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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

Specialty

Clinical trial phase

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

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)

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

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)

Synovial Sarcoma Registry/Biospecimen Repository

The Synovial Sarcoma Registry and Biospecimen Repository was opened in June of 2023 with the goal of furthering research and improving outcomes for patients with synovial sarcoma. This study is open to anyone treated in the United States. You do NOT have to travel to Philadelphia to participate.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, Remote (Online / Phone Call Only) Studies

Conditions: Soft tissue sarcomas, Synovial sarcoma

Tegavivint for the Treatment of Solid Tumors, Lymphomas & Desmoids

This study has two parts. Part A enrolls patients who have been diagnosed with a solid tumor, lymphoma or desmoid tumor that has either come back ("relapsed") or does not respond to therapy ("is refractory"). Part B enrolls patients with a relapsed or refractory Ewing sarcoma, desmoid tumor, osteosarcoma, liver tumor, Wilms tumor, or tumors with changes in a gene family known as the Wnt pathway. This study involves taking a study drug called tegavivint. The study will be testing different doses of the study drug to find the safest dose.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Desmoid tumors , Ewing sarcoma, Hepatoblastoma (liver cancer), Non-Hodgkin Lymphoma in Children, Osteosarcoma (bone cancer in children), Wilms Tumor (Kidney Tumor)

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