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 1 open-label study to estimate the safety, manufacturing feasibility, and efficacy of intravenously administered, lentivirally transduced T cells expressing anti-CD123 in pediatric subjects with relapsed/refractory Acute Myeloid Leukemia (AML). Main study procedures include medical record review, apheresis, CART123 infusion, blood draws, physical exams, imaging, and disease assessments through bone marrows and lumbar punctures. If interested in the study or if you have any questions, please contact 267-426-0762.

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.

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.

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.

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.

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.

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.

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).

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).