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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

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)

22CT015: CART 19-22 co-administration

CAR T cell therapy has shifted the treatment landscape for pediatric and young adult patients with advanced B-cell leukemias. This study aims to treat patients with relapsed disease by administering two CART cell products, huCART19 and CART22-65s. The study will determine the safety and efficacy and usefulness of administering two CAR T cell products in patients with advanced B-cell leukemias.

This is a Phase 1/2b trial for the treatment of patients with either advanced or refractory B cell Acute Lymphoblastic Leukemia or B-lineage lymphoblastic lymphoma. Possible procedures include medical record review, apheresis, huCART19 infusion, blood draws, physical exams, imaging, and disease assessments through bone marrows and lumbar punctures. Overall, approximately 93 subjects will be enrolled on this clinical trial.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL)

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

Conditions: Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL), Pediatric Leukemias

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

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

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

BA3011 for advanced solid tumors

This study involves taking a study drug called BA3011. The overall goal of this study is to see if BA3011 is a possible treatment for patients with sarcoma.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

Beckwith-Wiedemann Syndrome (BWS) Overgrowth Registry

The primary objective of this registry/repository is to store ongoing clinical data and biospecimens to facilitate the conduct of research on disorders of growth, genetic, and epigenetic alterations. This can also include storage of samples and data from relatives of the index patients to serve as study controls.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, Healthy Controls

Conditions: Beckwith-Wiedemann syndrome/spectrum

Cabo/Ifos in relapsed/refractory sarcomas (CaIRS trial)

This study enrolls patients between 5 and 40 years of age with a diagnosis of any sarcoma 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)

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