Center for Precision Medicine for High-Risk Pediatric Cancer Clinical Studies

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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.

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.

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.

Synovial Sarcoma Registry / Biospecimen Repository

This study enrolls patients of any age with a diagnosis of synovial sarcoma. The purpose of this study is to establish a registry and biorepository for synovial sarcoma patients by collecting and storing data and biospecimens, such as blood, saliva and/or buccal (cheek) swabs, and tumor tissue. 
If you or your child are interested in participating in this study, please click on this REDCap Survey to complete a study interest form. A study investigator will contact you to discuss the study in more detail. 

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. 


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