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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

Eligible age

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 in Solid or CNS tumors with RET-Alterations

The purpose of this study is to test the safety and efficacy of the investigational drug called Selpercatinib in children whose cancers have a specific genetic change (RET-fusion or RET-mutation). Selpercatinib has been FDA-approved for adults and pediatric patients 12 years of age or older for certain types of lung and thyroid cancers. Selpercatinib is administered in capsule form, liquid suspension, or may also be administered by a feeding tube for subjects who are unable to swallow.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Selpercatinib in Thyroid Cancer (Collaboration with the International Thyroid Oncology Group)

This study involves taking a study drug called selpercatinib. The overall goal of this study is to find out how effective the study drug is in combination with radioactive iodine (I-131) in treating people with thyroid cancer. The study drug will be given in combination with treatments you will receive as part of your regular cancer care (I-131 and thyrotropin alfa).

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Thyroid Cancer in Children

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.

Actively recruiting: Yes

Category: Adults, Children

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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