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.

OBJECTIVE

To evaluate the efficacy of 2 different doses of aerosolized surfactant delivered by a drug/device combination system (APC-0101) in reducing the use of instilled liquid surfactant in preterm infants with respiratory distress syndrome (RDS).

STUDY

DESIGN

Two hundred sixty-one preterm infants at 26-31 weeks' gestation with early RDS treated with nasal continuous positive airway pressure or noninvasive ventilation and who had not received instilled liquid surfactant were randomized on the first day of life to 1 of 3 groups: control, low-dose APC-0101, or high-dose APC-0101. Infants in the 2 APC-0101 groups received up to 4 aerosolized surfactant treatments while on nasal continuous positive airway pressure/noninvasive ventilation. The primary outcome was the percentage of infants who were subsequently treated with instilled liquid surfactant. Respiratory severity score thresholds were prespecified for subject selection and treatment algorithms.

RESULTS

In the intent-to-treat sample, the frequency of liquid surfactant instillation in the control group was 50.0% compared with 44.2% in the high-dose APC-0101 group (P = .648). In an exploratory analysis, the incidence of surfactant administration in the low-dose APC-0101 group was 34.9% (P = .045 vs control). There were no differences between groups in other respiratory outcomes.

CONCLUSION

The prespecified high dose of aerosolized surfactant, delivered with the APC-0101 system, did not reduce the use of instilled liquid surfactant in infants with early RDS. An exploratory analysis of the low dose of APC-0101 showed a reduction in instilled liquid surfactant, an observation that may merit evaluation in a subsequent study.

TRIAL REGISTRATION: ClinicalTrials.gov ID NCT03969992, https://clinicaltrials.gov/study/NCT03969992.