Department of Pediatrics Clinical Studies

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LUNA 3

The LUNA 3 study is for teenagers and adults (ages 12 and up) with primary immune thrombocytopenia (ITP) for which standard medications are no longer working. The goal of the study is to see if the study drug, Rilzabrutinib can safely increase platelet counts and improve symptoms for people with ITP. Rilzabrutinib is an investigational medication designed to stop your immune system from destroying your platelets. You may be able to join the study if you are:
  • 12 years of age or older.
  • Diagnosed with primary ITP for at least 6 months for ages 12 through 17 or for at least 3 months for ages 18 and up.
  • Have taken IVIg or steroids, but they are no longer working.
  • Have platelet counts of less than 30,000. 
Other study requirements will apply.

Healthy Controls EndoPAT

The purpose of this research study is to learn more about the normal function of blood vessels in healthy children and adolescents. This will help us discover differences between healthy individuals and children with lupus. Healthy control participants will be asked to make 1 visit to CHOP to complete a brief survey, a fasting blood draw and a test to measure the blood pressure in your fingers.

Hodgkin Lymphoma (NHL) (BIANCA)

This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah™) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL). The study is enrolling children and young adults up to age 25 with CD19-expressing mature B-cell NHL, including Burkitt leukemia/lymphoma, that has relapsed after one or more prior therapies or is primary refractory (not achieved a complete remission (CR) or partial response (PR) after the first line of therapy). This study involves the administration of the study drug, blood draws, assessment of lymphoma response, and chart reviews. After a process called leukapheresis, in which T cells (a type of white blood cell) are removed from the subject's blood and modified in order to target the lymphoma, they are returned back to the subject as the study drug. Reimbursement for travel may be provided. If you have questions or would like to learn more about the study, please contact the Cancer Immunotherapy Program at (215) 425-7193. The Principal Investigator is Dr. Shannon Maude.

NCGD

The purpose of this research study is to learn more about Graves’ disease and PTC/Hypothyroidism, How it effects brain function and brain function changes after starting medical treatment.

Endotheliopathy in Oncology

The purpose of this research study is to determine if certain markers in your blood are increased over the duration of your chemotherapy, especially as you get sicker, such as developing a severe infection (sepsis) or severe respiratory failure (acute respiratory distress syndrome).