Respiratory Distress Syndrome in Premature InfantPhase 2NCT03969992
A Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact
A Phase 2 study testing AeroFact and nCPAP in people with Respiratory Distress Syndrome in Premature Infant, run by Aerogen Pharma Limited at 41 sites in 2 countries.
Trial summary
The purpose of this two-part Phase 2 study is to assess the safety, tolerability and efficacy of aerosolized SF-RI 1 (AeroFact) when delivered via nCPAP at two different doses. Read more about this study
- Who is it for?
- Ages 26 to 31 weeks
- Conditions the study is for
- Respiratory Distress Syndrome in Premature Infant
- What is being tested?
- AeroFact and nCPAP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 261 participants
- Where
- 41 sitesin 2 countries
- Study dates
- 4 Mar 2020to 5 Aug 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 26 to 31 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parental consent obtained prior to study procedures being performed (pre-natal consent is allowed)
- 26 0/7 to 30 6/7 weeks of gestational age
- Weight <2.0 Kg
- Respiratory Severity Score (RSS) 1.4-2.0
Who cannot join
- Apgar score less than or equal to 5 at five minutes after birth
- Need for chest compressions or administration of epinephrine or bicarbonate in the delivery room
- Premature rupture of membranes (PROM) > 14 days
- Need for intubation and/or mechanical ventilation prior to enrollment
- Active pneumothorax requiring chest tube
- Significant congenital anomaly, chromosomal abnormality
- Concomitant treatments with inhaled nitric oxide
Trial locations and contacts
41 sites in 2 countries.
41 sites in 2 countries
United States37 sites
Children's and Women's (C & W) Mott Hospital
Ann Arbor, Michigan, 48109
Northside Hospital Atlanta
Atlanta, Georgia, 30042
University of Colorado
Aurora, Colorado, 80045
University of Maryland
Baltimore, Maryland, 21201
Alta Bates Summit
Berkeley, California, 94705
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Maimonides Hospital
Brooklyn, New York, 11219
Levine Children's Hospital
Charlotte, North Carolina, 28203
University of Virginia
Charlottesville, Virginia, 22947
University of Illinois
Chicago, Illinois, 60612
Grant Hospital
Columbus, Ohio, 43205
Texas Health-Methodist Harris
Fort Worth, Texas, 76104
Women's Hospital of Texas
Houston, Texas, 77054
University of Mississippi
Jackson, Mississippi, 39216
University of Florida Health
Jacksonville, Florida, 32209
Wolfson Children's Hospital
Jacksonville, Florida, 32209
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
University of Louisville
Louisville, Kentucky, 40202
South Miami Hospital
Miami, Florida, 33146
Children's Health Care d/b/a Children's Minnesota
Minneapolis, Minnesota, 55404
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Mount Sinai Kravis Children's Hospital
New York, New York, 10029
Columbia University Medical Center/New York Presbyterian Hospital
New York, New York, 10032
NY Presbyterian Morgan Stanley Children's Hospital-Columbia
New York, New York, 10032
Children's Hospital Orange County
Orange, California, 92868
Advent Health
Orlando, Florida, 32803
Winnie Palmer Hospital
Orlando, Florida, 32806
Barbara Bush Medical Center
Portland, Maine, 04102
Mayo Clinic
Rochester, Minnesota, 55905
Children's Hospital of San Antonio (CHofSA)
San Antonio, Texas, 78207
Methodist Children's Hospital
San Antonio, Texas, 78229
North Central Baptist Hospital
San Antonio, Texas, 78258
University of Washington
Seattle, Washington, 98195
Holy Cross Hospital
Silver Spring, Maryland, 20910
Hospital of South Bend
South Bend, Indiana, 46601
West Chester Medical Center-Maria Fareri Children's Hospital
Valhalla, New York, 10595
Canada4 sites
IWK Hospital
Halifax, Nova Scotia
The Ottawa Hospital
Ottawa, Ontario
CHU de Québec-Université Laval
Québec, Quebec
Mt. Sinai Hospital
Toronto, Ontario
About this study
Official title: A Partially Blinded, Randomized, Controlled, Parallel-Group, Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact in Preterm Infants at Risk of Worsening Respiratory Distress Syndrome
The purpose of this two-part Phase 2 study is to assess the safety, tolerability and efficacy of aerosolized SF-RI 1 (AeroFact) when delivered via nCPAP at two different doses.
Read the detailed description
Part I Primary Objective To determine an optimal dose of AeroFact administered by nasal continuous positive airway pressure (nCPAP) versus stand of care in reducing the rate of intubation/cannulation and bolus surfactant instillation in the first 7 days after birth.
Part II Primary Objective To evaluate pulmonary outcomes and respiratory utilization at 3, 6, 9, and 12 months post-menstrual age (PMA)
- Being tested
- AeroFact, nCPAP
- Keywords
- Respiratory Distress Syndrome, BPD, surfactant, Preterm Infant
- Medical subject headings
- Respiratory Distress Syndrome, Premature Birth
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AeroFactDrug
Also called SF-RI 1
Aerosolized SF-RI 1
nCPAPOther
nCPAP (nasal continuous positive airway pressure) alone
| Study group | Role | What they receive |
|---|---|---|
| nCPAP alone | Other | Standard of Care (nasal continuous positive airway pressure-nCPAP) with instilled bolus surfactant when medically necessary Receives: Other: nCPAP |
| Drug: Low Dose AeroFact | Experimental | AeroFact-low dose SF-RI 1 Receives: Drug: AeroFact |
| Drug: High Dose AeroFact | Experimental | AeroFact-high dose SF-RI 1 Receives: Drug: AeroFact |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intubation/Cannulation and Instilled Surfactant
Number of patients who require intubation/cannulation with bolus surfactant instillation
Measured: First 7 days of life
Secondary outcomes (5)
Time to First Intubation/Cannulation and Bolus Surfactant Instillation
Time from randomization to intubation/cannulation and bolus surfactant instillation in the first 7 days of life
Measured: First 7 days of life
Proportion of Infants Who Received Multiple Doses of Bolus Surfactant
Measured: First 7 days of life
Number of Days on Invasive Mechanical Ventilation
Total number of days each participant required support with invasive mechanical ventilation
Measured: Assessed daily from birth to 40 weeks post-menstrual age or discharge
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 36 Weeks Post-menstrual Age (PMA)
Total number of patients per treatment group who were alive and did not experience BPD at 36 weeks PMA
Measured: Birth to 36 weeks post-menstrual age
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 40 Weeks Post-menstrual Age (PMA)
Total number of patients per treatment group who were alive and did not experience BPD at 40 weeks PMA
Measured: Birth to 40 weeks post-menstrual age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2019
- Study start4 Mar 2020
- Primary completion4 Aug 2023
- Study completion5 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Respiratory Distress Syndrome in Premature InfantStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Partially Blinded, Randomized, Parallel-Group Dose Ranging
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 261 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aerogen Pharma Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Durand, MD, Study director, Aerogen Pharma Corp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03969992
- Sponsor's protocol ID
- APC-AF-CLN-002
- Status last verified
- Aug 2025
- First submitted
- 5 Apr 2019
- Last updated
- 7 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Durand DJ, Hudak ML, Jain A, Courtney SE, Ahmad K, Fink JB, Clark AR, Doto J, Asselin J, Redeker A, O'Riordan T; AeroFact Phase 2b Study Group. 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. J Pediatr. 2026 Jul 13;298:115229. doi: 10.1016/j.jpeds.2026.115229. Online ahead of print.
PubMed 42442657
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Partially Blinded, Randomized, Controlled, Parallel-Group, Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact in Preterm Infants at Risk of Worsening Respiratory Distress Syndrome
- ClinicalTrials.gov ID
- NCT03969992
- Acronym / sponsor ID
- APC-AF-CLN-002
- Keywords
- Respiratory Distress Syndrome, BPD, surfactant, Preterm Infant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 261 participants (actual)
- Lead sponsor
- Aerogen Pharma LimitedIndustry (pharma, biotech, devices)
- Study start
- 4 Mar 2020 (actual)
- Primary completion
- 4 Aug 2023 (actual)
- Study completion
- 5 Aug 2024 (actual)
- First posted
- 31 May 2019 (actual)
- Results
- Posted 7 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this two-part Phase 2 study is to assess the safety, tolerability and efficacy of aerosolized SF-RI 1 (AeroFact) when delivered via nCPAP at two different doses.
Detailed description
Part I Primary Objective To determine an optimal dose of AeroFact administered by nasal continuous positive airway pressure (nCPAP) versus stand of care in reducing the rate of intubation/cannulation and bolus surfactant instillation in the first 7 days after birth.
Part II Primary Objective To evaluate pulmonary outcomes and respiratory utilization at 3, 6, 9, and 12 months post-menstrual age (PMA)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Partially Blinded, Randomized, Parallel-Group Dose Ranging
| Arm | Type | Receives |
|---|---|---|
| nCPAP alone | Other | Other: nCPAPStandard of Care (nasal continuous positive airway pressure-nCPAP) with instilled bolus surfactant when medically necessary |
| Drug: Low Dose AeroFact | Experimental | Drug: AeroFactAeroFact-low dose SF-RI 1 |
| Drug: High Dose AeroFact | Experimental | Drug: AeroFactAeroFact-high dose SF-RI 1 |
Who may qualify
- Ages
- 26 to 31 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Parental consent obtained prior to study procedures being performed (pre-natal consent is allowed)
- 26 0/7 to 30 6/7 weeks of gestational age
- Weight <2.0 Kg
- Respiratory Severity Score (RSS) 1.4-2.0
Key exclusion criteria (7 listed)
- Apgar score less than or equal to 5 at five minutes after birth
- Need for chest compressions or administration of epinephrine or bicarbonate in the delivery room
- Premature rupture of membranes (PROM) > 14 days
- Need for intubation and/or mechanical ventilation prior to enrollment
- Active pneumothorax requiring chest tube
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AeroFactAlso: SF-RI 1Aerosolized SF-RI 1 | Drug | Drug: High Dose AeroFact, Drug: Low Dose AeroFact |
| nCPAPnCPAP (nasal continuous positive airway pressure) alone | Other | nCPAP alone |
Primary outcome
Intubation/Cannulation and Instilled Surfactant
Time frame: First 7 days of life
Number of patients who require intubation/cannulation with bolus surfactant instillation
5 secondary outcomes
Time to First Intubation/Cannulation and Bolus Surfactant Instillation
Time frame: First 7 days of life
Proportion of Infants Who Received Multiple Doses of Bolus Surfactant
Time frame: First 7 days of life
Number of Days on Invasive Mechanical Ventilation
Time frame: Assessed daily from birth to 40 weeks post-menstrual age or discharge
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 36 Weeks Post-menstrual Age (PMA)
Time frame: Birth to 36 weeks post-menstrual age
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 40 Weeks Post-menstrual Age (PMA)
Time frame: Birth to 40 weeks post-menstrual age
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42442657Durand DJ, Hudak ML, Jain A, Courtney SE, Ahmad K, Fink JB, Clark AR, Doto J, Asselin J, Redeker A, O'Riordan T; AeroFact Phase 2b Study Group. 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. J Pediatr. 2026 Jul 13;298:115229. doi: 10.1016/j.jpeds.2026.115229. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Durand, MD study director
Aerogen Pharma Corp
41 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AeroFact and nCPAP in people with Respiratory Distress Syndrome in Premature Infant, run by Aerogen Pharma Limited at 41 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Intubation/Cannulation and Instilled Surfactant (time frame: First 7 days of life). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03969992, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2025.