The study will enroll male and female patients between 2 years to 25 years of age with sickle cell disease or beta thalassemia major, who do not have a matched related donor, and their doctor feels their condition should be treated with hematopoietic stem cell transplantation (HSCT).        

·       -For Sickle Cell Disease, individuals must have significant disease. We would consider you for transplant if you have had severe neurologic symptoms including stroke, frequent vaso-occlusive events (examples include pain episodes requiring pain management or acute chest syndrome), or administration of regular and frequent red blood cell transfusions to prevent disease complications. 

·       -For Beta-Thalassemia Major, you would be considered a candidate for transplant if you have genetic test confirming Beta Thalassemia and have required 8 or more transfusions per year in the past two years.

The purpose of this research study is to test a new method of cell processing for stem cell transplants with a closely matched unrelated donor. The new method is called alpha/beta T cell and B cell depletion using a device called CliniMACs. We want to see if this method can reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to establish a new immune system. The use of the CliniMACs device for alpha/beta T and B cell depletion is experimental and is considered research.

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa). The FDA has not approved this drug for the treatment of GSDIa but has approved us to use this drug as part of this clinical trial. The purpose of this research study is to see if DTX401 can help people with GSDIa have normal blood sugar levels and fewer episodes of hypoglycemia. DTX041 works by delivering copies of the G6PC gene to your liver through a one-time infusion. Participation in this study will last for 2 years and will involve 30 study visits. It is possible that treatment with the study drug infusion may improve your blood sugar levels. Travel costs will be covered by the study and you will receive compensation for participating.

The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as UCART22 (“study drug”). UCART22 cells are special types of cells called T-cells that have been collected from a donor and then are genetically modified (changed). T-cells are related to your immune system, which helps to fight infection. These T-cells will be changed in a way that will allow the cells to identify and kill your tumor cells.

Patients with B-cell Acute Lymphoblastic Leukemia (B-ALL) whose disease has come back or have not responded to treatment may be eligible for this study.

This study involves taking a study drug called lurbinectedin. The overall goal of this study is to see if different doses of lurbinectedin are safe and effective at treating children and young adults with recurrent or relapsed solid tumors, including Ewing Sarcoma.

The purpose of this study is to evaluate social interactions in adolescents and whether changes in information can influence social behaviors. This study will focus on behavioral assessments of social processing over time in adolescents. The information collected in this study is essential for developing clinical treatments focused on addressing social processes in adolescents with restrictive eating disorders and to improve our understanding of social processing in adolescents with restrictive eating disorders, informing both future research and clinical care.

The purpose of the study is to learn more about the experience of caregivers who have a child diagnosed with autism. We also want to better understand what is working well in healthcare in the US. This study is open to primary caregivers of a child (aged 3-17) who has been diagnosed with autism spectrum disorder in the last year. Participants must have immigrated to the US and identify as Hispanic or Latinx. Study staff will interview participants about their experiences as a caregiver of an autistic child. This one-time interview will last 60-90 minutes in Spanish or English.

This study enrolls patients between 5 and 40 years of age with a diagnosis of Ewing sarcoma (including Ewing-like sarcoma) or osteosarcoma that has progressed on or relapsed after upfront initial therapy. The purpose of this study is to find the best dose of a drug called cabozantinib (“the study drug”) to give to patients in combination with a routine chemotherapy drug called ifosfamide.