Project Summary

PCORI has identified the need for large studies that look at real-life questions faced by diverse patients, caregivers, and clinicians. To address this need, PCORI launched the Pragmatic Clinical Studies initiative in 2014. Pragmatic clinical studies allow for larger-scale studies with longer timelines to compare the benefits and harms of two or more approaches known to be effective for preventing, diagnosing, treating, or managing a disease or symptom. They focus on everyday care for a wide range of patients. This research project is one of the studies PCORI awarded as part of this program.

This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final. In the meantime, results have been published in peer-reviewed journals, as listed below.

What is the research about?

Crohn’s disease affects about 600,000 Americans, including approximately 40,000 children. The condition causes inflammation in the digestive tract. People with Crohn’s disease experience abdominal pain, cramping, and bloody diarrhea that can reduce their quality of life and may even require hospitalization or surgery. For children, Crohn’s disease often gets worse over time and may affect both physical and emotional development.

Most people with Crohn’s disease need lifelong treatment. Doctors may prescribe antibiotics, steroids, or other types of medicines that reduce inflammation or change the way the immune system behaves. This study focuses on children and young adults with Crohn’s disease who are starting treatment with medicine called anti-TNF treatment to reduce inflammation. The study looks at whether anti-TNF treatment works better alone or with a medicine called low-dose methotrexate, which reduces the body’s immune response.

Who can this research help?

Children and young adults with Crohn’s disease, their caregivers, and their doctors can use information from this study to help choose a treatment.

What is the research team doing?

The research team is recruiting 425 children and young adults with Crohn’s disease at up to 50 medical centers across the United States. ImproveCareNow—a network of patients, families, researchers, and healthcare professionals who are affected by Crohn’s disease or doing research on it—is helping the team to identify people under age 21 who have Crohn’s disease and are starting anti-TNF treatment.

All patients in the study take anti-TNF treatment. In addition, the research team is assigning patients by chance to take either a low dose of methotrexate or a sugar pill that has no medical effect. Patients do not know whether they are receiving methotrexate or the sugar pill. The team is following patients for two years to track whether symptoms get better and whether the improvement lasts. They are also looking at whether any symptoms that reappear need to be treated with steroids, hospitalization, or surgery, and whether patients develop side effects. The research team is also tracking other symptoms that are important to patients, such as pain and tiredness.

The research team is involving healthcare professionals in planning the study. The team is also working with an advisory group that includes patients, parents, hospitals, healthcare professionals, a Crohn’s disease organization, pharmaceutical companies, and organizations that pay for health care.

Research methods at a glance

Design Element Description
Study Design Randomized controlled trial

People under 21 years with Crohn’s disease who are starting anti-TNF treatment

  • Low-dose orgal methotrexate
  • Sugar pill

Primary: remission of symptoms without steroids, hospitalization, or surgery 

Secondary: patient-reported pain and fatigue, occurrence of moderate or serious side effects related to treatment

Timeframe 2-year follow-up for primary outcome

Project Information

Michael Kappelman, MD, MPH
The University of North Carolina at Chapel Hill
Anti-TNF Monotherapy versus Combination Therapy with Low Dose Methotrexate in Pediatric Crohn’s Disease

Key Dates

April 2015
September 2023

Study Registration Information


Has Results
Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: April 3, 2023