Solid Tumor Program Clinical Studies

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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.

Selpercatinib in Thyroid Cancer (Collaboration with the International Thyroid Oncology Group)

This study involves taking a study drug called selpercatinib. The overall goal of this study is to find out how effective the study drug is in combination with radioactive iodine (I-131) in treating people with thyroid cancer. The study drug will be given in combination with treatments you will receive as part of your regular cancer care (I-131 and thyrotropin alfa). 

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). 

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. 

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. 

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.

Selpercatinib in Solid or CNS tumors with RET-Alterations

The purpose of this study is to test the safety and efficacy of the investigational drug called Selpercatinib in children whose cancers have a specific genetic change (RET-fusion or RET-mutation). Selpercatinib has been FDA-approved for adults and pediatric patients 12 years of age or older for certain types of lung and thyroid cancers. Selpercatinib is administered in capsule form, liquid suspension, or may also be administered by a feeding tube for subjects who are unable to swallow. 

Repotrectinib for ALK, ROS1, or NTRK1-3 Alterations

The purpose of this study is to test the safety and efficacy of the investigational drug called repotrectinib in children whose cancers have specific genetic changes (in the ALK, ROS1, or NTRK1-3 genes). This is the first study using this drug in children under 12 years of age. Repotrectinib is administered in capsule form or liquid suspension.  

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.


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