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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

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

AUTO1 for B-Lymphoblastic Leukemia and B-cell Non-Hodgkin Lymphoma

The purpose of this study is to determine whether AUTO1, an experimental form of therapy, is safe and effective in helping the immune system fight cancer. AUTO1 uses immune cells, called T cells, that are collected from the blood using a process called leukapheresis and then modified in a lab to recognize and target cancer cells. These modified cells are then put back into the body through infusion.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Children

Conditions: Acute Lymphoblastic Leukemia (ALL), Non-Hodgkin Lymphoma in Children

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)

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)

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

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)

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: No

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML)

Phase 2 trial of CART19 for Orphan Indications Of Pediatric B Cell Acute Lymphoblastic Leukemia (B-ALL)

This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patients with hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D). Possible procedures include medical record review, apheresis, CART19 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 II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Lymphoblastic Leukemia (ALL), Pediatric Leukemias

PHOX2B CAR T-Cells for Relapsed Neuroblastoma

The purpose of this study is to test an experimental approach called cell therapy for neuroblastoma. This study will take a patient's own white blood cells (T cells) and change them to turn against the cancer. CART cells have been used to treat other cancers, but PHOX2B CART cells have not been administered to human patients prior to this clinical trial. Participants will be given PHOX2B cells via an IV and then monitored for up to 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 PHOX2B CART treatment.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Relapsed or Refractory Neuroblastoma

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