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.

Summary

In a randomized Phase 2b trial of 261 preterm infants with early RDS, high-dose aerosolized surfactant (APC-0101) did not reduce liquid surfactant instillation compared with control, while an exploratory analysis suggested a reduction with low-dose APC-0101. No differences were observed in other respiratory outcomes.

Key Findings

• Randomized 261 preterm infants (26–31 weeks) with early RDS to control, low-dose, or high-dose APC-0101
• High-dose APC-0101 did not reduce liquid surfactant instillation vs control (44.2% vs 50.0%; P=0.648, ITT)
• Exploratory analysis showed lower instillation in low-dose APC-0101 (34.9%; P=0.045 vs control); no differences in other respiratory outcomes

Clinical Implications

Current evidence does not support routine use of high-dose aerosolized surfactant via APC-0101 for early RDS; low-dose regimens warrant confirmatory trials before clinical adoption.

Limitations

• Primary endpoint negative at pre-specified high dose; low-dose effect is exploratory and hypothesis-generating
• Trial not powered for long-term outcomes; potential dose–response uncertainty

Future Directions

Conduct a confirmatory RCT focused on optimized low-dose regimens, with standardized weaning criteria and longer-term respiratory and neurodevelopmental outcomes.