Oncology Clinical Studies

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Palbociclib, Irinotecan, and Temozolomide in Ewing Sarcoma

The purpose of the Phase 2 portion of this study is to evaluate the safety and efficacy of the study drug called palbociclib when given in combination with two other drugs (temozolomide and irinotecan) in patients with Ewing sarcoma and when given in combination with topotecan and cyclophosphamide in patients with neuroblastoma.

Tiragolumab and Atezolizumab in SMARCB1 or SMARCA4 Deficient Tumors

This study enrolls patients that have been diagnosed with SMARCB1 or SMARCB4 deficient cancer that has either come back (“relapsed”) or does not respond to therapy (“is refractory”) or have been newly diagnosed with a SMARCB1 or SMARCB4 deficient cancer that has no known standard treatment. This study involves taking study drugs called tiragolumab and atezolizumab. These drugs are a type of immunotherapy that try to make the immune system fight cancer. The purpose of the study is to learn more about the safety of tiragolumab in children and to see how well the combination of tiragolumab and atezolizumab works when given to children and adults with these types of tumors. This study has two parts. In Part A, participants will receive tiragolumab. In Part B, participants will receive both tiragolumab and atezolizumab. 

Lutathera in Somatostatin Neuroendocrine Tumors

This study involves taking a study drug called Lutathera. The overall goal of this study is to see if Lutathera is safe in adolescent subjects with either a gastroenteropancreatic neuroendocrine tumor (GEP-NET), or a pheochromocytoma and/or paraganglioma (PPGL). This study will also look at the levels of radioactivity after Lutathera administration.

Tegavivint for the Treatment of Solid Tumors, Lymphomas & Desmoids

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. 

BA3011 for advanced solid tumors

This study involves taking a study drug called BA3011. The overall goal of this study is to see if BA3011 is a possible treatment for patients with sarcoma.

Uproleselan in Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or Mixed Phenotype Acute Leukemia (MPAL)

This study enrolls patients that have been diagnosed with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or mixed phenotype acute leukemia (MPAL) that has either come back (‘relapsed’) or does not respond to therapy (‘refractory’). This study involves taking a drug called uproleselan that is approved for adults with AML but is not yet approved for children. The purpose of the study is to find a safe dose of the study drug in children that can be given with standard chemotherapy without causing severe side effects. 

ON-TRK

The goal of this observational study is to describe the safety and effectiveness of larotrectinib in patients with locally advanced or metastatic TRK fusion cancer for whom a decision to treat with larotrectinib has been made before enrollment.
This study will enroll participants with any tumors harboring the NTRK gene fusion including Soft Tissue Sarcomas, Central Nervous System, Melanoma, and Thyroid cancers.

GD2CART for Osteosarcoma or Neuroblastoma

This study enrolls patients that have been diagnosed with osteosarcoma or neuroblastoma that has come back (relapsed) or has not responded to standard treatments (refractory). This study involves an investigational cellular therapy called GD2CART. The study will be testing different doses of the study drug to find the safest dose.

Lurbinectedin in Ewing Sarcoma

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.

SAR443579 in AML, B-ALL, or MDS

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. 
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