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Principal Investigator Jeffrey A. Towbin, MD, co-executive director of Le Bonheur’s Heart Institute, and Kathryn Sherman, BSN, RN, clinical research coordinator, enrolled the first patient in the United States in the VALOR clinical trial.

This trial is a phase 2/3, randomized, placebo-controlled, double-blind, clinical study to evaluate the efficacy, safety and pharmacokinetics of vericiguat in pediatric participants with heart failure due to systemic left ventricular systolic dysfunction. This condition is a specific cause of heart failure where the function of the pumping action of the left ventricle of the heart is reduced.

Vericiguat is approved by the U.S. Food and Drug Administration (FDA) for heart failure in adults. This study will help determine if vericiguat can be safely given to infants, children and teenagers with systemic left ventricular systolic dysfunction and see if it will improve their heart failure.

“The Heart Institute continues to be on the forefront of clinical, genetic and basic cardiovascular research and clinical trials focusing on heart failure in children,” said Towbin. “We are proud to have been the first site to enroll a patient in this trial and look forward to what the results could mean for kids.”

Jeffrey A. Towbin, MD, principal investigator for the VALOR clinical trial

Heart failure in children is thought to have an incidence ranging from 0.87 to 7.4 children per 100,000 globally and, in the United States, heart failure-related hospitalizations occur in 11,000 to 14,000 children each year with an in-hospital rate of death as high as 7.4%. Pediatric cardiomyopathy patients have one- and five-year rates of death or transplantation of 31% and 46%, respectively.

Due to differences between children and adults, very few heart failure medications have been tested and approved for use in children. As a result, children have typically received the same treatments and medications that have been tested and approved by the FDA for adults without knowing if the risks and benefits are similar for children.

The majority of cardiovascular medications prescribed to children, in fact, have never been tested specifically for children. Studies like the VALOR clinical trial are important to add to the body of evidence to show which medications work best for children with heart failure.

The Le Bonheur Heart Institute, under Towbin’s leadership, is on the forefront of understanding and treating children with heart failure and this trial is one of the many studies on heart failure and heart muscle disease with which the Heart Institute is involved.