UTHealth Houston to lead data coordinating center on $11.4M multi-site clinical trial
UTHealth Houston researchers will serve as the principal investigators of the data coordinating center on an $11.4 million, groundbreaking clinical trial studying the efficacy of inhaled nitric oxide (iNO) in treating newborns with congenital diaphragmatic hernia (CDH). The study aims to evaluate the discontinuation of iNO during postnatal resuscitation and stabilization, working in collaboration with 19 institutions across the United States.
Funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health, the study will investigate differences in the combined outcome of life support use on mortality among newborns. Researchers also will assess the cost-effectiveness of iNO and its impact on patient care and future practice.
CDH is a rare condition occurring in one to four per 10,000 births (1). The condition occurs when the diaphragm of the fetus does not fully develop, leaving a hole between the abdomen and chest. The trial will analyze the de-implementation of the current, common practice of iNO for fetuses with CDH.
“This will be a landmark trial where we investigate a specific therapy, factors driving clinician behaviors, and the economic implications for neonates with a very complex congenital anomaly,” said associate professor of pediatric surgery, Matthew T. Harting, MD, MS, a co-PI on the trial. “This team effort combines the strengths of McGovern Medical School, such as clinical care and an established, multicenter, collaborative study group, with the strengths of the School of Public Health, including clinical trial management and data coordination.”
Jose-Miguel Yamal, PhD, professor in Biostatistics and Data Science at UTHealth Houston School of Public Health, will co-lead the data coordinating center of the study, along with Harting. The team will oversee data management, quality control, interim analyses, and communication with the Data Safety Monitoring Board to ensure protocols are followed in this randomized trial.
Among the 19 collaborating institutions, each will play a critical role in assessing the discontinuation of iNO as it will examine the effectiveness across 900 newborns by comparing health outcomes before and after de-implementation.
The study also will explore how each site and participating physicians will apply the de-implementation of current iNO practices, assuming its outcomes may be non-inferior to current practices. “No randomized trials have been conducted to determine whether iNO should be used for these neonates with CDH. Previous observational evidence suggests a lack of effectiveness and potential harm from iNO among neonates with CDH, yet it is still widely used. Our study aims to provide this much needed information to guide clinical care,” said Yamal, director of the Coordinating Center for Clinical Trials at the School of Public Health.
This trial represents a significant step forward in improving outcomes for infants with CDH and will guide successful strategies for future care of CDH patients.
Additional investigators overseeing the data coordinating center at UTHealth Houston include associate professor in the Department of Pediatrics, Claudia Pedroza, PhD. Further, the companion clinical coordinating center, through the University of Tennessee, includes co-PIs Timothy Jancelewicz, MD MA MS; Martin Blakely, MD MS; and Maria Fernandez, PhD.