Safety and Efficacy of APC-0101 in Preterm Infants With Respiratory Distress Syndrome

April 13, 2026 updated by: Aerogen Pharma Limited

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 is a 2-part, prospective, randomized, blinded, sham-controlled, multi-center study comparing preterm subjects with RDS who are treated with APC-0101 and nCPAP/NIV to subjects treated with nCPAP/NIV alone (Sham). In Part 1, subjects will be followed until they reach 40 weeks post-menstrual age (PMA) or are discharged from the NICU, whichever comes first. In Part 2, subjects will undergo post-term follow-up through 24 months corrected age.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

520

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Contact:
    • California
      • La Jolla, California, United States, 92037
    • Florida
      • Jacksonville, Florida, United States, 32209
      • Orlando, Florida, United States, 32803
    • Indiana
      • South Bend, Indiana, United States, 46601
    • New Jersey
      • Morristown, New Jersey, United States, 07960
    • New York
      • New York, New York, United States, 10032
        • Recruiting
        • The Trustees of Columbia University in the City of New York
        • Contact:
      • The Bronx, New York, United States, 10461
      • Valhalla, New York, United States, 10595
        • Recruiting
        • Maria Farreri Children's Hospital
        • Contact:
    • Texas
      • San Antonio, Texas, United States, 78207
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia School Of Medicine
        • Contact:
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • Recruiting
        • WVU Medicine Children's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Sham Comparator: Control Group
Other: Control
Subjects in the Control group will be changed from their current nCPAP/NIV interface to the APC-0101 nCPAP/NIV interface but an empty vial will be used instead of APC-0101.
Experimental: APC-0101 Group
Combination product: APC-0101 (SF-RI 1 surfactant for inhalation combined with a dedicated delivery system)
Subjects in the APC-0101 group will be changed from their current nCPAP/NIV interface to the APC-0101 nCPAP/NIV interface and APC-0101 treatment will be started.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Failure Criteria
Time Frame: First 7 days of life
Number (%) of subjects who meet the predetermined criteria for failure of nCPAP/NIV
First 7 days of life

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Instilled Bolus Surfactant
Time Frame: First 72 hours of life
The number (%) of subjects in each group who receive instilled bolus surfactant.
First 72 hours of life
Multiple Bolus Surfactant Doses
Time Frame: First 72 hours of life
The number (%) of subjects in each group who receive multiple doses of bolus surfactant.
First 72 hours of life
Total Number of Bolus Surfactant Doses
Time Frame: First 72 hours of life
The total number of bolus doses of surfactant received per subject in each group.
First 72 hours of life

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Aerogen Pharma Limited

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 24, 2025

Primary Completion (Estimated)

May 1, 2027

Study Completion (Estimated)

May 1, 2029

Study Registration Dates

First Submitted

January 10, 2025

First Submitted That Met QC Criteria

January 10, 2025

First Posted (Actual)

January 15, 2025

Study Record Updates

Last Update Posted (Actual)

April 14, 2026

Last Update Submitted That Met QC Criteria

April 13, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APC-AF-CLN-003

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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