A Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact

Study Overview
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Brief Summary
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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
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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)

Official Title
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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

Conditions
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Respiratory Distress Syndrome in Premature Infant

Intervention / Treatment
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* Drug: AeroFact
* Other: nCPAP


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 26 Weeks
Maximum Age: 31 Weeks

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Partially Blinded, Randomized, Parallel-Group Dose Ranging
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: nCPAP alone<br>Standard of Care (nasal continuous positive airway pressure-nCPAP) with instilled bolus surfactant when medically necessary | Other: nCPAP<br>* nCPAP (nasal continuous positive airway pressure) alone<br>|
| Experimental: Drug: Low Dose AeroFact<br>AeroFact-low dose SF-RI 1 | Drug: AeroFact<br>* Aerosolized SF-RI 1<br>* Other names: SF-RI 1;|
| Experimental: Drug: High Dose AeroFact<br>AeroFact-high dose SF-RI 1 | Drug: AeroFact<br>* Aerosolized SF-RI 1<br>* Other names: SF-RI 1;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of intubation/cannulation and instilled surfactant | Number of patients who require intubation/cannulation with bolus surfactant instillation | 7 days | 
| Percent of patients with respiratory symptoms requiring intervention across groups | Comparison of respiratory symptoms (Asthma/URI) and respiratory medications between groups | 12 months | 
| Percent of patients requiring supplemental oxygen and visits for medical care (Doctor's office/ER/Hospitalization) | Assess need for supplemental oxygen and other respiratory support and visits to physician office/ER/hospitalizations between groups | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent of patients requiring repeat surfactant dosing between groups | Assess the need for more than one dose of surfactant between groups | 7 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Respiratory Distress Syndrome, BPD, surfactant, Preterm Infant