This study is for patients with recurrent/progressive medulloblastoma, which is a type of childhood brain tumor. Participants in this study will receive intravenous (IV, into the veins) bevacizumab and intrathecal (into the spinal fluid) or intraventricular (into the fluid surrounding the brain) etoposide and cytarabine in combination with five oral (taken by mouth) chemotherapy drugs as a possible treatment for recurrent/progressive medulloblastoma. Total study duration is about 1 year and depending on how well a participant tolerates the medications and the response of the disease, the patient may continue the treatment after the first year.
Babies that are born extremely prematurely are at higher risk of developing chronic (long term) lung disease (CLD) and other complications (problems). The purpose of this study is to test the safety and effectiveness of an investigational drug called mecasermin rinfabate (rhIGF-1/rhIGTBP-3) or OHB-607. The researchers want to find out if OHB-607 can help reduce the risk of chronic lung disease in babies born prematurely and if it can help reduce the risk of other complications.
This study is for patients that have been diagnosed metastatic germ cell tumors. This study will compare the standard chemotherapy regimen with an accelerated chemotherapy regimen using the same drugs to see if the accelerated chemotherapy regimen is beneficial but not more toxic than the standard chemotherapy regimen. The accelerated chemotherapy is experimental. Participants can expect to be on study for about 3 months and continue to be followed for up to 5 years.
This study is for patients that have been diagnosed with Epstein-Barr Virus associated Post-Transplant Lymphoproliferative Disorder (EBV-PTLD). The investigational drug in this study is tabelecleucel. Tabelecleucel is a product containing special immune allogeneic cells, called EBV-Cytotoxic T Lymphocytes (EBV-CTLs), that are made in the laboratory starting with cells from a healthy person who is immune to EBV. The purpose of this study is to test how well tabelecleucel works to treat EBV-PTLD following SOT in patients who have not responded to rituximab or rituximab plus chemotherapy treatment and to see what side effects happen. Your participation will help us to understand more about tabelecleucel. In the first 12-month period, participants will be asked to come to the clinic for an estimated 11 to 19 study visits and a minimum of 2 scans, depending on the number of cycles of treatment they are given. After treatment is done, participants will enter into the follow-up phase, and will continue to come into the clinic for scheduled check-ups for up to 24 months after the first dose of tabelecleucel was given. Participant information on the status of their disease and any new treatment will be collected for up to an additional 3 years. Participants can expect to be in this study for up to five years total.
This study is for patients that have been diagnosed with Leukemia or Lymphoma. and have been treated on one of the following studies: Pediatric Oncology Group (POG) 9404, 9425, 9426, or Dana Farber Cancer Institute (DFCI) ALL Consortium 95-01. The purpose of this study is to look at the heart function of patients selected by change to get DRZ compared to the heart function of patients selected by chance not to get DRZ. In this study, they will also look at gender, age at cancer diagnosis, current age, dose of chemotherapy, and if chest radiation influences the risk of early heard damage. Participants in this clinical trial are expected to come to one clinic visit.
This study is for patients that have been diagnosed with Germ Cell Tumors. The purpose of this study is to evaluate whether a strategy of complete surgical resection followed by surveillance can maintain an overall survival rate of at least 95.7% at two years for pediatric, adolescent and adult patients (ages 0- 50 years) with Stage I (low risk) malignant germ cell tumors, and at least 98% for patients with ovarian pure immature teratoma. The drugs used in this study are carboplatin and cisplatin. Participants will be followed for up to 10 years.
This study is for patients that have been diagnosed with High Risk B-Lymphoblastic Leukemia (HR B-ALL). The investigational drug in this study is Ruxolitinib. The purpose of this study is to find out if the study drug, ruxolitinib, in combination with standard HR B-ALL treatment is safe and effective in children, adolescents, and young adults with HR B-ALL. Participants can expect to be in this study for the treatment period of approximately 26 months (females) or 38 months (males) plus the post-treatment follow-up. Subjects are considered on study during the post-treatment follow-up period until the subject is deceased, lost to follow-up, or until the study is completed. Subjects in this study will be followed until all enrolled subjects have been followed for 3 years from Day 1 or are deceased or lost to follow-up.
A device called the "Liposorber LA-15 System" has been approved by the United States Food and Drug Administration for treating kids with focal segmental
glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used, but the kidneys are still working okay. It can also be used if the subject has had a kidney transplant and the FSGS comes back after the transplant. Although the Liposorber System can be used for FSGS, we are not sure how well the Liposorber System works. So, we are doing this study to find out how well the treatment works.
In this research study, there will be up to 5 children who have FSGS enrolled at MUSC. Subjects will come back for up to 12 treatments over 9 weeks and then 5 visits to their study doctor over the next 2 years.
The purpose of this study is to create and maintain a registry, which is a database (a searchable collection of information) about children, adolescents and young adults with pediatric onset of rheumatic diseases. This data may help in the evaluation of the safety and benefit of medications that are prescribed to patients who have rheumatic diseases.
The overall goal of this study is to find out what effects, good and/or bad, a low
dose and a high dose of lenalidomide have on children, adolescents and young
adults with recurrent (has come back after being treated), refractory (has not gone
away with previous treatment), or progressive (is not responding to previous
treatments) Juvenile Pilocytic Astrocytomas (JPA) and Optic Pathway Gliomas
(OPG).