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.
Samiee-Zafarghandy S, Van den Anker JN, Laughon MM, Clark RH, Smith PB, and Hornik CP, Sildenafil and Retinopathy of Prematurity Risk in Very Low Birth Weight Infants, on behalf of the Best Pharmaceuticals for Children’s Act- Pediatric Trials Network Administrative Core Committee
American Journal of Perinatology • February 1, 2016, volume 36, issue 2, pages 137-140.
PubMed Central ID (PMCID): PMC4731250 [Free access available on 8/1/2016]
Matthew Laughon, MD, MPH
University of North Carolina
Chapel Hill, NC
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