Safety and Efficacy of a Novel Aerosolized Surfactant (APC-0101) for the Treatment of Respiratory Distress Syndrome in Preterm Infants: A Randomized Phase 2b Study.
David J Durand, Mark L Hudak, Amish Jain et al.
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In 30 seconds
This multicenter, randomized phase 2b trial evaluated two doses of aerosolized surfactant (APC-0101) in 261 preterm infants born at 26–31 weeks’ gestation with early respiratory distress syndrome receiving noninvasive respiratory support. The primary outcome was subsequent use of instilled liquid surfactant. This occurred in 50.0% of controls and 44.2% of infants assigned the prespecified high dose (P = .648), which did not demonstrate a reduction. An exploratory analysis found a rate of 34.9% with the low dose (P = .045 vs control); other respiratory outcomes did not differ between groups.
Key findings
- Instilled liquid surfactant was used in 50.0% of controls versus 44.2% with high-dose APC-0101 (P = .648).
- In an exploratory analysis, liquid surfactant use was 34.9% with low-dose APC-0101 (P = .045 vs control).
- The trial found no differences between groups in other respiratory outcomes.
Why it matters
Aerosolized surfactant could offer an alternative way to deliver surfactant to preterm infants receiving noninvasive support. In this trial, the prespecified high dose did not reduce subsequent liquid surfactant use, while the low-dose signal came from an exploratory analysis. The distinction matters when interpreting efficacy and judging whether further evaluation is warranted.
What to keep in mind
The apparent benefit with low-dose APC-0101 was exploratory, whereas the prespecified high-dose comparison was not significant; no differences were found in other respiratory outcomes.