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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

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.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors, Soft tissue sarcomas, Thyroid Cancer in Children

Oral Asciminib in CML

This study involves taking a study drug called asciminib. 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 Chronic Myeloid Leukemia (CML) and have not responded to treatment.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Children

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.

Phase: Phase II

Actively recruiting: No

Category: Adults, Children

Conditions: Neuroblastoma

PEEL-224 in Solid Tumors

This study enrolls patients with refractory, progressive, or relapsed solid tumors. The purpose of this study is to find out whether a drug called PEEL-224 ("the study drug") is safe and effective when given as monotherapy (a drug given by itself) and as part of a combination therapy together with other chemotherapy drugs.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Ewing sarcoma, Hepatoblastoma (liver cancer), Relapsed or Refractory Neuroblastoma, Osteosarcoma (bone cancer in children), Rhabdomyosarcoma, Soft tissue sarcomas, Wilms Tumor (Kidney Tumor)

Phase 2 trial of CART19 for Orphan Indications Of Pediatric B Cell Acute Lymphoblastic Leukemia (B-ALL)

This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patients with hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D). Possible procedures include medical record review, apheresis, CART19 infusion, blood draws, physical exams, imaging, and disease assessments through bone marrows and lumbar punctures. If interested in the study or if you have any questions, please contact 267-426-0762.

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Lymphoblastic Leukemia (ALL), Pediatric Leukemias

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)

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.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

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).

Phase: Phase II

Actively recruiting: No

Category: Adults, Children

Conditions: Thyroid Cancer in Children

Selpercatinib RAI Thyroid (RAISE Trial)

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 RET fusion. The study involves taking a study drug called selpercatinib 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).

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children, MRI/Imaging Studies

Conditions: Thyroid Cancer in Children, Differentiated Thyroid Cancer

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