Children's Hospital of Philadelphia leads or participates in hundreds of clinical trials. Use this database to learn the purpose of these trials, find out who can participate and more.
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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.
Children between the ages of 4 and 16 years old, both with and without cerebral palsy, may be eligible for the study to help researchers learn if there is a difference in sensory processes. The study involves one visit that lasts approximately 25 minutes and includes a medical record review, a brief physical exam to measure body length, and low levels of electrical stimulation delivered to the ankle.
Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, may be able to participate. A number of screening tests will be done to determine if subjects can participate. These include review of your past medical history, blood and urine tests, bone marrow aspirate/biopsy, bone age/ DEXA scan, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and liver biopsy. If eligible, blood stem cells will be collected by apheresis or by bone marrow harvest. These stem cells will then have a healthy beta globin gene inserted. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene, will then be given back to you. You will be followed for side effects and to see if the inserted gene helps you make hemoglobin.
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.
Dr. William Gaetz. has partnered with pediatric neurologist, Dr. Eric Marsh and neuropsychologist, Dr. Lisa Blaskey to better understand Epilepsy in children who have an Autism Spectrum Disorder (ASD). This study will use a non-invasive brain scanning technology as well as cognitive testing results to understand brain differences in children with Autism and Epilepsy. We hope that the information we get from this study will help children in the future who have ASD. For this research to be successful we need to work with male children with a diagnosis of ASD or ASD and Epilepsy who are 8 to 17 years of age. Families will be reimbursed for their time and have an opportunity to discus their child's cognitive testing results with our neuropsychologist post testing. Please contact our study staff at MEGstudies@email.chop.edu for more information.
This study seeks to understand why some babies born with a small jaw (micrognathia) have sleep apnea and/or trouble growing and others do not. By enrolling healthy babies, we hope to get a better understanding of how infants breathe during sleep and grow over the first months of life. This observational study involves two study visits that each include an overnight sleep study and some other non-invasive tests.