ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
This study is for patients that have been diagnosed with B-Lymphoblastic Leukemia (B-ALL) or Localized B-Lymphoblastic Lymphoma (B-LLy). The investigational drug used is Blinatumomab. The purpose of this study is to determine in a randomized manner if the addition of 2 cycles of blinatumomab to standard therapy improves disease-free survival (DFS) in patients with SR B-ALL and higher risk features (SR-High), and patients with standard-risk average (SR-Avg) B-ALL who are negative for minimal residual disease (MRD) by flow cytometry but have detectable or indeterminate MRD as measured by high-throughput sequencing (HTS) at end of Induction (EOI). Participants can expect to receive treatment on this study for about 2 years. After treatment, participants will be followed-up with every year for about 10 years.
The purpose of this study is to evaluate the effect a commonly used medication named clonidine has on the length of caudal nerve blocks. A caudal nerve block is an injection in the tailbone area to provide pain control after surgery.
The purpose of the study is to see if use of the RECELL device will help heal your
partial-thickness burn faster and decrease the need for skin grafting compared with a standardized wound dressing. The data collected in this current study will provide additional information about the safety and effectiveness of the RECELL® device for treatment of partial-thickness burns. The duration of each participant will last up to 12 months. Visits will include photograph documentation of the wound and various health assessments.
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 SHP607. The researchers want to find out if SHP607 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 phase III trial studies how well inotuzumab ozogamicin and post-induction chemotherapy work in treating patients with high-risk B-cell lymphoblastic lymphoma (B-ALL), mixed phenotype acute leukemia, and B-lymphoblastic lymphoma (B-LLy). Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a toxic agent called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. Drugs used in chemotherapy, such as cyclophosphamide, cytarabine, doxorubicin, daunorubicin, methotrexate, leucovorin, mercaptopurine, thioguanine, vincristine, and pegaspargase, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
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.