Juvenile idiopathic arthritis (JIA) is the most common childhood rheumatic disease in the world. JIA causes swelling and pain in joints. There is no cure for JIA. Children with JIA may have a lower quality of life and more disability when not treated well. The goal of treatment is to stop joint swelling so children can grow and develop normally.
There are many types of JIA. Polyarticular course (pcJIA) is one type where children have five or more swollen joints. These children are at high risk for not doing well. Their disease may stay active, and they could become disabled. Quickly finding treatment that works well is doctors’ goal and parents’ expectation.
Over the last 20 years, many new drugs have been approved to treat JIA. One class of drugs are tumor necrosis factor alpha inhibitors (TNFi). TNFi are most often the first biologic drug used to treat children with pcJIA. Unfortunately, at least 25 percent of children with pcJIA do not get better after taking the first TNFi and there are no data to help decide what to try next. Doctors and families use trial and error, trying one different drug after another, which often takes longer to control the disease. The delays raise the chance of poor long-term outcomes. Also, children take drugs that may not work but have risks. The SMART-JIA study looks to get data to direct treatment decisions in children with pcJIA who do not get better after taking a first TNFi.
SMART-JIA will compare the effectiveness of treatment plans for children with pcJIA. The project team will test using a second TNFi versus three drugs that work in ways different from a TNFi and that work differently from each other. SMART-JIA is a type of study where everyone involved knows which drug is given. This is called an open label study. Drug choices will be randomized, which means they will be made by chance. Seventy pediatric rheumatology centers from all over the world will be part of the study.
Children with pcJIA who have not gotten better after treatment with a first TNFi are eligible to join the study. Once they agree to join, they will be randomized to find out the drug they will take. Children in the study will be followed closely by their doctor to measure response to the drug and assure safety. If the child does not get better enough taking the assigned drug over three to nine months, they will be randomized for a second time to a different drug. Each child in the study will be followed for 12 months. At the end of the study, the study team will learn what order of drugs works best in each child.
A team of expert scientists, doctors, caregivers and research staff from all over the world have been gathered to plan and carry out the study. The proposed study is groundbreaking in its novel design. It is the first international collaboration between the Childhood Arthritis and Rheumatology Alliance, the Pediatric Rheumatology European Society, and the Pediatric Rheumatology International Trials Organisation.
The study team believes SMART-JIA will provide much needed proof to direct drug choices for children with pcJIA who are hard to treat. The study plan will be a model for answering treatment questions in childhood rheumatic diseases as well as other rare diseases.
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.