Research and Clinical Trials

Research and Clinical Trials

A researcher in the Neonatal Development Collaborative at Penn State examines a specimen under a microscope.

Participate in Research

We have a commitment to improving our understanding of disease, enhancing treatment options, and working to prevent childhood illnesses. You can help by participating.

A Penn State researcher helps a clinical trial patient.

Find a clinical trial

See pediatric research studies that are currently recruiting participants.

Search our active trials.

Contact the Pediatric Clinical Research Office

Research at the Children’s Hospital includes basic science and clinical investigation in a wide spectrum of pediatric fields and involves the University Park campus. This research aims to reduce morbidity and increase the success of treatment of many childhood diseases.

For more information, call 717-531-5656.

Current genetics trials

We are currently involved in three research studies for patients with PKU (phenylketonuria). All of these studies have something to do with Kuvan, a drug used to treat PKU. The studies are sponsored by BioMarin.

  • The first study is a registry that collects information about how patients are doing. This registry is for patients who are currently taking or who have tried or are planning to try Kuvan.
  • The next study is for patients up to age 6. It looks at information about safety, blood Phe levels, neurocognitive function (how the brain is working) and growth for patients with PKU who are responders to Kuvan.
  • The third study is for patients 12 years old and older. This study gathers information about the safety of taking Kuvan and the effect on Phe levels. It also looks at neurocognitive effects (how the brain is working) and the effects on ADHD, depression and anxiety.

Learn more about our Kufan research studies

Call Heidi Watts at 717-531-0003, extension 283765 for more information.

Get involved

Clinical trials are research studies that use human volunteers to advance medical knowledge. Participants are a critical part of the process of developing new and enhanced treatments for diseases and conditions. Find a clinical trial.

Research programs at Children’s Hospital study pediatric cancer, neonatal development, immunotherapy, and cardiovascular and pulmonary function. In the Pediatric Innovation Program, clinicians and engineers collaborate to create medical devices. See all research programs.

Through their generous giving, donors enable us to make rapid advances in research. Research at the Children's Hospital is actively supported by numerous scientific funding agencies and philanthropies, including Children's Miracle Network and Four Diamonds. Learn more about giving.