The purpose of this study is to determine how what you eat and drink (your diet), antibiotics you take, and the bacteria that live in your gut (microbiome) contribute to developing kidney stones. Kidney stone disease, known as nephrolithiasis, is also influenced by products of metabolism (metabolites) that are found in your urine. These factors will be examined in participants at least 4 years of age. Each participant will completed three 24-hour dietary recalls and will provide one stool sample and up to two urine samples. The information from this study will help doctors find new metabolic pathways that can be used for treatment of kidney stones.

The purpose of this study is to find out how well surveys can measure disease activity in kids with lower back arthritis. The study will last for either 3 or 6 months and take 2 or 3 visits. Each visit will last between 30 minutes and 2 hours and have either an MRI plus a survey or just a survey.

While recent research has demonstrated that whole genome sequencing is a powerful first-line diagnostic tool, important questions remain around its long-term impact on downstream clinical management approaches. The investigators hope to address these questions by way of chart review and survey administration in a small population of suspected leukodystrophy patients who receive whole genome sequencing as part of their clinical care.

The purpose of this research study is to see if melatonin improves sleep and the sleep-wake patterns (also known as circadian rhythm) in children, teenagers, and young adults (ages 11-30) with at-risk symptoms. Melatonin is naturally produced in the brain and controls sleep-wake patterns. At-risk symptoms can be feeling like you do not want to do be around other people as much, difficulties with focus, and having beliefs that most people around you don’t have. At-risk symptoms can also be experiences like having jumbled up thoughts, your mind playing tricks on you, or seeing or hearing things that are not there. To see if melatonin changes sleep in youth with at-risk symptoms, we are giving some youth with at-risk symptoms a melatonin pill and some a placebo (a pill without any medication in it) for 6 months.

CTX is typically a highly progressive disease, with case reports of symptoms that first appear at any time from birth through adulthood and then worsen over time. Because idiopathic bilateral cataracts occur at an early age in many children with CTX, biomarker testing of these children presents an opportunity for diagnosing children with CTX. Eligible participants will be asked to give a small blood sample and urine sample, and may have genetic testing done to see if they have the gene for CTX. This study will take place on 1 visit for approximately 30 minutes.

Vancomycin is a commonly administered antibiotic in critically ill children, but it can have unwanted side effects on the kidney (acute kidney injury). There are currently no effective methods to prevent development of acute kidney injury during vancomycin therapy. This study will collect blood and urine samples from children in the CHOP pediatric intensive care unit being prescribed vancomycin in an effort to describe how vancomycin behaves in the blood of critically ill children and to understand the relationship between new tests of kidney injury and function, called biomarkers, and the amount of vancomycin in the body. We hope this study will ultimately help improve the safety and efficacy of intravenous vancomycin administration in children by allowing us to develop an approach to provide personalized vancomycin dosing in critically ill children.

The purpose of this research study is to learn more about improving the treatment and outcomes for patients who have a diagnosis of a single ventricle, such as Hypoplastic Left Heart Syndrome (HLHS) or similar heart defects. You will be asked to fill out a questionnaire called the Ages and Stages Questionnaire about your child. It is used widely in pediatrics to check on babies’ development. We will ask you to fill this out about every three months during your child’s 15 months of life. Also, information will be collected about your child’s surgeries, clinic visits and hospital stays from diagnosis through the first year of life. The information may be used to help us understand an infant's response to surgery and medical treatment and may be used to improve care for children with single ventricles.

This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine less than 15 days per month. Eligible participants will be given either erenumab or placebo, assigned randomly (like the flip of a coin), followed by an open-label phase in which all participants receive erenumab. Erenumab has been FDA-approved for use in adults to decrease the frequency of migraine. Patients may be eligible to participate if they have had migraine for at least one year, and agree to come to CHOP's main hospital for several study visits. Patients whose headache started suddenly (New Daily Persistent Headache), patients whose headache started after a concussion (Post-Traumatic Headache), and patients with severe depression are not eligible for this study.

This is a multicenter natural history and clinical measure study. Our objectives are to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures in FA, collection of quantitative serial clinical data on patients and expansion of our established research network. A secondary objective is to build a parallel DNA and RNA repository for use in large scale translational research including modifier gene studies. We wish to also enroll control subjects for a hearing test and sample collection to compare to data from FA patients.

The purpose of this study is to test if the CD19 CAR T cell product tisagenlecleucel (also referred to as CTL019, or Kymriah™) is safe and has beneficial effects in children and young adults who have newly diagnosed high-risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL) that remains MRD-positive after 2 cycles of chemotherapy. The study is enrolling children and young adults between the ages of 1-25 who have received induction and consolidation chemotherapy for HR B-ALL but were still found to have remaining disease (MRD). If you agree to join in this study, you will get one dose of the tisagenlecleucel cells administered by intravenous infusion and be followed for leukemia response. This study also involves blood draws and assessments. Reimbursement for travel may be provided. If you have questions or would like to learn more about the study, please contact the Cancer Immunotherapy Program at (215) 425-7193. The Principal Investigator is Dr. Shannon Maude and the Immunotherapy Nurse Navigator is Brooke Leibfreid, RN.