UNC Children’s researcher awarded $8 million to determine superior treatment for Crohn’s disease

The Patient-Centered Outcomes Research Institute has awarded UNC's Dr. Michael Kappelman a 5-year, $8 million contract to lead a clinical trial comparing the effectiveness of two drug therapies in the treatment of Crohn’s disease in children.

UNC Children’s researcher awarded $8 million to determine superior treatment for Crohn’s disease click to enlarge Dr. Michael Kappelman

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Thursday, April 23, 2015

A multi-center, pragmatic clinical trial led by the University of North Carolina has been approved for an $8 million funding award by the Patient-Centered Outcomes Research Institute (PCORI). The study, led by Michael Kappelman, MD, MPH, aims to answer one of the most pressing questions parents of children diagnosed with Crohn’s disease face: which treatment will be most effective and cause the least side effects?

Crohn’s disease is an inflammatory bowel disease that affects approximately 38,000 children in the U.S., causing debilitating abdominal pain and bloody diarrhea and leading to frequent hospitalizations and surgery. 

“Treatment typically involves suppressing inflammation with drug therapies, balancing potential benefits against risks that can be life threatening,” explains Kappelman, an associate professor of pediatrics in the UNC School of Medicine and the study's principal investigator. “Parents turn to their child’s gastroenterologist to guide them in health decisions, but there are gaps in research where we can’t definitively say which therapy is better.”

One of those “gaps in research” motivated Kappelman to apply for funding through PCORI’s Pragmatic Clinical Studies Initiative. Anti-tumor necrosis factor, or anti-TNF, drugs are a class of drugs that has been used for more than a decade in the treatment of inflammatory diseases like rheumatoid arthritis. More recently, they have had a major impact in the treatment of Crohn’s disease. What remains unknown, however, is whether using anti-TNF drugs alone or in combination with methotrexate, another immune suppressant, leads to better outcomes.

The multi-disciplinary research team, which includes investigators from more than 50 pediatric gastroenterology programs across the country, will recruit participants through ImproveCareNow, a network of more 575 gastroenterologists in 71 practices. It will enlist 425 individuals under 18 years old with moderate to severe Crohn’s disease who are initiating anti-TNF therapy. Participants in the double-blind study will be randomized to receive either anti-TNF plus low dose oral methotrexate (combination therapy) or anti-TNF alone (monotherapy), and they will be followed for two years.

“This study will address one of the most challenging treatment decisions faced by patients with pediatric Crohn's disease, their families, and their physicians,” says Kappelman. “It presents a tremendous opportunity to generate strong scientific knowledge that will directly inform and impact patient care—and for the pediatric gastroenterology community to further integrate research and clinical care so that we can learn from each and every patient encounter.”

Unique among clinical trials, parents and patients will be involved in all aspects of planning and conducting the trial and will also guide the development of recruitment materials and the informed consent process.

“Time and again, basic questions about children’s illnesses are understudied,” says David Wohl, MD, a UNC physician and Crohn’s disease father, who will lead the study’s parent engagement core. “This trial represents an unprecedented collaboration between researchers and parents as partners. As the parent of a child who struggles with Crohn’s disease, I am excited to see this study launched and hope parents at UNC and the other centers will help us learn what works best for our kids.”

PCORI’s Pragmatic Clinical Studies Initiative is an effort to produce results that are broadly applicable to a diverse range of patients and care situations and can be more quickly taken up in routine clinical practice. Pragmatic clinical studies test a treatment’s effectiveness in “real-life” practice situations, such as typical hospitals and outpatient clinics, and can include a wider range of study participants, making their findings more generally applicable.

“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other healthcare stakeholders in a major study conducted in real-world settings, but also for its potential to fill a crucial evidence gap and answer an important question about the treatment of pediatric Crohn’s disease,” says Joe Selby, MD, MPH, executive director of PCORI. “We look forward to following the study’s progress and working with UNC to share its results.”

PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions.

UNC’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract. For more information about PCORI’s funding awards, visit the Research and Results page at pcori.org.

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