E.W. "Al" Thrasher
Status: Funded - Open
Steroids and Surfactant in Extremely Low Gestational Age Infants (SASSIE), Pilot Dose Escalation Trial
Cynthia McEvoy, MD
BACKGROUND: Extremely low gestational age newborns (ELGANS) are at high risk for bronchopulmonary dysplasia (BPD), a chronic lung disease that is associated with death or continuing pulmonary disease, including asthma, as well as neurodevelopmental disability; currently there no highly effect preventative treatments. Recent pilot studies in which lungs of infants with respiratory distress syndrome were treated with budesonide, a synthetic glucocorticoid with strong anti-inflammatory action, suspended in surfactant found reduced BPD without apparent adverse effects.
GAP: Information is needed to determine the optimal dose and frequency of treatment with budesonide administered in surfactant for both benefit and safety before performing a large clinical study among critically ill infants.
HYPOTHESIS: We hypothesize that an appropriate dose of budesonide delivered directly to the lung in surfactant will be effective and safe in decreasing the short term clinical respiratory needs and the inflammatory response that contributes to BPD in ventilated preterm infants.
METHODS: We will treat 8 infants at each of 4 dosing levels of budesonide in surfactant, starting with the lowest dose, then evaluating benefit and safety parameters before treating the next group of infants (total of 32 infants). ELGANS who require intubation and mechanical ventilation between 3 and 10 days after birth will be enrolled; clinical data, safety data, lung fluid, and blood samples will be collected to measure drug efficacy.
RESULTS: Results are pending.
IMPACT: Results from this dose escalation study will allow us to apply to the National Heart Lung and Blood Institute to fund a large clinical trial that will be carried out by our established network of Neonatal Intensive Care Units; positive results will lead to a change in practice in the care of premature infants and reduce the risk for continuing lung disease and neurological impairment.
Neonatal, Pharmacokinetics, Bronchopulmonary Displasia, Respiratory, Treatment, Human
Oregon Health & Science University
California, Oregon, Utah, Florida, Florida, California