In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
This study is for patients that have been diagnosed with a hepatic malignancy. The purpose of this research is to reduce therapy associated toxicity for patients with non-metastatic hepatoblastoma (HB) and hepatocellular carcinoma (HCC) without adversely affecting long term outcomes. Participants can expect to receive treatment for up to 3 months. After treatment, participants will be followed for up to 10 years.
This study is for participants that have been diagnosed with a high-risk neuroblastoma that cannot be treated or did not improve with existing therapies, or the cancer came back after treatment with existing therapies. This study involves investigational drugs called 64Cu-SARTATE and 67Cu-SARTATE. The investigational drugs will be given as an IV injection. The study is divided into 2 parts: The Dose Escalation Phase and the Cohort Expansion Phase. The phase that participants will enroll to will depend on when they enter the study. The age range for participants is from 12 months to 25 years. Participants can expect to be in this study for approximately 14 months.
Growth hormone may increase the size of tonsils in the airway. Children who take growth hormone may be at an increased risk for sleep apnea. The goal of this study is to check for sleep problems in children before and after starting growth hormone. This research will also help us know if and when other children who start growth hormone need to be checked for sleep problems.
This study is for patients that have newly diagnosed High-Risk B-ALL, Risk-Adapted Post-Induction therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy. The treatment involves medicine called chemotherapy, which fights cancer. Some patients may also need radiation therapy depending on whether the cancer has spread to the brain and spinal fluid, or the testes for males. The investigational drug on this study is inotuzumab ozogamicin. Participants can expect to be on this study for a little over 2 years and followed for up to 10 years.
This study is for patients that have been diagnosed with Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT). The treatment involves cancer fighting medicine called chemotherapy plus radiation therapy and/or surgery. Participants can expect to be on this study for up to 10 months and be followed by the treatment team for up to 5 years.
We will aim to enroll children who have had a nasal swab test showing infection with SARS-CoV-2, the virus that causes COVID-19, or children with a serious complication of COVID-19 called the Multi-system Inflammatory Syndrome in Children (MIS-C). This study does not offer any treatment for COVID-19 or MIS-C. The purpose of the study is to collect information that will help us understand what happens to children and young adults who get infected with SARS-CoV-2, and how their immune system responds. Most of the information that we will collect is part of usual care for young people who have COVID-19 or MIS-C. In addition, we will do some extra clinical and blood tests. Children and young adults who are enrolled in the study will be followed for one year. Our goal is to enroll up to 250 children and young adults (up to the time of your 21st birthday) from about 20 hospitals in the United States. At our center we plan to enroll about 20 participants.
This observational study will determine the spectrum and time course of coronary artery involvement, left ventricular (LV) systolic function, and arrhythmias or conduction system disturbances within the first year from illness onset, and to define associated clinical and laboratory factors in a cohort of MIS-C affected patients.
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.