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.
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.
This study involves taking a study drug called lurbinectedin. The overall goal of this study is to see if different doses of lurbinectedin are safe and effective at treating children and young adults with recurrent or relapsed solid tumors, including Ewing Sarcoma.
Safe oral feeding is a particular challenge for premature infants and those born with complex congenital heart disease (CHD). Safe oral feeding requires coordination of oral cavity changes with respiration. Neoneur is a novel hand-held mobile device which innovatively measures patterns of oral cavity pressure synchronized with respiration, providing a quantitative assessment of feeding skills. Infants will be fed with the Neoneur device 2-3 times a week from the initiation of oral feeding until discharge by a speech and language pathologist. The feeding assessment takes place during routine feeding, with no changes made to the feeding care plan.
The purpose of this research study is to test how well a common, standardized quality of life tool measures quality of life among children on the autism spectrum who are minimally verbal or have an intellectual disability. Parents/ caregivers of autistic children (ages 5-17) who are minimally verbal or have an intellectual disability are eligible to participate in the study.
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.
This study aims to find out if the study drug, GWP42003-P, works to improve symptoms for people with ASD. The study will also look at:
Other eligibility criteria apply.
The purpose of this study is to evaluate social interactions in adolescents and whether changes in information can influence social behaviors. This study will focus on behavioral assessments of social processing over time in adolescents. The information collected in this study is essential for developing clinical treatments focused on addressing social processes in adolescent anorexia nervosa (AN) and to improve our understanding of social processing in adolescent AN, informing both future research and clinical care.
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.