This study is for children age 0-2 who have been diagnosed with severe combined immunodeficiency (SCID). In this study, participants will be randomized (select by chance) into groups that will decide the dose of chemotherapy they will receive, receive chemotherapy prior to a blood stem cell transplant, and have blood drawn for research tests. The purpose of this research study is to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help patients with SCID, and to see if the device the CliniMACS® is effective in preparing donor stem cells before the transplant. Participants can expect to be in this study for up to 3 years.
This study will evaluate the biomarkers of children, younger than 12 years old, with moderate to severe eczema, also known as atopic dermatitis, whose disease is not adequately controlled with topical therapies (creams or lotions) or when those therapies are not medically advisable.
This is not a treatment study. This study is only recruiting volunteers who are enrolled into an atopic dermatitis registry known as Pedistad. Participation in this study will involve 6 visits every 12 months over a period of 5 years.The information collected in this study may lead to an improved understanding of atopic dermatitis and may provide healthcare providers with important information for treating atopic dermatitis in the future.
The goal of the study is to characterize the features of Infantile Hemangiomas before and after treatment. Certain characteristics of the hemangioma can be seen more clearly with a closer and more resolute image of the lesion (abnormal vessels etc.). Developing a greater understanding of these characteristics? may help clinicians better predict the course of infantile hemangiomas in children.
Specific aim 1: to correlate images seen on dermoscopy with regression of the hemangioma.
Specific aim 2: to provide features that may help to predict a better response to treatment.
Pediatric traumatic injury (PTI) ? defined as unintentional injury requiring hospitalization and, often, extended periods of physical and emotional recovery ? is experienced by 300,000 children in the U.S. annually. Roughly 20-40% of children and caregivers develop posttraumatic stress disorder (PTSD) and/or depression following PTI, yet most U.S. trauma centers fail to provide even basic mental health screening post-injury. It is critical to advance our knowledge of available mental health services in trauma centers for this frequently overlooked population to accelerate their physical and emotional recovery. In this project, trauma center providers across the U.S. will complete a survey and a qualitative interview to assess their current protocols and resources available to screen and treat children and families' mental health in the aftermath of PTI, as well as their opinions regarding feasibility of implementing protocols to better address the emotional health recovery within this population.
The purpose of this study is to evaluate how effective MEDI8897 is at preventing lung disease caused by Respiratory Syncytial Virus (RSV) and to evaluate the safety and tolerability of MEDI8897 in healthy infants compared with placebo. A placebo is a saline solution that looks like the study drug but it does not contain the active ingredient.
This study is for patients that have had pediatric hematopoietic stem cell transplants and/or have been donors. The purpose of this study is to better understand the experiences of children and adolescents who donate hematopoietic stem cells. This is a quality of life survey study consisting of telephone interviews. Each interview should take approximately 15 minutes to complete and will be conducted 4 times over the course of 1 year after transplant.
MEDLEY is a clinical study researching an investigational medication called MEDI8897, for babies at high risk for respiratory syncytial virus (RSV) disease. The study is looking to see how safe MEDI8897 is, and if it works as well as a medication, Synagis, already approved to protect against RSV.
Babies may be eligible for the study if they are in their first year of life and:
? were born prematurely (at 35 weeks or earlier) or have a heart or lung condition
? have not received any RSV preventive medication. Subjects will receive 5 monthly intramuscular injections throughout RSV season (October to March)
This study is for pediatric patients that have been diagnosed with advanced solid tumors and hematological malignancies that have stopped responding to standard therapy. The investigational drugs in this study are durvalumab and tremelimumab. The purpose of this research study to find out if the study drugs called durvalumab and tremelimumab will work and be safe for the treatment of advanced cancer in children. Participants can expect to be on this study for up to 6 years.
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 who have been diagnosed with osteosarcoma and were once treated with a chemotherapy drug called cisplatin with or without ifosafamide. The purpose of this study is to look at potential effects on fertility in male osteosarcoma survivors treated with cisplatin with or without ifosfamide compared to male controls without a history of cancer. Participants can expect to be on this study for up 3 months.