Skip to main content

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

Lutathera in Somatostatin Neuroendocrine Tumors

This study involves taking a study drug called Lutathera.The overall goal of this study is to see if Lutathera is safe in adolescentsubjects with either a gastroenteropancreatic neuroendocrine tumor (GEP-NET),or a pheochromocytoma and/or paraganglioma (PPGL). This study will also look atthe levels of radioactivity after Lutathera administration.

Phase: Phase II

Actively recruiting: Yes

Category: Children

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

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

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: Yes

Category: Adults, Children

Phase 1 Study Of Lentivirally Transduced T Cells Engineered To Contain Anti-CD123 In Subjects With Refractory Or Relapsed Acute Myeloid Leukemia

Phase 1 open-label study to estimate the safety, manufacturing feasibility, and efficacy of intravenously administered, lentivirally transduced T cells expressing anti-CD123 in pediatric subjects with relapsed/refractory Acute Myeloid Leukemia (AML). Main study procedures include medical record review, apheresis, CART123 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 I

Actively recruiting: Yes

Category: Adults, Children

PTEN Hamartoma Tumor Syndrome Phenotyping

Children and adults with PTEN Hamartoma Tumor Syndrome (PHTS) are at risk for specific long-term medical complications including autism spectrum disorder and cancer. While protocols for cancer screening exist, we do not yet have protocols for screening other complications of this syndrome, including neurobehavioral differences and immunologic changes. This pilot study will assess these outcomes in children and young adults with PHTS ages 1 to 20 years.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

PTX-022 in the Treatment of Microcystic Lymphatic Malformations

The purpose of this study is to see if a new investigational topical (applied to the skin) formulation of sirolimus, PTX-022 (the study drug), is safe and effective in treating participants with microcystic Lymphatic Malformations.

Phase: Phase III

Actively recruiting: Yes

Category: Adults, Children

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

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: Yes

Category: Adults, Children

Jump back to top