Philadelphia Campus

SAR443579 in AML, B-ALL, or MDS

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).

GD2CART for Osteosarcoma or Neuroblastoma

This study enrolls patients that have been diagnosed with osteosarcoma or neuroblastoma that has come back (relapsed) or has not responded to standard treatments (refractory).

Neurophysiological Biomarkers in Rett Syndrome

The purpose of this study to learn more about the brain function of females with Rett Syndrome and how it compares to the brain function of typically developing females.

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.

Uproleselan in Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or Mixed Phenotype Acute Leukemia (MPAL)

This study enrolls patients that have been diagnosed with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or mixed phenotype acute leukemia (MPAL) that has either come back (‘relapsed’) or does not respond to therapy (‘refractory’).

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").

Tiragolumab and Atezolizumab in SMARCB1 or SMARCA4 Deficient Tumors

This study enrolls patients that have been diagnosed with SMARCB1 or SMARCB4 deficient cancer that has either come back (“relapsed”) or does not respond to therapy (“is refractory”) or have been newly diagnosed with a SMARCB1 or SMARCB4 deficient cancer that has no known standard treatment.