This study will evaluate the pharmacokinetics and safety of sildenafil in premature infants. Up to 24 infants will be enrolled. Up to 8 will receive a single intravenous dose of sildenafil, and up to 16 additional participants will be enrolled if they receive sildenafil as part of standard care. The study will be open to children of both sexes and all racial and ethnic groups. The study is expected to last approximately 24 months; each subject in the single-dose study will participate for up to 8 days.
The most common health problem associated with premature birth is bronchopulmonary dysplasia. Up to 20% of infants with bronchopulmonary dysplasia develop pulmonary arterial hypertension, and up to 40% of these infants die. Currently, few drugs are available to prevent bronchopulmonary dysplasia, and none can reduce mortality in infants with bronchopulmonary dysplasia and pulmonary arterial hypertension.
Sildenafil is approved by the U.S. Food and Drug Administration for the treatment of pulmonary arterial hypertension in adults. The effectiveness of sildenafil in pediatric pulmonary hypertension patients has not been established. Although sildenafil studies in premature infants at risk for or diagnosed with pulmonary arterial hypertension are limited, the drug is increasingly being used off-label in this fragile population.
This study will determine the single intravenous dose pharmacokinetics of sildenafil in premature infants and enroll infants who are receiving sildenafil as part of local clinical care. We will determine the levels of sildenafil in each infant, thus helping to determine the best dose of sildenafil to reduce pulmonary arterial hypertension in premature infants.
We will enroll up to 24 premature infants less than 28 weeks gestational age. Sildenafil will either be as a single dose (and the infants will be monitored for another 7 days for any drug side effects) or administered as part of local clinical care.
- Sildenafil and retinopathy of prematurity risk in very low birth weight infants American Journal of Perinatology • February 2016. Samiee-Zafarghandy S, van den Anker JN, Laughon MM, Clark RH, Smith PB, Hornik CP; Pharmaceuticals for Children Act – Pediatric Trials Network Administrative Core Committee. We identified premature infants who were discharged from Pediatrix Medical Group neonatal intensive care units from 2003–2012 and who received an ophthalmologic exam. We matched ...
Matthew Laughon, MD, MPH
University of North Carolina
Chapel Hill, NC
- Sildenafil safety study achieves major milestone The Pediatric Trials Network (PTN) recently enrolled the fortieth infant in the Safety of Sildenafil in Premature Infants study. This is a major study milestone, as it rounds out the first cohort (or group of study participants). The study aims to assess the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia (BPD) and ...
- Sildenafil safety studied for premature infants with bronchopulmonary dysplasia The Pediatric Trials Network (PTN), with support from the Eunice Kennedy Shriver National Institute of Child Health and Human Development, is conducting an interventional study to examine the safety and efficacy of sildenafil in treating bronchopulmonary dysplasia (BPD) in premature infants. BPD is a common chronic lung disease that can affect premature newborns, often leading to ...
- A busy week for the PTN sildenafil trial The week of February 18–22 saw many milestones in the PTN sildenafil trial. On Tuesday, the first investigator’s meeting was held. By Wednesday, the trial’s first two sites had been activated — one at the Medical University of South Carolina Children’s Hospital (MUSC) and the other at the University of North Carolina–Chapel Hill. And, on ...