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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
Date results information 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.
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.
Secondary ID [1] 292574 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Respiratory Distress Syndrome 304241 0
Condition category
Condition code
Respiratory 303593 303593 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 298777 0
Treatment: Drugs
Intervention code [2] 298994 0
Treatment: Devices
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 302946 0
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.
Timepoint [1] 302946 0
Oxygenation and respiratory parameters will be obtained prior to dosing, during dosing, and up to 72 hours post-dosing.
Primary outcome [2] 303470 0
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.
Timepoint [2] 303470 0
Vital signs will be obtained prior to dosing, during dosing, and up to 24 hours post-dosing.
Primary outcome [3] 303471 0
Monitoring of adverse events during treatment and post treatment. Known adverse events of surfactant include oxygen desaturation, bradycardia, and apnea.
Timepoint [3] 303471 0
Adverse events will be monitored throughout the treatment period up until the time of discharge from the Neonatal Intensive Care Unit (NICU).
Secondary outcome [1] 337531 0
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.
Timepoint [1] 337531 0
Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
Secondary outcome [2] 337532 0
Changes in the requirements of nasal continuous positive airway pressure (nCPAP) as determined by pressure levels and duration of nCPAP.
Timepoint [2] 337532 0
Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
Secondary outcome [3] 339027 0
Changes to the need for surfactant rescue therapy as determined by number of instillations of surfactant administered compared to control group.
Timepoint [3] 339027 0
Monitored prior to dosing, during dosing, and up to 72 hours post-dosing
Secondary outcome [4] 339028 0
Changes to the need for invasive respiratory support as determined by duration of mechanical ventilation
Timepoint [4] 339028 0
Need for invasive respiratory support; Timepoint – monitored prior to dosing, during dosing, and up to 72 hours post-dosing
Secondary outcome [5] 339029 0
Changes to the need for endotracheal intubation as determined by the number of intubations, compared to control group.
Timepoint [5] 339029 0
Need for endotracheal intubation; Timepoint - monitored prior to dosing, during dosing, and up to 72 hours post-dosing
Secondary outcome [6] 339030 0
Changes in bronchopulmonary dysplasia outcomes as determined by survival without BPD at 36 weeks postmenstrual age.
Timepoint [6] 339030 0
Monitored at 36 weeks PMA

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.
Minimum age
0 Hours
Maximum age
2 Hours
Gender
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Safety
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.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 8906 0
Royal Hospital for Women - Randwick
Recruitment hospital [2] 8907 0
Mater Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 17152 0
2031 - Randwick
Recruitment postcode(s) [2] 17153 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 297153 0
Commercial sector/Industry
Name [1] 297153 0
Aerogen Pharma Limited
Address [1] 297153 0
1660 S Amphlett Boulevard
San Mateo, California 94402
Country [1] 297153 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
CPR Pharma Services
Address
28 Dalgleish Street
Thebarton, South Australia 5031
Country
Australia
Secondary sponsor category [1] 296167 0
Commercial sector/Industry
Name [1] 296167 0
Aerogen Pharma Limited
Address [1] 296167 0
1660 S Amphlett Boulevard
San Mateo, California 94402
Country [1] 296167 0
United States of America

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 298320 0
South Eastern Sydney Local Health District
Ethics committee address [1] 298320 0
Human Research Ethics Committee
Room G71 East Wing, Edmund Blacket Building
Prince of Wales Hospital
Barker Street
Randwick, New South Wales 2031
Ethics committee country [1] 298320 0
Australia
Date submitted for ethics approval [1] 298320 0
22/06/2017
Approval date [1] 298320 0
27/06/2017
Ethics approval number [1] 298320 0
17/076 (HREC/17/POWH/181

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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 76706 0
Prof Kei Lui
Address 76706 0
Department of Newborn Care
Royal Hospital for Women
Barker Street
Randwick, New South Wales 2031
Country 76706 0
Australia
Phone 76706 0
+61 2 9382 6190
Fax 76706 0
Email 76706 0
Kei.Lui@health.nsw.gov.au
Contact person for public queries
Name 76707 0
Ms Jeanette Asselin MS, RRT-NPS, FAARC
Address 76707 0
Aerogen Pharma
1660 S Amphlett Blvd
Suite 360
San Mateo CA 94402
Country 76707 0
United States of America
Phone 76707 0
+1 510 428 3763
Fax 76707 0
Email 76707 0
jasselin@mail.cho.org
Contact person for scientific queries
Name 76708 0
Dr James Fink PhD, RRT, FCCP
Address 76708 0
Aerogen Pharma
1660 S Amphlett Blvd
Suite 360
San Mateo CA 94402
Country 76708 0
United States of America
Phone 76708 0
+1 650 703 7083
Fax 76708 0
Email 76708 0
jfink@aerogenpharma.com

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
No
Other publications
Have study results been made publicly available in another format?
No
Results – basic reporting
Results – plain English summary