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A prospective open label, multicenter study to evaluate the feasibility and side effects of using molecularly guided therapy in combination with standard therapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.
Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days by mouth difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 twice daily on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.
Treatment of cHL in children and young adults – evaluation of pembrolizumab in a subgroup with inadequate early response to frontline chemotherapy.
The purpose of this study is to assess the safety and tolerability profile of TB-403 (humanized monoclonal antibody against placental growth factor (PlGF)) in pediatric subjects with relapsed or refractory Medulloblastoma.
This is a research study to determine the effectiveness and safety profile of VIGIL in combination with standard relapsed chemotherapy for children and young adults with first relapse of Ewing’s Sarcoma.
The purpose of this study is to test the safety of neratinib at different dose levels and to find out what effects, good and bad, it has on the patients and the cancer.
The purpose of this research is to evaluate the safety, tolerability, and pharmacokinetics of idasanutlin as a single agent and the safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin in combination with either chemotherapy or venetoclax in children and young adults with acute leukemias or solid tumors that are recurrent or refractory to standard therapy.
This study evaluates the use of carfilzomib in combination with cyclophosphamide and etoposide for children with relapsed/refractory solid tumors or leukemia.
This is a phase II, multi-center, study with eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.