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Trial registered on ANZCTR
Registration number
ACTRN12617001458325
Ethics application status
Approved
Date submitted
14/09/2017
Date registered
16/10/2017
Date last updated
5/04/2019
Date data sharing statement initially provided
5/04/2019
Type of registration
Prospectively registered
Titles & IDs
Public title
A pilot study in premature infants who are born at less than 29 weeks to determine if surfactant given by aerosol is safe and well-tolerated.
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Scientific title
An Open-Label Study of the Safety and Tolerability of AeroFactTM (Aerosolized Alveofact®) in Preterm Infants on nCPAP at Risk for Worsening Respiratory Distress Syndrome.
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Secondary ID [1]
292574
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None
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Respiratory Distress Syndrome
304241
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Condition category
Condition code
Respiratory
303593
303593
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0
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Other respiratory disorders / diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
AeroFact is an investigational drug/device combination product consisting of a naturally-derived surfactant (Aerosolized Alveofact®) of bovine origin administered via inhalation at a dosage of 108 mg/kg, with up to 4 doses administered over the first 72 hours of life.
The AeroFact drug delivery system is designed to accurately and precisely administer aerosolized surfactant to the lungs of preterm infants who require supplemental oxygen and nasal continuous positive airway pressure (nCPAP) support via mechanical ventilation. The AeroFact drug delivery system consists of three commercially-available products, (1) Aerogen Solo/NIVO Nebulizer, (2) Aerogen Pro-X Controller, and (3) Sensirion Gas Flow Meter/Cable, which are connected to an investigational Aerogen Pharma Controller and investigational nCPAP prong interface.
In Part I of the study, infants will receive a single aerosol dose of the study medication. Following initial aerosol dose to the lung, infants will continue on nCPAP.
In Part II of the study, infants will receive an initial aerosol dose of the study medication. They will continue on nCPAP following the initial dose. Re-dosing of the study medication will occur if the Respiratory Severity Score to maintain oxygen saturation between 90 and 95% reaches 1.5 and at least six hours has elapsed since the previous dose. Up to three additional doses of study medication within 72 hours will be allowed.
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Intervention code [1]
298777
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Treatment: Drugs
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Intervention code [2]
298994
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Treatment: Devices
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Comparator / control treatment
No control group
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Control group
Uncontrolled
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Outcomes
Primary outcome [1]
302946
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Changes from baseline in oxygenation indices. This will be determined by measuring FiO2, oxygen saturation, and respiratory severity score.
FiO2 will be measured by the concentration of oxygen that is inhaled by the patient.
Oxygen saturation will be measured by pulse oximetry.
Respiratory Severity Score will be measured by multiplying the Continuous Positive Airway Pressure (CPAP) and FiO2.
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Assessment method [1]
302946
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Timepoint [1]
302946
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Oxygenation and respiratory parameters will be obtained prior to dosing, during dosing, and up to 72 hours post-dosing.
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Primary outcome [2]
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Changes from baseline in vital signs. This will be determined by measurement of blood pressure, heart rate and respiration rate, comparing prior to dosing to 24 hours post dosing.
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Assessment method [2]
303470
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Timepoint [2]
303470
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Vital signs will be obtained prior to dosing, during dosing, and up to 24 hours post-dosing.
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Primary outcome [3]
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Monitoring of adverse events during treatment and post treatment. Known adverse events of surfactant include oxygen desaturation, bradycardia, and apnea.
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Assessment method [3]
303471
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Timepoint [3]
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Adverse events will be monitored throughout the treatment period up until the time of discharge from the Neonatal Intensive Care Unit (NICU).
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Secondary outcome [1]
337531
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The change in fraction of inspired oxygen (FiO2) as determined by the levels and duration of oxygen.
FiO2 will be measured by recording the concentration of oxygen that is inhaled by the patient, over time.
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Assessment method [1]
337531
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Timepoint [1]
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Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
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Secondary outcome [2]
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Changes in the requirements of nasal continuous positive airway pressure (nCPAP) as determined by pressure levels and duration of nCPAP.
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Assessment method [2]
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Timepoint [2]
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Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
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Secondary outcome [3]
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Changes to the need for surfactant rescue therapy as determined by number of instillations of surfactant administered compared to control group.
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Assessment method [3]
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Timepoint [3]
339027
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Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
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Secondary outcome [4]
339028
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Changes to the need for invasive respiratory support as determined by duration of mechanical ventilation
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Assessment method [4]
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Timepoint [4]
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Need for invasive respiratory support; Timepoint – monitored prior to dosing, during dosing, and up to 72 hours post-dosing
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Secondary outcome [5]
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Changes to the need for endotracheal intubation as determined by the number of intubations, compared to control group.
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Assessment method [5]
339029
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Timepoint [5]
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Need for endotracheal intubation; Timepoint - monitored prior to dosing, during dosing, and up to 72 hours post-dosing
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Secondary outcome [6]
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Changes in bronchopulmonary dysplasia outcomes as determined by survival without BPD at 36 weeks postmenstrual age.
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Assessment method [6]
339030
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Timepoint [6]
339030
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Monitored at 36 weeks PMA
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Eligibility
Key inclusion criteria
Preterm infants born at 26 0/7 to 29 6/7 weeks gestational age, with weight appropriate for age, who are on nCPAP, and a FiO2 less than 0.30.
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Minimum age
0
Hours
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Maximum age
2
Hours
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Preterm infants who required mechanical ventilation and prior instillation of surfactant; significant congenital anomaly; other diseases that might interfere with cardiopulmonary functions; known or suspected chromosomal abnormality; concomitant treatment with inhaled nitric oxide.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
Safety
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Statistical methods / analysis
Descriptive summary statistics include the number of preterm infants, mean, median,
percent coefficient of variation (%CV) (if required), standard deviation, and
minimum and maximum values (quantitative variables) or the number of preterm
infants and percentages by category (qualitative variables). Changes will be
assessed as the difference between baseline measurements and the appropriate postdose
measurements unless otherwise specified.
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Recruitment
Recruitment status
Completed
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Date of first participant enrolment
Anticipated
18/10/2017
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Actual
3/11/2017
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Date of last participant enrolment
Anticipated
31/05/2018
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Actual
1/08/2018
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Date of last data collection
Anticipated
29/06/2018
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Actual
24/10/2018
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Sample size
Target
30
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Accrual to date
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Final
30
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Recruitment in Australia
Recruitment state(s)
NSW,QLD
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Recruitment hospital [1]
8906
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Royal Hospital for Women - Randwick
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Recruitment hospital [2]
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Mater Children's Hospital - South Brisbane
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Recruitment postcode(s) [1]
17152
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2031 - Randwick
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Recruitment postcode(s) [2]
17153
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4101 - South Brisbane
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Funding & Sponsors
Funding source category [1]
297153
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Commercial sector/Industry
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Name [1]
297153
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Aerogen Pharma Limited
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Address [1]
297153
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1660 S Amphlett Boulevard
San Mateo, California 94402
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Country [1]
297153
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United States of America
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Primary sponsor type
Commercial sector/Industry
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Name
CPR Pharma Services
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Address
28 Dalgleish Street
Thebarton, South Australia 5031
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Country
Australia
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Secondary sponsor category [1]
296167
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Commercial sector/Industry
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Name [1]
296167
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Aerogen Pharma Limited
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Address [1]
296167
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1660 S Amphlett Boulevard
San Mateo, California 94402
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Country [1]
296167
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United States of America
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
298320
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South Eastern Sydney Local Health District
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Ethics committee address [1]
298320
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Human Research Ethics Committee Room G71 East Wing, Edmund Blacket Building Prince of Wales Hospital Barker Street Randwick, New South Wales 2031
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Ethics committee country [1]
298320
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Australia
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Date submitted for ethics approval [1]
298320
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22/06/2017
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Approval date [1]
298320
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27/06/2017
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Ethics approval number [1]
298320
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17/076 (HREC/17/POWH/181
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Summary
Brief summary
The aim of this pilot study in premature infants who are on nasal continuous positive airway pressure (nCPAP) is to determine if surfactant given by aerosol is safe, tolerable, and able to reduce the need for mechanical ventilation and use of instilled surfactant administered using an endotracheal tube.
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Trial website
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
76706
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Prof Kei Lui
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Address
76706
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Department of Newborn Care
Royal Hospital for Women
Barker Street
Randwick, New South Wales 2031
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Country
76706
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Australia
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Phone
76706
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+61 2 9382 6190
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Fax
76706
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Email
76706
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Kei.Lui@health.nsw.gov.au
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Contact person for public queries
Name
76707
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Jeanette Asselin MS, RRT-NPS, FAARC
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Address
76707
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Aerogen Pharma
1660 S Amphlett Blvd
Suite 360
San Mateo CA 94402
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Country
76707
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United States of America
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Phone
76707
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+1 510 428 3763
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Fax
76707
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Email
76707
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jasselin@mail.cho.org
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Contact person for scientific queries
Name
76708
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James Fink PhD, RRT, FCCP
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Address
76708
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Aerogen Pharma
1660 S Amphlett Blvd
Suite 360
San Mateo CA 94402
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Country
76708
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United States of America
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Phone
76708
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+1 650 703 7083
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Fax
76708
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Email
76708
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jfink@aerogenpharma.com
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Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Documents added manually
No documents have been uploaded by study researchers.
Documents added automatically
Source
Title
Year of Publication
DOI
Embase
Pulmonary Surfactant: A Unique Biomaterial with Life-saving Therapeutic Applications.
2022
https://dx.doi.org/10.2174/0929867328666210825110421
Embase
Trial of aerosolised surfactant for preterm infants with respiratory distress syndrome.
2022
https://dx.doi.org/10.1136/archdischild-2021-321645
N.B. These documents automatically identified may not have been verified by the study sponsor.
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