Clinical trial NCT03969992: A Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Respiratory Distress Syndrome in Premature Infant
Keywords: Respiratory Distress Syndrome
BPD
surfactant
Preterm Infant
Lead sponsor: Aerogen Pharma Limited
Sponsor class: INDUSTRY
First posted: 2019-05-31
Enrollment: 261
Sex: ALL
Minimum age (years): 26
Maximum age (years): 31
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Partially Blinded, Randomized, Parallel-Group Dose Ranging
Primary purpose: TREATMENT

Brief 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.

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)

Eligibility criteria: Inclusion Criteria:

1. Parental consent obtained prior to study procedures being performed (pre-natal consent is allowed)
2. 26 0/7 to 30 6/7 weeks of gestational age
3. Weight \<2.0 Kg
4. Respiratory Severity Score (RSS) 1.4-2.0

Exclusion Criteria:

1. Apgar score less than or equal to 5 at five minutes after birth
2. Need for chest compressions or administration of epinephrine or bicarbonate in the delivery room
3. Premature rupture of membranes (PROM) \> 14 days
4. Need for intubation and/or mechanical ventilation prior to enrollment
5. Active pneumothorax requiring chest tube
6. Significant congenital anomaly, chromosomal abnormality
7. Concomitant treatments with inhaled nitric oxide

Primary outcomes:
measure: Incidence of intubation/cannulation and instilled surfactant
timeFrame: 7 days
measure: Percent of patients with respiratory symptoms requiring intervention across groups
timeFrame: 12 months
measure: Percent of patients requiring supplemental oxygen and visits for medical care (Doctor's office/ER/Hospitalization)
timeFrame: 12 months
Secondary outcomes:
measure: Percent of patients requiring repeat surfactant dosing between groups
timeFrame: 7 days
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Berkeley
country: United States
facility: Alta Bates Summit
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Jacksonville
country: United States
facility: University of Florida Health
state: Florida
city: Jacksonville
country: United States
facility: Wolfson Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Advent Health
state: Florida
city: Atlanta
country: United States
facility: Northside Hospital Atlanta
state: Georgia
city: Chicago
country: United States
facility: University of Illinois
state: Illinois
city: South Bend
country: United States
facility: Hospital of South Bend
state: Indiana
city: Louisville
country: United States
facility: University of Kentucky
state: Kentucky
city: Baltimore
country: United States
facility: University of Maryland
state: Maryland
city: Silver Spring
country: United States
facility: Holy Cross Hospital
state: Maryland
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Minneapolis
country: United States
facility: Children's Health Care d/b/a Children's Minnesota
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Children's Hospital
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi
state: Mississippi
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Brooklyn
country: United States
facility: Maimonides Hospital
state: New York
city: New York
country: United States
facility: Mount Sinai Kravis Children's Hospital
state: New York
city: New York
country: United States
facility: NY Presbyterian Morgan Stanley Children's Hospital-Columbia
state: New York
city: Valhalla
country: United States
facility: West Chester Medical Center-Maria Fareri Children's Hospital
state: New York
city: Charlotte
country: United States
facility: Levine Children's Hospital
state: North Carolina
city: Columbus
country: United States
facility: Grant Hospital
state: Ohio
city: Fort Worth
country: United States
facility: Texas Health-Methodist Harris
state: Texas
city: Houston
country: United States
facility: Women's Hospital of Texas
state: Texas
city: San Antonio
country: United States
facility: Children's Hospital of San Antonio (CHofSA)
state: Texas
city: San Antonio
country: United States
facility: Methodist Children's Hospital
state: Texas
city: San Antonio
country: United States
facility: North Central Baptist Hospital
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Halifax
country: Canada
facility: IWK Hospital
state: Nova Scotia
city: Ottawa
country: Canada
facility: The Ottawa Hospital
state: Ontario
city: Toronto
country: Canada
facility: Mt. Sinai Hospital
state: Ontario
city: Québec City
country: Canada
facility: CHU de Québec-Université Laval
state: Quebec
Study officials:
affiliation: Aerogen Pharma Corp
name: Andy Clark, PhD
role: STUDY_DIRECTOR
References: