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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.
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)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| nCPAP alone | Other | Standard of Care (nasal continuous positive airway pressure-nCPAP) with instilled bolus surfactant when medically necessary |
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| Drug: Low Dose AeroFact | Experimental | AeroFact-low dose SF-RI 1 |
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| Drug: High Dose AeroFact | Experimental | AeroFact-high dose SF-RI 1 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AeroFact | Drug | Aerosolized SF-RI 1 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Intubation/Cannulation and Instilled Surfactant | Number of patients who require intubation/cannulation with bolus surfactant instillation | First 7 days of life |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | First 7 days of life |
| Proportion of Infants Who Received Multiple Doses of Bolus Surfactant |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David Durand, MD | Aerogen Pharma Corp | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States | ||
| Alta Bates Summit |
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| ID | Title | Description |
|---|---|---|
| FG000 | Control: nCPAP Alone | nCPAP (nasal continuous positive airway pressure) alone |
| FG001 | Drug: Low Dose AeroFact | AeroFact 108 mg/kg/dose |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1: Birth to Discharge/40 Weeks PMA |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 9, 2022 |
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Partially Blinded, Randomized, Parallel-Group Dose Ranging
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Masking within active arms
| nCPAP | Other | nCPAP (nasal continuous positive airway pressure) alone |
|
| First 7 days of life |
| Number of Days on Invasive Mechanical Ventilation | Total number of days each participant required support with 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) | Total number of patients per treatment group who were alive and did not experience BPD at 36 weeks PMA | 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 | Birth to 40 weeks post-menstrual age |
| Berkeley |
| California |
| 94705 |
| United States |
| Children's Hospital Orange County | Orange | California | 92868 | United States |
| University of Colorado | Aurora | Colorado | 80045 | United States |
| University of Florida Health | Jacksonville | Florida | 32209 | United States |
| Wolfson Children's Hospital | Jacksonville | Florida | 32209 | United States |
| South Miami Hospital | Miami | Florida | 33146 | United States |
| Advent Health | Orlando | Florida | 32803 | United States |
| Winnie Palmer Hospital | Orlando | Florida | 32806 | United States |
| Northside Hospital Atlanta | Atlanta | Georgia | 30042 | United States |
| University of Illinois | Chicago | Illinois | 60612 | United States |
| Hospital of South Bend | South Bend | Indiana | 46601 | United States |
| University of Louisville | Louisville | Kentucky | 40202 | United States |
| Barbara Bush Medical Center | Portland | Maine | 04102 | United States |
| University of Maryland | Baltimore | Maryland | 21201 | United States |
| Holy Cross Hospital | Silver Spring | Maryland | 20910 | United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | United States |
| Children's and Women's (C & W) Mott Hospital | Ann Arbor | Michigan | 48109 | United States |
| Children's Health Care d/b/a Children's Minnesota | Minneapolis | Minnesota | 55404 | United States |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55454 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| University of Mississippi | Jackson | Mississippi | 39216 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Maimonides Hospital | Brooklyn | New York | 11219 | United States |
| Mount Sinai Kravis Children's Hospital | New York | New York | 10029 | United States |
| Columbia University Medical Center/New York Presbyterian Hospital | New York | New York | 10032 | United States |
| NY Presbyterian Morgan Stanley Children's Hospital-Columbia | New York | New York | 10032 | United States |
| West Chester Medical Center-Maria Fareri Children's Hospital | Valhalla | New York | 10595 | United States |
| Levine Children's Hospital | Charlotte | North Carolina | 28203 | United States |
| Grant Hospital | Columbus | Ohio | 43205 | United States |
| Texas Health-Methodist Harris | Fort Worth | Texas | 76104 | United States |
| Women's Hospital of Texas | Houston | Texas | 77054 | United States |
| Children's Hospital of San Antonio (CHofSA) | San Antonio | Texas | 78207 | United States |
| Methodist Children's Hospital | San Antonio | Texas | 78229 | United States |
| North Central Baptist Hospital | San Antonio | Texas | 78258 | United States |
| University of Virginia | Charlottesville | Virginia | 22947 | United States |
| University of Washington | Seattle | Washington | 98195 | United States |
| IWK Hospital | Halifax | Nova Scotia | Canada |
| The Ottawa Hospital | Ottawa | Ontario | Canada |
| Mt. Sinai Hospital | Toronto | Ontario | Canada |
| CHU de Québec-Université Laval | Québec | Quebec | Canada |
| FG002 | Drug: High Dose AeroFact | AeroFact 216 mg/kg/dose |
| COMPLETED |
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| NOT COMPLETED |
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| Part 2: Discharge/40 Wks PMA to 12 Mos |
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All randomized patients were included in the ITT and Safety analysis populations, regardless of whether or not they received study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Control: nCPAP Alone | nCPAP (nasal continuous positive airway pressure) alone |
| BG001 | Drug: Low Dose AeroFact | AeroFact-low dose SF-RI 1 |
| BG002 | Drug: High Dose AeroFact | AeroFact-high dose SF-RI 1 |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Gestational age in weeks | Mean | Standard Deviation | weeks gestational age |
| ||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Birth weight | Mean | Standard Deviation | grams |
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| Vaginal delivery | Count of Participants | Participants |
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| Multiple gestation | Count of Participants | Participants |
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| Age at Randomization | Mean | Standard Deviation | hours |
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| FiO2 at Randomization | Fraction of inspired oxygen; Higher scores indicate more supplemental oxygen was needed | Mean | Standard Deviation | Percentage of inspired oxygen |
| ||||||||||||||
| Respiratory Severity Score at Randomization | mean airway pressure [MAP] × FiO2; Higher scores suggest greater breathing difficulty | Mean | Standard Deviation | units on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Intubation/Cannulation and Instilled Surfactant | Number of patients who require intubation/cannulation with bolus surfactant instillation | Per Protocol population | Posted | Count of Participants | Participants | First 7 days of life |
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| Secondary | 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 | Per protocol population | Posted | Median | Full Range | hours | First 7 days of life |
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| Secondary | Proportion of Infants Who Received Multiple Doses of Bolus Surfactant | Per protocol population | Posted | Count of Participants | Participants | First 7 days of life |
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| Secondary | Number of Days on Invasive Mechanical Ventilation | Total number of days each participant required support with invasive mechanical ventilation | Per protocol population | Posted | Mean | Standard Deviation | Days | Assessed daily from birth to 40 weeks post-menstrual age or discharge |
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| Secondary | 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 | Per protocol population | Posted | Count of Participants | Participants | Birth to 36 weeks post-menstrual age |
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| Secondary | 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 | Per protocol population | Posted | Count of Participants | Participants | Birth to 40 weeks post-menstrual age |
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AEs and SAEs were collected from randomization through NICU discharge or 40 weeks post-menstrual age (PMA) in Part 1. Only fatal SAEs were collected for up to 12 months in Part 2.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Control: nCPAP Alone | nCPAP (nasal continuous positive airway pressure) alone | 2 | 92 | 7 | 92 | 43 | 92 |
| EG001 | Drug: Low Dose AeroFact | AeroFact-low dose SF-RI 1 | 3 | 83 | 9 | 83 | 45 | 83 |
| EG002 | Drug: High Dose AeroFact | AeroFact-high dose SF-RI 1 | 2 | 86 | 5 | 86 | 42 | 86 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Necrotizing enterocolitis neonatal | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Gastrointestinal perforation | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Meconium ileus | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Pulmonary hemorrhage neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Cardiac dysfunction | Cardiac disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Cardiac tamponade | Cardiac disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA 22.1 | Non-systematic Assessment |
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| Intraventricular hemorrhage neonatal | Nervous system disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Intraventricular hemorrhage neonatal | Nervous system disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Retinopathy or prematurity | Eye disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Infantile apnea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Neonatal respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Pulmonary hemorrhage neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Patent ductus arteriosus | Congenital, familial and genetic disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Necrotizing enterocolitis neonatal | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Gastrointestinal perforation | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Inguinal hernia | Gastrointestinal disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA 22.1 | Non-systematic Assessment |
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| Bradycardia neonatal | Cardiac disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Patent ductus arteriosus repair | Surgical and medical procedures | MedDRA 22.1 | Non-systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA 22.1 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| David Durand, MD | Aerogen Pharma | 510-928-2111 | ddurand@aerogenpharma.com |
| Jan 24, 2025 |
| Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D012128 | Respiratory Distress Syndrome |
| D047928 | Premature Birth |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012120 | Respiration Disorders |
| D007752 | Obstetric Labor, Premature |
| D007744 | Obstetric Labor Complications |
| D011248 | Pregnancy Complications |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
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| ID | Term |
|---|---|
| C078272 | SF-RI 1, bovine surfactant preparation |
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| Withdrawal by Subject |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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