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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

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)

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

INBRX-109 in Ewing Sarcoma and Gastrointestinal Stromal Tumor (GIST)

This study involves taking a study drug called INBRX-109. The overall goal of the study is to find out how safe and effective the study drug is in treating people that have been diagnosed with advanced or metastatic Ewing sarcoma or SDH-deficient solid tumor/Gastrointestinal Stromal Tumor (GIST).

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Ewing sarcoma, Central Nervous System (CNS) Germ Cell Tumors

Lurbinectedin in Ewing Sarcoma

This study involves taking a study drug called lurbinectedin. The overall goal of this study is to see if different doses of lurbinectedin are safe and effective at treating children and young adults with recurrent or relapsed solid tumors, including Ewing Sarcoma.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors, Ewing sarcoma

NEPENTHE - Next Generation Personalized Neuroblastoma Therapy

This is a clinical trial for patients with neuroblastoma that has returned (relapsed) or has not responded to standard treatment. The patients will have a biopsy of their tumor that will be tested for genetic changes and then matched to available drugs. If the results from the testing show molecular features in the tumor that allow for study treatment, patients will be invited for further testing to see if they are eligible for new treatments. Experimental therapies are all taken orally and may consist of one drug or a combination of drugs

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children, MRI/Imaging Studies

Conditions: Neuroblastoma

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

Phase 1 Trial of CUDC-907

This study involves taking a study drug called CUDC-907. CUDC-907 is an oral drug that blocks certain proteins in tumor cells; these proteins may be important in the growth of some cancers. The overall goals of this study are to evaluate the side effects and benefits of CUDC-907 in patients with relapsed or treatment refractory solid tumor, brain tumor, or lymphoma, and to determine the best dose of CUDC-907 for children and adolescents. This study will also look at the levels of CUDC-907 in the blood over time after a dose.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors, Pediatric Brain Tumors

Repotrectinib for ALK, ROS1, or NTRK1-3 Alterations

The purpose of this study is to test the safety and efficacy of the investigational drug called repotrectinib in children whose cancers have specific genetic changes (in the ALK, ROS1, or NTRK1-3 genes). This is the first study using this drug in children under 12 years of age. Repotrectinib is administered in capsule form or liquid suspension.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors

SAR443579 in AML, B-ALL, or MDS

This study involves taking a study drug called SAR443579. The overall goal of this study is to find out if the study drug is safe and effective at treating adults and children with relapsed or refractory acute myeloid leukemia (R/R AML), high risk myelodysplastic syndrome (HR-MDS), or B-cell acute lymphoblastic leukemia (B-ALL). This is a first-in-human research study.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

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

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

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