The Pediatric Trials Network (PTN) plans to open the third and final cohort (or group of study participants), in the Safety of Sildenafil in Premature Infants study in the near future. A total of 80 premature infants have been enrolled in the study so far, into the first and second study cohorts. These first and second cohorts received 0.125 mg/kg and 0.5 mg/kg of sildenafil in order to determine the safety in premature infants. The third, and final, cohort will receive the highest dose of 1 mg/kg.
The study aims to assess the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia (BPD) and determine preliminary effectiveness and pharmacokinetics (how a drug travels through the body). BPD is a common chronic lung disease that can affect premature newborns, often leading to life-long medical problems, prolonged hospitalization, and even death. Approximately 17,500 U.S. infants develop BPD each year.
Sildenafil, which is approved for the treatment of pulmonary hypertension in adults, may help improve lung development and is increasingly being used off-label in premature infants with BPD. However, the efficacy and safety of sildenafil in premature infants at risk for BPD is currently unknown.
“More than 17,000 infants develop BPD each year so finding a drug that can help is an important public health goal. BPD can create prolonged hospitalization and in some cases, death. We hope that our study findings will help support families of infants with BPD so that there are less unknowns regarding the effectiveness of Sildenafil in treatment of infants with BPD.” said Dr. Jason Lang, the study’s Principal Investigator.
To learn more about Safety of Sildenafil in Premature Infants study, visit ClinicalTrials.gov.