chemotherapy for Patients with JAK-Mutant B-ALL
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.
AAML1921; Bosutinib for Chronic Myeloid Leukemia
The overall goals of this study are to find the best dose of bosutinib that we can give safely, and to find out what effects, good and/or bad, bosutinib has on children and adolescents with Chronic Myeloid Leukemia (CML).
Abemaciclib For Solid Tumors
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).
Alpelisib in PIK3CA-related overgrowth syndrome
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.
ALRN-6924 in Pediatric Cancer
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.
ANBL1232 Response & Biology Risk Factors in Patients with Non-High-Risk Neuroblastoma
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.