Cancer Center Clinical Studies

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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. 

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. 

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. 

AALL2121: SNDX-5613 for Relapsed or Refractory Leukemia

This study enrolls patients that have been diagnosed with acute leukemia associated with a KMT2A (MLL) gene rearrangement (referred to as KMT2Arearranged, or KMT2A-R). The KMT2A-R leukemia has come back after treatment (relapsed) or is not responding to treatment (it is refractory). This study involves taking a study drug called SNDX-5613. The study is divided into two phases: one in which the study drug is given in combination with chemotherapy and a phase where the study drug is given alone. The purpose of the study is to test the safety of the study drug with chemotherapy, and how well the treatment regimen works for infants and young children with relapsed or refractory KMT2A-R leukemia.

Larotrectinib RAI Thyroid

This study enrolls patients with newly diagnosed differentiated thyroid cancer (papillary thyroid cancer or follicular thyroid cancer) that has spread to the lungs and has a genetic mutation called and NTRK fusion. The study involves taking a study drug called larotrectinib for approximately 6 months prior to receiving radioiodine therapy (RAI). The overall goal of this study is to find out if taking the study drug before RAI works better than receiving RAI alone (the usual standard of care treatment). 

Cabo/Ifos in Ewing Sarcoma and Osteosarcoma

This study enrolls patients between 5 and 40 years of age with a diagnosis of Ewing sarcoma (including Ewing-like sarcoma) or osteosarcoma that has progressed on or relapsed after upfront initial therapy. The purpose of this study is to find the best dose of a drug called cabozantinib (“the study drug”) to give to patients in combination with a routine chemotherapy drug called ifosfamide. 

TAPISTRY

The purpose of this study is to evaluate the effects, good or bad, of targeted therapies or immunotherapy (drugs that help the body's immune system fight cancer cells) in patients who have solid tumors with specific genetic alterations or with a high number of mutations. Patients will be assigned to cohorts (groups) based on having a specific type of genetic alteration in their tumor. The study includes cohorts for patients whose tumors have genetic alterations in the ROS1, NTRK, PIK3CA, BRAF, or RET genes or a high number of mutations in their tumor.
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