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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

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

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

Neurofibromatosis 1 Optic Glioma's - Understanding the mechanisms of Vision Loss for Visual Restoration Strategies

In this study we are seeking to learn how different eye tests and devices can help understand vision in patients with NF1, including how vision loss might show up on different eye imaging devices that measure the nerves or the blood vessels of the eye. The study aims to understand the mechanisms of vision loss caused by neurofibromatosis type 1 (NF1)-associated optic pathway gliomas, or NF1-OPGs. Subjects eight years and older with NF1 are eligible to enroll.
Subjects may not benefit directly from participating in this study, but the knowledge gained will help doctors understand the potential benefit of using these tests and other procedures to improve their ability to identify the best tests to monitor visual function, and ultimately identify treatments to improve vision, in children and adults with NF1.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Conditions: Neurofibromatosis type 1

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)

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

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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