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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

Prodigy huCART19

CAR T cell therapy has shifted the treatment landscape for pediatric and young adult patients with advanced B-cell leukemias. However, the manufacturing process remains in its first generation: difficult, time-consuming, and not automated. The time and significant resources in this process can result in patient safety issues - with patients growing sicker, with harder to control leukemias - in the waiting period. Use of the CliniMACS Prodigy platform can help to surmount these issues. This study will determine the safety and usefulness of using the CliniMACS Prodigy platform to manufacture huCART19 cells.

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 who will be treated with huCART19, which has been manufactured using the CliniMACs Prodigy Platform. 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 89 subjects will be enrolled on this clinical trial.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Lymphoblastic Leukemia (ALL)

PTEN Hamartoma Tumor Syndrome Phenotyping

Children and adults with PTEN Hamartoma Tumor Syndrome (PHTS) are at risk for specific long-term medical complications including autism spectrum disorder and cancer. While protocols for cancer screening exist, we do not yet have protocols for screening other complications of this syndrome, including neurobehavioral differences and immunologic changes. This pilot study will assess these outcomes in children and young adults with PHTS ages 1 to 20 years.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

PTX-022 in the Treatment of Microcystic Lymphatic Malformations

The purpose of this study is to see if a new investigational topical (applied to the skin) formulation of sirolimus, PTX-022 (the study drug), is safe and effective in treating participants with microcystic Lymphatic Malformations.

Phase: Phase III

Actively recruiting: Yes

Category: Adults, Children

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: Yes

Category: Adults, Children

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

Selpercatinib RAI Thyroid (RAISE Trial)

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 RET fusion. The study involves taking a study drug called selpercatinib 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

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

T2017-002 TACL Ixazomib Study

CHOP and the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium is conducting a study for children and young adults 21 years old or younger who have relapsed or refractory acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma. This study adds the study drug 'Ixazomib' to standard relapsed ALL chemotherapy drugs. For more information about this study, including specific eligibility criteria, please visit clinicaltrials.gov and search for "NCT03817320" or visit the TACL website at https://tacl.chla.usc.edu/Tacl/#!/Page/3. To hear more about this and other available trails or get any questions answered please contact our Cancer Intake Specialist by phone at 267-426-0762 or email Oncointake@email.chop.edu.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Tara-002 in Lymphatic Malformations

This study is for people with growing abnormal lymph vessels called lymphatic malformations (LMs). This study has 3 parts and involves taking a study drug called TARA-002. The purpose of this study is to find out if the study drug is safe and effective (can help) people with LMs. Participants receive the study drug by injection and will complete other research assessments throughout the study.

Phase: Phase II

Actively recruiting: Yes

Category: Children

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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