APC-0101 with noninvasive breathing support for preterm infants with respiratory distress syndrome

A Randomized, Controlled, Blinded, Parallel Group Study of the Safety and Efficacy of APC-0101 (SF-RI 1 Surfactant for Inhalation Combined With a Dedicated Delivery System) in Preterm Infants With Respiratory Distress Syndrome

This will test whether inhaled APC-0101 given with nCPAP/NIV helps preterm infants with respiratory distress syndrome breathe better than nCPAP/NIV alone.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 520 (estimated)
• Ages: 1 Hour to 24 Hours
• Sex: All
• Sponsor: Aerogen Pharma Limited (Industry-sponsored)
• Locations: 12 sites (Little Rock, Arkansas and 11 other locations)
• Trial ID: NCT06776783 on ClinicalTrials.gov

What this trial studies

This is a two-part, prospective, randomized, blinded, sham-controlled, multicenter Phase 3 study comparing inhaled APC-0101 plus noninvasive respiratory support (nCPAP/NIV) to nCPAP/NIV with a sham delivery. APC-0101 consists of an inhaled surfactant formulation delivered via a dedicated device and is given to eligible preterm infants soon after birth; the control group receives standard nCPAP/NIV without active surfactant. In Part 1 infants are followed until 40 weeks post-menstrual age or NICU discharge, and in Part 2 selected infants undergo follow-up through 24 months corrected age to capture longer-term outcomes. Randomization occurs within 1–24 hours of life for inborn infants of 26–33 weeks gestation who meet respiratory support and imaging criteria.

Who should consider this trial

Good fit: Ideal candidates are inborn preterm infants born at 26–33 weeks gestation, 1–24 hours old, weighing ≤2000 g and appropriate for gestational age, who are on nCPAP/NIV with FiO2 ≥ 0.24 and imaging consistent with RDS.

Not a fit: Infants who are already intubated or have received prior surfactant, those on excluded respiratory supports (e.g., high-flow >2 LPM, SiPAP/RAM cannula), extremely preterm infants outside the gestational window, or those with key exclusion conditions are unlikely to benefit from this approach.

Why it matters

Potential benefit: If successful, the intervention could reduce the need for intubation and instilled surfactant and improve short-term respiratory stability and longer-term lung outcomes for some preterm infants.

How similar studies have performed: Previous small studies of aerosolized or inhaled surfactant have shown mixed but promising results, so the approach is plausible but not yet proven in large trials.

Eligibility criteria

Inclusion Criteria:

1. Inborn at the study site's hospital (i.e., not transferred from another hospital following delivery)
2. Gestational age at birth of 26 through 33 weeks PMA
3. Birth weight appropriate for gestational age (AGA, weight 3rd to 97th percentile on Fenton Growth Curve)
4. Birth weight ≤ 2000 grams
5. Post-natal age 1 to 24 hours at randomization
6. On nCPAP or NIV for at least 30 minutes with RSS = 1.4 - 2.0 to maintain SpO2 90-95% at randomization. RSS is calculated as (nCPAP cm H2O) × (FiO2) or as (NIV mean airway pressure cm H2O) × (FiO2).
7. FiO2 ≥ 0.24 at randomization
8. nCPAP or mPaw ≥ 6 cm H2O at randomization
9. Chest radiograph (CXR) or lung ultrasound compatible with RDS prior to randomization

Exclusion Criteria:

1. On SiPAP®, RAM® cannula, or high flow nasal cannula (HFNC) (\> 2 liters per minute \[LPM\]) at the time of randomization
2. Prior instillation of surfactant
3. Premature rupture of membranes (PROM) occurring \> 14 days before birth
4. Significant congenital/chromosomal anomaly (e.g., Pierre Robin syndrome, clinically significant congenital heart disease, or trisomy)
5. Pneumothorax
6. Other etiologies of respiratory distress
7. Enrollment in another interventional study with similar efficacy endpoints
8. Apgar score at 5 min of 0-3
9. Prior cardiopulmonary resuscitation (CPR) or epinephrine
10. Base Deficit \> 15 mEq/L on most recent arterial blood gas (not capillary blood gas, venous blood gas, or cord gas) prior to randomization. Note that arterial blood gas is not required prior to randomization.
11. Partial pressure of carbon dioxide (PaCO2) \> 65 mmHg on most recent arterial blood gas (not capillary blood gas, venous blood gas, or cord gas) prior to randomization.
12. Triplet or higher order multiple birth

Where this trial is running

Little Rock, Arkansas and 11 other locations

• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States (Recruiting)
• Jacobs Medical Center — La Jolla, California, United States (Recruiting)
• UF Health Jacksonville — Jacksonville, Florida, United States (Recruiting)
• Advent Health — Orlando, Florida, United States (Recruiting)
• Memorial Hospital of South Bend — South Bend, Indiana, United States (Recruiting)
• Goryeb Children's Hospital — Morristown, New Jersey, United States (Recruiting)
• The Trustees of Columbia University in the City of New York — New York, New York, United States (Recruiting)
• Montefiore Medical Center — The Bronx, New York, United States (Recruiting)
• Maria Farreri Children's Hospital — Valhalla, New York, United States (Recruiting)
• Christus Children's Hospital — San Antonio, Texas, United States (Recruiting)
• University of Virginia School of Medicine — Charlottesville, Virginia, United States (Recruiting)
• WVU Medicine Children's Hospital — Morgantown, West Virginia, United States (Recruiting)

Study contacts

• Study coordinator: Shannon Strom

Conditions Respiratory Distress Syndrome; Pre-term Infants; respiratory distress syndrome; surfactant; pre-term infant