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
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Aerogen Pharma Limited
- Enrollment
- 261
- Locations
- 41
- Primary Endpoint
- Intubation/Cannulation and Instilled Surfactant
Study Overview
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)
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Masking within active arms
Eligibility Criteria
- Ages
- 26 Weeks to 31 Weeks (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Arms & Interventions
nCPAP alone
Standard of Care (nasal continuous positive airway pressure-nCPAP) with instilled bolus surfactant when medically necessary
Intervention: nCPAP (Other)
Drug: Low Dose AeroFact
AeroFact-low dose SF-RI 1
Intervention: AeroFact (Drug)
Drug: High Dose AeroFact
AeroFact-high dose SF-RI 1
Intervention: AeroFact (Drug)
Outcomes
Primary Outcomes
Intubation/Cannulation and Instilled Surfactant
Time Frame: First 7 days of life
Number of patients who require intubation/cannulation with bolus surfactant instillation
Secondary Outcomes
- Time to First Intubation/Cannulation and Bolus Surfactant Instillation(First 7 days of life)
- Proportion of Infants Who Received Multiple Doses of Bolus Surfactant(First 7 days of life)
- Number of Days on Invasive Mechanical Ventilation(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)(Birth to 36 weeks post-menstrual age)
- Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 40 Weeks Post-menstrual Age (PMA)(Birth to 40 weeks post-menstrual age)