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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No Description Available
This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver. In the liver is where factor VIII protein is made. The study product, SPK-8011, is available only to participants in this research study. This product has not been approved yet for use in the general public. This will be the first hemophilia A gene transfer research study done in the United States. To learn more about the benefits and risks related to this study, please contact Dr. Lindsey George at (267) 425-0132. In order to participate in this study, you must be male, 18 years or older, have hemophilia A with FVIII activity levels less or equal to 2%, a history of bleeding that requires prophylaxis or on demand therapy, and no history of inhibitor to factor VIII. This study requires a number of visits over the course of 12 months. All costs related to participation in the study will either be covered or reimbursed by the Sponsor of the study, Spark Therapeutics, and participants will be compensated for their time.
The TRaCk LIGHT study is a research study that will help doctors and scientists learn whether an investigational medicine can improve the signs and symptoms of Crohn’s disease. The investigational medicine is an injection taken in the abdomen once every 2 weeks that helps block a protein in the intestine that contributes to inflammation. The investigational medicine has not been approved by the US Food and Drug Administration (FDA). You may be able to participate in this study if you are 18 years old or older, have had a diagnosis of Crohn’s disease, and have not responded to or lost response to anti-TNF alpha medicines that treat Crohn’s disease. If interested, please contact Caitlin Walsh at email@example.com or 267-426-9249.
The purpose of this research study is to find out if intranasal oxytocin (the hormone oxytocin, delivered through a nasal spray solution) will help with easy weight gain in children, adolescents, and adults with brain tumors and hypothalamic obesity syndrome. In children and adolescents with hypothalamic obesity syndrome, the production of oxytocin may be too low. Oxytocin is important for the body’s metabolism, or use of energy. Low oxytocin production could be a cause of easy weight gain in people with hypothalamic obesity syndrome. Participants will undergo a telephone screening and then an in-person screening visit to determine eligibility. Subjects will be randomized and begin 8 weeks of treatment with either Oxytocin or placebo. After a 4-week "washout", participants will cross-over to 8 weeks of treatment with the other condition (i.e., placebo or OXT). The proposed study has the potential to identify a rational, effective, and safe treatment strategy for a highly morbid condition for which no alternatives currently exist.
Patients with who have had Fontan surgery are at risk for the development of liver fibrosis and cirrhosis. New MRI based imaging techniques allow for non-invasive assessment of liver stiffness and fibrosis. These methods have been used in other diseases, but have not been studied in those who have undergone the Fontan operation. In this study we will test the feasibility of MRI based methods for the assessment of liver disease, and compare the results of these studies to those obtained from clinical liver biopsy.
The goal of this project is to work with CHOP families to build a database of volunteers who will help move science forward, faster. In order to be successful, we need families like yours to join our database so it can grow to be a useful resource for CHOP researchers to learn more about topics like autism, more quickly.
The purpose of this study is to see if a new investigational drug (Upadacitinib) is safe and well tolerated and also to determine the appropriate dose for subjects with polyarticular course juvenile arthritis (pcJIA). The study is a two part study; in Part 1 subjects will be categorized based on age group, and the dose administered for seven consecutive days will be based on four different body weight categories. Subjects who complete Part 1 and are benefiting from study drug with no ongoing adverse events of special interest or serious adverse events, based on investigator's clinical judgment and with subject/family's agreement, will have the option to enroll in Part 2 to receive open-label upadacitinib. Participants will remain in part 2 for up to 156 weeks. Researchers will also be testing the pharmacokinetics (PK) of the study drug, which looks at how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body. Subjects will be asked to come into the Rheumatology clinic for blood and urine testing, a joint exam, and administration of study drug.
The Center for Pediatric Inflammatory Bowel Disease is conducting a research study to determine if a structured yoga program, in addition to standard medical treatment, improves quality of life in pediatric patients diagnosed with Inflammatory Bowel Disease. Participants between the ages of 10 and 17 will participate in 12 yoga classes, offered once a week at CHOP outpatient locations or in community settings. The classes include relaxation techniques, breathing exercises and yoga poses felt to be beneficial for a healthy digestive system. Participants also answer a series of questionnaires, four times over the course of the study.
No Description Available
This is a randomized controlled trial that tests whether a program designed to maintain a high fluid intake decreases kidney stone recurrence. Patients 12 years of age or older who have had a kidney stone in the last 3 years may be eligible.