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. You can also look for studies that offer remote participation from home!
The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as UCART22 (“study drug”). UCART22 cells are special types of cells called T-cells that have been collected from a donor and then are genetically modified (changed). T-cells are related to your immune system, which helps to fight infection. These T-cells will be changed in a way that will allow the cells to identify and kill your tumor cells.
Patients with B-cell Acute Lymphoblastic Leukemia (B-ALL) whose disease has come back or have not responded to treatment may be eligible for this study.
CAR T cell therapy has shifted the treatment landscape for pediatric and young adult patients with advanced B-cell leukemias. However, the manufacturing process remains in its first generation: difficult, time-consuming, and not automated. The time and significant resources in this process can result in patient safety issues - with patients growing sicker, with harder to control leukemias - in the waiting period. Use of the CliniMACS Prodigy platform can help to surmount these issues. This study will determine the safety and usefulness of using the CliniMACS Prodigy platform to manufacture huCART19 cells.
This is a Phase 1/2b trial for the treatment of patients with either advanced or refractory B cell Acute Lymphoblastic Leukemia or B-lineage lymphoblastic lymphoma who will be treated with huCART19, which has been manufactured using the CliniMACs Prodigy Platform. Possible procedures include medical record review, apheresis, huCART19 infusion, blood draws, physical exams, imaging, and disease assessments through bone marrows and lumbar punctures. Overall, approximately 89 subjects will be enrolled on this clinical trial.
The goal of this research study is to transform outcomes for all people affected by single ventricle heart disease, particularly those with Fontan circulation. The Fontan Outcomes Network (FON) strives to advance our understanding of, and care for, single ventricle heart disease and address some of the major challenges. The ultimate goal is to dramatically improve physical health and functioning, neurodevelopment, and emotional health and resilience. To achieve that, we will collaborate between many care centers and with individuals with single ventricle heart disease and their families, to systematically collect information across the entire lifespan. FON includes a registry study, meaning information will be collected by your doctors and the researchers and entered and stored in a bank of information called a registry to be used for research as well as a learning network for quality improvement initiatives.
The purpose of this investigation is to evaluate the impact of non-persistent and persistent chemical exposures on autoimmunity, gene expression and the microbiome in children with and without Crohn’s disease. If these chemical exposures are associated with differences in the epigenome and microbiome that are known to predispose for Crohn’s disease, the proposed research could further support changes in dietary behaviors to limit exposure, and efforts to regulate manufacturing processes that unintentionally and intentionally add these chemicals to food.
This study aims to design and optimize the Hypothyroidism Treatment Satisfaction Questionnaire (ThyTSQ) for measuring treatment satisfaction among teenage patients on thyroid hormone supplementation and parents of children of any age treated for hypothyroidism.
Inherited Metabolic Disorders (IMD) are single gene conditions that affect metabolic pathways. IMD are individually rare, but collectively common. Advances in diagnosis and management have greatly increased the life expectancy of patients with IMD. Gaps in our knowledge about adult neurocognitive outcomes and quality of life confounds the ability to obtain critical therapeutic supports, including social services and vocational rehabilitation. The lack of information also complicates our ability to provide anticipatory guidance to the families of younger patients about prognosis and how to optimize outcome. This study examines neurocognitive outcomes for adults with IMD with a focus on executive and adaptive function, domains necessary for managing life skills, using an entirely remote format.
Participants in this study will complete online surveys on their life skills, medical self-management skills, and quality of life. Thinking skills will be assessed through online questionnaires and a web-based collection of games that test executive function. Some participants will be asked to take part in an interview about their experience as an adult and the transition from pediatric to adult-centered healthcare.
CAR T cell therapy has shifted the treatment landscape for pediatric and young adult patients with advanced B-cell leukemias. This study aims to treat patients with relapsed disease by administering two CART cell products, huCART19 and CART22-65s. The study will determine the safety and efficacy and usefulness of administering two CAR T cell products in patients with advanced B-cell leukemias.
The purpose of this study is to help children who are tube-fed transition to eating orally. The goal is to evaluate the effectiveness of treatment with an experimental drug (megestrol) as part of a 24-week behavioral feeding protocol in transitioning from tube to oral feedings in a pediatric population. All children will receive feeding skills coaching to help them learn to eat by mouth, and children will follow a tube weaning protocol to transition from tube to oral feeds. More information can be found at www.ikaneat.org
The purpose of this study is to test an investigational medication in children and adolescents aged 5-17 years old with irritability associated with autism spectrum disorder (ASD). The name of the medication being tested in this study is pimavanserin. This medication is investigational because it has not been approved by the FDA for the treatment of irritability associated with autism. The main risks of this study are from the medication, which include nausea, dizziness, and sleepiness.
If you are eligible and agree to participate, you will be randomly assigned to receive either placebo (a pill with no medication in it) or the study medication. Participation will last approximately 15 weeks and involve 8 study visits and one phone call. You will need to take the study drug for 6 weeks.
Are you interested learning about opportunities to participate in research? Sign up to receive information about new research studies at CHOP. It’s a great way to support the work of the Research Institute.