Pediatric Clinical Trials

Clinical trials typically test a specific intervention-- which may be a drug, medical device, or procedure-- following a research protocol developed by an investigator. Children and adolescents are not small adults, and their bodies’ physiology and chemistry react very differently to illness and disease. Thus, pediatric clinical trials are critical to developing treatments and cures for childhood disease and illnesses. The Children’s Hospital of Philadelphia (CHOP) was founded in 1855 on the basic premise that children require their own unique care. CHOP has been a pioneer in the conduct of pediatric clinical trials.

While children and their healthcare providers often have to rely on drugs and devices based on what is known to work in adults, CPCE researchers conduct pediatric clinical trials to create an evidence base for treatment protocols that are safe and effective for children.

CPCE’s pediatric clinical trials are designed to gather data to answer important diagnostic or therapeutic questions for translating ideas and discoveries into clinical practices that save children’s lives. CPCE is designated as a CHOP Research Institute Center of Emphasis, which provides our researchers with access to various support mechanisms throughout the pediatric clinical trial process, which typically includes:

  • developing research questions
  • securing funding
  • developing formal protocol
  • gaining Institutional Review Board (IRB) approval
  • enrolling participants
  • performing study procedures
  • collecting data
  • analyzing results
  • publishing and promoting research findings in peer-reviewed journals

Essential to pediatric clinical trial methodology are human participants, as well as approval from institutional, state and federal authorities to test the given intervention. Clinical trials range in scope from small pilot studies emphasizing safety, to large multi-site studies which may, for example, compare the effect of a new drug with a placebo or another drug. Clinical trials must have the rigor to ensure scientific validity and reproducibility of results. 

CPCE Projects Involving Pediatric Clinical Trial Methodology

Antimicrobial Stewardship

Prophylactic Antibiotics for VesicoUreteral Reflux

Short Course vs. Standard Course Antibiotic Therapy for Common UTI

Shared Decision-making for Critically Ill Pediatric Patients


Find a research study at CHOP.