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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Respiratory Distress Syndrome in Premature Infant
• Intervention / Treatment: Other: nCPAP; Drug: AeroFact

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 Type: Interventional
• Enrollment (Actual): 261
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Judy Doto, RN, BSN; Phone Number: 484-716-5438; Email: judydoto@aerogenpharma.com
• Study Contact Backup: Name: Jeanette Asselin, MS; Phone Number: 510-813-1177; Email: jasselin@aerogenpharma.com

Study Locations

• Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada
      • IWK Hospital
  • Ontario
    • Ottawa, Ontario, Canada
      • The Ottawa Hospital
    • Toronto, Ontario, Canada
      • Mt. Sinai Hospital
  • Quebec
    • Québec City, Quebec, Canada
      • CHU de Quebec-Universite Laval
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences
  • California
    • Berkeley, California, United States, 94705
      • Alta Bates Summit
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Florida
    • Jacksonville, Florida, United States, 32209
      • Wolfson Children's Hospital
    • Jacksonville, Florida, United States, 32209
      • University of Florida Health
    • Orlando, Florida, United States, 32803
      • Advent Health
  • Georgia
    • Atlanta, Georgia, United States, 30042
      • Northside Hospital Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60612
      • University of Illinois
  • Indiana
    • South Bend, Indiana, United States, 46601
      • Hospital of South Bend
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Kentucky
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland
    • Silver Spring, Maryland, United States, 20910
      • Holy Cross Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Beth Israel Deaconess Medical Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • University of Minnesota Masonic Children's Hospital
    • Minneapolis, Minnesota, United States, 55404
      • Children's Health Care d/b/a Children's Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • University of Mississippi
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth Hitchcock Medical Center
  • New York
    • Brooklyn, New York, United States, 11219
      • Maimonides Hospital
    • New York, New York, United States, 10029
      • Mount Sinai Kravis Children's Hospital
    • New York, New York, United States, 10032
      • NY Presbyterian Morgan Stanley Children's Hospital-Columbia
    • Valhalla, New York, United States, 10595
      • West Chester Medical Center-Maria Fareri Children's Hospital
  • North Carolina
    • Charlotte, North Carolina, United States, 28203
      • Levine Children's Hospital
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Grant Hospital
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Texas Health-Methodist Harris
    • Houston, Texas, United States, 77054
      • Women's Hospital of Texas
    • San Antonio, Texas, United States, 78258
      • North Central Baptist Hospital
    • San Antonio, Texas, United States, 78229
      • Methodist Children's Hospital
    • San Antonio, Texas, United States, 78207
      • Children's Hospital of San Antonio (CHofSA)
  • Virginia
    • Charlottesville, Virginia, United States, 22947
      • University of Virginia
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 months to 8 months (Child)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Aerogen Pharma Limited
• Investigators: Study Director: Andy Clark, PhD, Aerogen Pharma Corp

Study record dates

Study Major Dates

• Study Start (Actual): March 4, 2020
• Primary Completion (Actual): August 4, 2023
• Study Completion (Estimated): August 31, 2024

Study Registration Dates

• First Submitted: April 5, 2019
• First Submitted That Met QC Criteria: May 30, 2019
• First Posted (Actual): May 31, 2019

Study Record Updates

• Last Update Posted (Actual): January 30, 2024
• Last Update Submitted That Met QC Criteria: January 29, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: BPD; surfactant; Respiratory Distress Syndrome; Preterm Infant
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Disease; Infant, Newborn, Diseases; Infant, Premature, Diseases; Syndrome; Respiratory Distress Syndrome; Respiratory Distress Syndrome, Newborn
• Other Study ID Numbers: APC-AF-CLN-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No