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 research study is to learn more about the normal function of blood vessels in healthy children and adolescents. This will help us discover differences between healthy individuals and children with lupus. Healthy control participants will be asked to make 1 visit to CHOP to complete a brief survey, a fasting blood draw and a test to measure the blood pressure in your fingers.
The purpose of this study is to learn how to monitor heart health in children and teens with lupus. This information will help us find ways to prevent early heart disease in children and teens with lupus. Participants will visit CHOP 3 times over 18 months. During the visits, participants will complete a fasting blood draw and imaging tests of the heart and blood vessels. Participants will also be asked to wear a blood pressure monitor for 24 hours at home after each visit.
Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, 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, 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. These stem cells will then be modified using gene editing to increase fetal hemoglobin production. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with the edited gene, will then be given back to you. You will be followed for side effects and to see if the cells with the edited gene help you make more fetal hemoglobin.
The purpose of this study is to find out if an investigational gene therapy called HMI-102 works and may be tolerated better than standard treatments for treating phenylketonuria (PKU). You may be eligible for this study if you are between the ages of 18-55 years old and have been diagnosed with classic PKU, due to phenylalanine hydroxylase deficiency. This study includes study drug infusion and hospitalization, blood draws, electrocardiogram, chart review, and questionnaires. Compensation will be provided. If you have questions or would like to learn more about the study, please contact thestudy team at MetabolismResearch@email.chop.edu .
Children and adults with PTEN Hamartoma Tumor Syndrome (PHTS) are at risk for specific long-term medical complications including autism spectrum disorder and cancer. While protocols for cancer screening exist, we do not yet have protocols for screening other complications of this syndrome, including neurobehavioral differences and immunologic changes. This pilot study will assess these outcomes in children and young adults with PHTS ages 1 to 20 years.
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-8016, is available only to participants in this research study. This product has not been approved yet for use in the general public. 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%, and a history of bleeding that requires prophylaxis or on demand therapy. 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.