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: Retinoblastoma (Eye Cancer in Children), Relapsed or Refractory Neuroblastoma

ALRN-6924 in Pediatric Cancer

This study involves taking a study drug called ALRN-6924. ALRN-6924 is an investigational cancer treatment that activates p53, which suppresses the growth of tumors by blocking proteins in the cell called MDM2 and MDMX that inhibit p53 from doing its job. The overall goals of this study are to evaluate the side effects and benefits of ALRN-6924 in patients with relapsed or treatment refractory solid tumor or brain tumor, and to determine the best dose of ALRN-6924 for children and adolescents. This study will also look at how the body breaks down ALRN-6924 by measuring the amount of ALRN-6924 in the blood over time after a dose.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Pediatric Brain Tumors

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)

CEST MRI Study for Children with Brain Tumors

This study aims to explore a new type of magnetic resonance imaging (MRI) called Chemical Exchange Saturation Transfer (CEST) in children aged 6-18 years with brain tumors. An MRI is a machine that takes detailed pictures of the brain. The CEST MRI used in this study is still being tested and has not been approved by the FDA yet. The study wants to find out if this new type of MRI can help doctors evaluate brain tumors. CEST MRI creates images using water molecules and protons in the brain. Early research shows that CEST could be helpful in understanding brain tumor changes.

This study will enroll 5 participants.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, MRI/Imaging Studies, One-Time Visit Studies

Conditions: Pediatric Brain Tumors

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)

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

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

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

Jump back to top