Venkatesh Sampath, MBBS, MRCPCH

Sosland Endowed Chair in Neonatal Research; Director, Neonatal Diseases Research Program; Professor of Pediatrics, University of Missouri-Kansas City School of Medicine; Research Associate Professor of Pediatrics, University of Kansas School of Medicine

Full Biography

Venkatesh Sampath, MBBS, MRCPCH, Sosland Chair in Neonatology, received a $446,100 Translational Research in Maternal and Pediatric Pharmacology and Therapeutics (R21 Clinical Trial Optional) grant from the National Institute of Health’s Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

The title for Dr. Sampath’s study is “Inhaled ciclesonide - a phase I study in preterm infants” and the grant covers a project period of Aug. 5, 2024-July 31, 2026 (Grant Number: 1R21HD116421-01).

This study investigates the use of ciclesonide (CIC), a synthetic glucocorticoid (sGC) pro-drug that in the inhaled form is FDA approved for use in asthma and allergic rhinitis in older children, to prevent bronchopulmonary dysplasia (BPD) in preterm infants. BPD is a chronic lung disease which affects premature infants born before 31 weeks gestation. Infants who develop BPD are at risk of death and often develop long-term respiratory and neurodevelopmental impairments.

Clinical trials have shown that current treatments for BPD such as the natural glucocorticoid, hydrocortisone, or synthetic steroids such as dexamethasone (DEX) either don’t prevent BPD or prevent BPD at the risk of severe side effects such as cerebral palsy and growth suppression.

 “In neonatal models of brain and lung injury, our team has shown that ciclesonide is efficacious in preventing lung injury without causing significant brain or systemic toxicity compared to other steroids,” said Dr. Sampath. “We are very enthusiastic about ciclesonide’s potential in preterm infants.”

In this study, the team will conduct a phase I dose escalation clinical study using an inhaled preparation of ciclesonide to establish its safety and toxicity profile in preterm infants, while also assessing levels of CIC metabolites and glucocorticoid receptor activation profile. The team will also study the ability of CIC to help preterm infants wean off respiratory support.

“We believe our study is impactful and translationally relevant as it addresses an unmet need for effective GC therapy to prevent BPD in premature infants without encumbering the neurological and bodily adverse effects,” said Dr. Sampath. “Successful testing of our hypothesis will pave the way for a large, multicenter randomized control trial of CIC therapy in premature infants to prevent BPD.”

Co-investigators on the study include Mariana Theodoro, MD, Neonatology, Hung-Wen “Henry” Yeh, PhD, MS, Health Services and Outcomes Research, Children’s Mercy, Donald DeFranco, PhD, University of Pittsburgh, and Namasivayam Ambalavanan, MD, University of Alabama at Birmingham. Cheri Gauldin, BSN, CCRC, and Heather Menden, MS, will help in conducting of the study.

The contents are those of the investigator and do not necessarily represent the official views of, nor an endorsement, by NIH, or the U.S. Government.