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Research Studies

Cancer Center Research Studies

Find research studies available to children cared for by the Cancer Center team.

Existing patients or family members
New patients, referrals and second opinions

Eligible age

Afamitresgene Autoleucel in Pediatric Subjects with MAGE-A4 Positive Tumors

This study involves an investigational cellular therapy called Afamitresgene Autoleucel. The overall goal of this study is to find out how safe and effective this cellular therapy is in treating people that have been diagnosed with synovial sarcoma, malignant peripheral nerve sheath tumors (MPNST), neuroblastoma or osteosarcoma that has grown or returned after being treated.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Neuroblastoma, Osteosarcoma (bone cancer in children), Soft tissue sarcomas

Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Relapsed AML

This study involves taking a study drug called venetoclax with or without standard of care chemotherapy. The overall goal of this study is to see if adding venetoclax to standard chemotherapy leads to improved survival for children and young adults with relapsed acute myeloid leukemia (AML). The study may last up to 9 years and will enroll male and female patients from the ages of 29 days to 21 years of age.

Phase: Phase III

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML)

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.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Neuroblastoma, Osteosarcoma (bone cancer in children)

Head Start 4 Protocol for Children with Medulloblastoma and CNS Embryronal Tumors

Children (less than 10 years of age) are eligible if they have been diagnosed with either a medulloblastoma tumor or CNS embryonal tumors of the brain or spinal cord. The standard approach to the treatment of these brain tumors in young children may include surgery, radiation therapy to the brain and spinal cord and chemotherapy. Radiation therapy is the use of high energy x-rays to kill cancer cells. Chemotherapy means treatment with cancer killing drugs. This research study will compare two different treatment regimens to see which is better for treating children with this type of brain cancer.

Phase: Phase III

Actively recruiting: Yes

Category: Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors

INBRX-109 in Ewing Sarcoma and Gastrointestinal Stromal Tumor (GIST)

This study involves taking a study drug called INBRX-109. The overall goal of the study is to find out how safe and effective the study drug is in treating people that have been diagnosed with advanced or metastatic Ewing sarcoma or SDH-deficient solid tumor/Gastrointestinal Stromal Tumor (GIST).

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Ewing sarcoma, Central Nervous System (CNS) Germ Cell Tumors

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors, Ewing sarcoma

NEPENTHE - Next Generation Personalized Neuroblastoma Therapy

This is a clinical trial for patients with neuroblastoma that has returned (relapsed) or has not responded to standard treatment. The patients will have a biopsy of their tumor that will be tested for genetic changes and then matched to available drugs. If the results from the testing show molecular features in the tumor that allow for study treatment, patients will be invited for further testing to see if they are eligible for new treatments. Experimental therapies are all taken orally and may consist of one drug or a combination of drugs

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children, MRI/Imaging Studies

Conditions: Neuroblastoma

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Pediatric Leukemias, Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL)

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors

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