The Overcoming Cohort study is a studies looking for variations in DNA,for populations less than or 25 years of age, that either protect people from COVID-19 disease or make them more susceptible. The study will sequence patients' entire genome or the smaller group of genes that code for proteins (exome). The study would gain a better understanding of COVID-19 that would improve diagnostic, prevention, and treatment measures.
This is an observational study, which means that only information is collected on treatment, examinations or diagnostic tests which are a part of the routine standard of care. The participant is being asked to volunteer because they have been diagnosed with locally advanced or metastatic TRK fusion cancer treated with larotrectinib. The purpose of this study is to learn more about the safety profile and the effectiveness of VITRAKVI® under routine practice conditions.
Participants will be followed for a minimum of 5 years from the time they agree to participate in the study.
This study is for patients that have been newly diagnosed with BRAF v600-Mutant High-Grade Glioma (HGG). The overall goal of this study is to see if using two drugs called dabrafenib and trametinib after radiation treatment will be better than treatments used in the past in helping to get rid of or shrink HGG. The treatment involves cancer fighting medicines plus radiation. The treatment on this study takes a little over 2 years. It is divided into 2 phases of therapy.
People who recover from COVID-19 do so, at least in part, because their blood contains substances called antibodies, which are capable of fighting the virus that causes the illness. It turns out that for some other diseases caused by respiratory viruses, giving people the liquid portion of blood, called plasma, obtained from those who have recovered from the virus, leads to more rapid improvement of the disease. We think that patients with COVID-19 may improve faster if they receive plasma from those who have recovered from COVID-19, because it may have the ability to fight the virus that causes COVID-19.
We are performing this study to see if children admitted to the hospital with COVID-19 may have an improved chance of recovery by receiving plasma from someone who has recovered from COVID-19.
Remestemcel-L will be evaluated in children or adolescents with multisystem inflammatory syndrome (MIS-C) associated with coronavirus disease (COVID-19) who have baseline myocardial complications. The primary objective of this study is to assess the safety and tolerability of remestemcel-L in Multisystem Inflammatory Syndrome in Children (MIS-C) associated with coronavirus disease (COVID-19).
This study is for patients that have been diagnosed with newly diagnosed High-Grade Glioma (HGG). The investigational drug in this study is veliparib. The purpose of this study is to see if veliparib given with radiation therapy followed by veliparib with temozolomide given as maintenance therapy works better than treatments that have been tried in the past. Participants can expect to be in this study for about 1 year and be followed for up to 10 years.
This study will implement and evaluate the Enhanced Recovery in Children Pathway (ENRICH-P) for pediatric patients with inflammatory bowel disease. Participants (or participants' parent) will be asked to complete quality of life surveys pre-operatively and up to 6 weeks after surgery.
To evaluate the safety and clinical benefit of NexoBrid in hospitalized children (0-18 years) with thermal burns of 1-30% total body surface area as compared to standard of care. Patients enrolled in this trial will undergo daily assessments in hospital after their treatment until they are discharged. They will then have weekly follow-ups until completed wound closure. Once this is achieved, there will be a 6 week, 12 week, 6 month, 12 month, 18 month, and 24 month follow up visit.
The main purpose of this study is to see if taking dupilumab is effective in treating Eosinophilic Esophogitis (EoE). The study will determine if dupilumab is an effective treatment compared with placebo in adult and adolescent patients. Dupilumab is administered through a subcutaneous injection (shot). Dupilumab is is an investigational drug, which means that it is not yet approved to treat EoE. Participation in the study will last up to 48 weeks.
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.