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

22CT012: GPC2 CAR T Cell Trial

The purpose of this study is to test an experimental approach called cell therapy for neuroblastoma or retinoblastoma. This study will take a patient's own white blood cells (T cells) and change them to turn against the cancer. These cells are an experimental treatment and have not been approved by the FDA. CAR T cells have been used to treat other cancers, but GPC2 CAR T cells have not been administered to human patients prior to this clinical trial.

If they choose to participate in this study, active participation will last approximately 12 months. Participants will also be asked to sign consent to a separate long term follow-up protocol to monitor their health for up to 15 years after their last GPC2 CAR T treatment.

This study will assess the safety and effectiveness of GPC2 CAR T cells. Overall, approximately 45 subjects will be enrolled on this study.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Neuroblastoma, Retinoblastoma (Eye Cancer in Children)

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

CD33 CART AML

This phase 1/2 study will determine the safety, feasibility, and efficacy of CD33 CART cells following lymphodepleting chemotherapy in patients with acute myeloid leukemia (AML). Patients who are between ages 1 to 30 years with relapsed or refractory AML will be eligible for this study.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML)

Endotheliopathy in Oncology

The purpose of this research study is to determine if certain markers in your blood are increased over the duration of your chemotherapy, especially as you get sicker, such as developing a severe infection (sepsis) or severe respiratory failure (acute respiratory distress syndrome).

Phase: Pilot

Actively recruiting: No

Category: Adults, Children

Conditions: Pediatric Leukemias, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Myelogenous Leukemia (CML), Juvenile Myelomonocytic Leukemia (JMML), Non-Hodgkin Lymphoma in Children, Hodgkin Lymphoma

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)

Larotrectinib RAI Thyroid (LANTERN)

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).

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children, MRI/Imaging Studies

Conditions: Thyroid Cancer in Children, Differentiated Thyroid Cancer

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

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

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)

Jump back to top