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

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AALL2121: SNDX-5613 for Relapsed or Refractory Leukemia

This study enrolls patients that have been diagnosed with acute leukemia associated with a KMT2A (MLL) gene rearrangement (referred to as KMT2Arearranged, or KMT2A-R). The KMT2A-R leukemia has come back after treatment (relapsed) or is not responding to treatment (it is refractory). This study involves taking a study drug called SNDX-5613. The study is divided into two phases: one in which the study drug is given in combination with chemotherapy and a phase where the study drug is given alone. The purpose of the study is to test the safety of the study drug with chemotherapy, and how well the treatment regimen works for infants and young children with relapsed or refractory KMT2A-R leukemia.

Phase: Phase II

Actively recruiting: Yes

Category: 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

Alpelisib in PIK3CA Associated Lymphatic Malformations

This study has several parts and involves taking a study drug called alpelisib. Some participants enrolled into Stage 2 will receive a placebo for the first part of the study (24 weeks) and will then receive the study drug. The purpose of this study is to find out if the study drug is safe and effective (can help) people with a confirmed diagnosis of lymphatic malformation (LyM) and a documented mutation in the PIK3CA gene

Phase: Phase II, Phase III

Actively recruiting: Yes

Category: Adults, Children

Alpelisib in PIK3CA-related overgrowth syndrome

The purpose of this study is to find out if alpelisib (BYL719;the “study drug”), is safe and effective (can help) people who have PIK3CA-related overgrowth spectrum (PROS) disease.

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

BA3011 for advanced solid tumors

This study involves taking a study drug called BA3011. Theoverall goal of this study is to see if BA3011 is a possible treatment forpatients with sarcoma.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

Cabo/Ifos in Ewing Sarcoma and Osteosarcoma (CaIRS trial)

This study enrolls patients between 5 and 40 years of age with a diagnosis of Ewing sarcoma (including Ewing-like sarcoma) or osteosarcoma that has progressed on or relapsed after upfront initial therapy. The purpose of this study is to find the best dose of a drug called cabozantinib (“the study drug”) to give to patients in combination with a routine chemotherapy drug called ifosfamide.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Ewing sarcoma, Osteosarcoma (bone cancer in children)

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), Myelodysplastic Syndrome, Juvenile Myelomonocytic Leukemia (JMML)

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

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