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 Thursday, MUSC enrolled the trial’s very first patient.

Congratulations to the MUSC site team—Dr. Andrew Atz (principal investigator) and Patricia Infinger (study coordinator)—for their swift action in helping to kick off this important study!

The PTN sildenafil trial is evaluating the pharmacokinetics and safety of sildenafil in preemies suffering from bronchopulmonary dysplasia, the most common health problem associated with premature birth. 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 death among infants with bronchopulmonary dysplasia and pulmonary arterial hypertension.

Sildenafil is approved by the FDA for the treatment of pulmonary arterial hypertension in adults, but its effectiveness in children has not been determined. In spite of this knowledge gap, sildenafil is increasingly being used off-label in premature infants at risk for or diagnosed with pulmonary arterial hypertension. The PTN hopes to establish the best dose of the drug to reduce pulmonary arterial hypertension in these fragile patients, thereby reducing the potential for harm and optimizing outcomes.

Find out more about the PTN sildenafil trial at clinicaltrials.gov.


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