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.
This is a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.
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).
the United States, it is standard treatment for patients with high-risk neuroblastoma (NBL) to receive the drugs carboplatin, etoposide and melphalan (CEM) as the preparative regimen in Consolidation therapy prior to Autologous Stem Cell Transplant (ASCT). BuMel Consolidation therapy has recently been studied in patients with high-risk NBL in some European countries. The findings from those studies indicate that the use of BuMel prior to ASCT may be linked to an increase in the survival rate for patients when compared to CEM. Those studies also indicate that the chance of the disease coming back (a relapse) may be lower among the patients who received BuMel Consolidation therapy. In North America the BuMel combination is considered experimental. In this study, researchers want to find out if a combination of busulfan and melphalan (BuMel) can be given as Consolidation therapy prior to ASCT for subjects with newly diagnosed high-risk NBL. The main goal of this study is to find out what effects, good and/or bad, a BuMel preparative regimen given before ASCT has on people with newly diagnosed high-risk NBL.
Ependymoma is a type of rare childhood cancer that occurs in the brain and spinal cord. Survival statistics are generally disappointing with a 5-year survival of 50-64%. The standard of care for ependymoma is maximal surgical resection followed by radiation therapy directed at the primary site of disease.
Radiation therapy is associated with immediate and long-term toxicities in children, especially young children. For this reason, it has been the practice of some doctors not to give radiation therapy to children with ependymoma when the tumor has been completely surgically removed. The investigators who designed this study have created strict measures to choose those who will not receive additional treatment after surgery and careful follow-up to minimize the risks to those who are assigned to observation only.
To collect information about the patient's leukemia and to seek the optimal treatment for children with ALL based on the individual level of risk of the cancer coming back after treatment. The risk groups are defined as a result of recent research conducted by the Children's Oncology Group (COG). We would like to learn if the use of an experimental intrathecal therapy (ITT), which has been given to many people with ALL and has been well tolerated, would be better at preventing relapse in the central nervous system and improve disease outcomes in children with High Risk ALL.
The primary purposes of this study are to:
•Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study.
•Assess how well and how quickly blood counts return to normal after transplant in recipients on this study.