Clinical Trial Investigates New Treatment for Children with Metabolic Dysfunction-Associated Steatotic Liver Disease

March 04, 2024

Researchers at Ann & Robert H. Lurie Children’s Hospital of Chicago are helming a clinical trial studying a new treatment option for adolescents with steatotic liver disease, also commonly referred to as metabolic dysfunction-associated steatotic liver disease (MASLD).

MASLD is the most prevalent liver disease in children and currently there are no effective treatments for it. The condition can lead to cardiovascular complications and severe scarring on the liver (cirrhosis). Some with MASLD ultimately require a liver transplant.

In February, Lurie Children’s investigators, led by Justin Ryder, PhD, an expert in pediatric obesity and vice chair of research for Lurie Children’s Department of Surgery, was awarded a $2.7 million National Institutes of Health grant to study the efficacy of a drug that may improve the condition.

“Currently, there are limited treatment options available for kids with steatotic liver disease,” Dr. Ryder said. “If successful, our study will take a step towards a new medical therapy for this highly prevalent and progressive disease."

The first-in-pediatrics, randomized control trial will investigate whether the diabetes medication known commercially as Jardiance improves MASLD in 12- to 20-year-olds. Known as a mechanistic clinical trial, the effort will also seek to understand how and why the drug affects the disease. Enrollment of patients for the study is expected to begin in late spring in Chicago.

The study is led by Lurie Children’s Dr. Ryder (principal investigator); Dr. Mark Fishbein, attending physician in gastroenterology, hepatology and nutrition; Dr. Laura Torchen, attending physician in endocrinology; and researchers at the University of Minnesota.

Research at Ann & Robert H. Lurie Children’s Hospital of Chicago is conducted through Stanley Manne Children’s Research Institute, which is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s is a nonprofit organization committed to providing access to exceptional care for every child. It is ranked as one of the nation’s top children’s hospitals by U.S. News & World Report. Lurie Children’s is the pediatric training ground for Northwestern University Feinberg School of Medicine.

Learn more at Stanley Manne Children’s Research Institute