Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling.
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This is an intergrated study of patients with congenital glycosylation disorder, including genetic conditions in the synthesis of glycoconjugates (congenital disorder of glycosyaltion), degradation of glycoconjugates (majority of lysosomal storage conditions), as well as the metabolism of glycoconjugates (such as certain inborn errors of metabolism that involved in nucleitide sugar metabolism, such as galactosemia, fructose intolarance, or certain glycogen storage conditions). Study methods includes glycomics analysis of body fluid or cells or tissues from patients, relevant metabolomics and genomics analysis.
The purpose of the TII study is to see how the prebiotic inulin changes the gut bacteria (microbiome) of children and young adults (ages 8-21) with IBD and determine if this dietary intervention can help reduce disease activity.
In patients with systemic juvenile idiopathic arthritis (sJIA), it is often difficult for physicians to distinguish between infection and inflammatory disease activity. The purpose of this study is to see if two biomarkers, called CP and PCT, can help physicians identify when patients have an infection versus a flare of their sJIA. This study involves two study visits, one at the time of a sJIA flare and one approximately 60 days after. We will ask participants to provide 3 teaspoons of blood for laboratory assessments, as well as answer brief questionnaires about their disease. The researchers will also review each participant's medical records for information regarding the history of their disease.
We are looking at how infants and young children breathe in different positions. This will help us understand how children with different types of lung hypoplasia breathe and how the lung changes and grows. To do this, we will measure regional lung ventilation by electrical impedance tomography (EIT) in three positions: lying on the back, right- and left-side in children with pulmonary hypoplasia and in healthy children without lung disease. EIT has been well established as a safe, non-invasive, radiation free method of assessing regional lung ventilation. There is no compensation available for this study.
CHOP is conducting a research study that will help make it possible to do future studies in children and family members both with and without problems with their brain and nervous system. This study involving collecting blood and other types of samples from participants along with medical information. These samples and the information will be stored in a "repository" for an indefinite amount of time. We'll use these tools in the future to create and develop new research studies that may help us find answers to some of these difficult illnesses.
The purpose of this research study is to help us understand how well tofacitinib works as a treatment for ulcerative colitis or indeterminate colitis in pediatric patients. We are looking to enroll, males or females ages 6 to 21 years old with a diagnosis of ulcerative colitis or indeterminate colitis who have started tofacitinib therapy prescribed by their primary gastroenterologist within the past 2 weeks or will be starting tofafacitinib within 1 month.
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 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.
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 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.