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    Clinical Trial Results:
    A Multicenter, Randomized, Open-Label, Controlled Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Preterm Neonates 26 to 28 Weeks PMA.

    Summary
    EudraCT number
    		 2015-005624-26
    Trial protocol
    		 PL  
    Global end of trial date
    09 Jul 2017

    Results information
    Results version number
    		 v1
    This version publication date
    27 Oct 2022
    First version publication date
    27 Oct 2022
    Other versions
    		 v2
    Summary report(s)
    		 03-CL-1401 CSR Version 1.0 FINAL

    Trial information

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    Trial identification
    Sponsor protocol code
    03-CL-1401
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02528318
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Windtree Therapeutics, Inc.
    Sponsor organisation address
    2600 Kelly Road, Warrington, United States, 18976
    Public contact
    Steven G. Simonson, MD, MS, Windtree Therapeutics, Inc., +1 (215) 488-9300, psimmons@windtreetx.com
    Scientific contact
    Steven G. Simonson, MD, MS, Windtree Therapeutics, Inc., +1 (215) 488-9300, psimmons@windtreetx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    01 Nov 2017
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Jul 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosolized dose in 4 escalating doses to preterm, neonates 26 to 28 completed weeks PMA with receiving nCPAP for RDS compared with neonates receiving nCPAP alone.
    Protection of trial subjects
    A Safety Review Committee was established to review safety data for this study. SRC was formed to evaluate the degree of risk involved in study subject participation within each dosing group to determine if study continuation in accordance with the current protocol held the potential to institute any undue harm, or threat to the safety and welfare of study subjects. After all active subject within Dosing Group 1 completed Study Day 7, a safety assessment (AEs incl. ADEs, SAEs and additional safety endpoints) was performed. Dosing was allowed to continue to the next higher dosing group (ie. Dosing Group II) immiediately following completion of nerollment within the current dosing group. The SRC provided recommendations following their review of the safety data. This process of continuing to the next higher dosing group and reviewing safety and tolerability was to be continued for the 100 and 150mg TPL/kg group. Subject were followed for safety evaluations until the subect were 36 weeks PMA or were discharged. A final visit ocured at 36 weeks PMA or at time of discharge or withdrawal for all subjects.
    Background therapy
    		 Standard of care (nCPAP alone)
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Oct 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 5
    Country: Number of subjects enrolled
    Canada: 16
    Country: Number of subjects enrolled
    Chile: 1
    Country: Number of subjects enrolled
    United States: 26
    Worldwide total number of subjects
    48
    EEA total number of subjects
    5
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    48
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 1.Signed ICF from legally representative 2.Gestational age 26 to 28 weeks PMA 3. Implementation of controlled nCPAP within 90min after birth 4.Spontaneous breathing 5.Chest radiograph consistent with RDS 6.Within first 20hours after birth, required nCPAP of5-6cm H2O and FiO2 of 0,25-0,50 that was clinically indicated for min. 30mins

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    This study was an open-label study to determine the safety and tolerability of lucinactant for inhalation. No blinding or masking procedures were implemented.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 50mg/kg
    Arm description
    		 Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lucinactant for inhalation
    Investigational medicinal product code
    Other name
    AEROSURF®
    Pharmaceutical forms
    Nebuliser suspension
    Routes of administration
    Inhalation use
    Dosage and administration details
    50 mg TPL/kg administered over 30 minutes in conjunction with nCPAP. 1 Repeat dose of 50 mg TPL/kg administered was allowed if repeat dosing criteria were met

    Arm title
    		 75mg/kg
    Arm description
    		 Lucinactant inhalation 75 mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lucinactant for inhalation
    Investigational medicinal product code
    Other name
    AEROSURF®
    Pharmaceutical forms
    Nebuliser suspension
    Routes of administration
    Inhalation use
    Dosage and administration details
    75 mg TPL/kg administered over 45 minutes in conjunction with nCPAP 1 repeat dose of 75 mg TPL/kg was allowed if repeat dosing criteria are met

    Arm title
    		 100 mg/kg
    Arm description
    		 Lucinactant fot inhalation 100mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dosing will be allowed if repeat criteria are met.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lucinactant for inhalation
    Investigational medicinal product code
    Other name
    AEROSURF®
    Pharmaceutical forms
    Nebuliser suspension
    Routes of administration
    Inhalation use
    Dosage and administration details
    100 mg TPL/kg administered over 60 minutes in conjunction with nCPAP 1 repeat dose ok 100 mg TPL/kg was allowed if repeat dosing criteria are met

    Arm title
    		 nCPAP only
    Arm description
    		 nCPAP therapy alone
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Started
    8
    8
    8
    24
    Completed
    8
    8
    8
    24

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    50mg/kg
    Reporting group description
    		 Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met.

    Reporting group title
    75mg/kg
    Reporting group description
    		 Lucinactant inhalation 75 mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met

    Reporting group title
    100 mg/kg
    Reporting group description
    		 Lucinactant fot inhalation 100mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dosing will be allowed if repeat criteria are met.

    Reporting group title
    nCPAP only
    Reporting group description
    		 nCPAP therapy alone

    Reporting group values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only Total
    Number of subjects
    		 8 8 8 24 48
    Age categorical
    Units: Subjects
        <=18 years
    		 8 8 8 24 48
        Between 18 and 65 years
    		 0 0 0 0 0
        >=65 years
    		 0 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 3 5 5 8 21
        Male
    		 5 3 3 16 27
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 3 1 2 7 13
        Not Hispanic or Latino
    		 5 7 6 17 35
        Unknown or not reported
    		 0 0 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 2 0 1 1 4
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 1 1 2 1 5
        White
    		 3 5 4 20 32
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 2 2 1 2 7
    Region of Enrollment
    Units: Subjects
        Canada
    		 1 3 4 8 16
        United States
    		 7 3 4 12 26
        Poland
    		 0 2 0 3 5
        Chile
    		 0 0 0 1 1
    Gestational age
    Gestational age, based on obstetrician's best estimate
    Units: Weeks
        arithmetic mean (standard deviation)
    		 27.6 ( 0.61 ) 27.2 ( 1.06 ) 27.2 ( 0.84 ) 27.5 ( 0.76 ) -
    Birth Weight
    Units: gram(s)
        arithmetic mean (standard deviation)
    		 1052 ( 174.7 ) 855 ( 84.6 ) 839 ( 224.9 ) 1005 ( 238.8 ) -
    Apgar Score at One Minute
    The Apgar Score measures a baby's health by assessing five characteristics (Appearance, Pulse, Grimace, Activity, and Respiration) . Each of the five characteristics are scored from 0 to 2, and the five scores are summed for the Apgar Score. Thus, the Apgar Score ranges from 0 (worst) to 10 (best), and scores over 7 indicate a baby in good health. Apgar Scores are performed at 1 minute and 5 minutes after birth.
    Units: Scores on a scale
        arithmetic mean (full range (min-max))
    		 5.4 (2 to 9) 5.5 (2 to 8) 6.4 (3 to 8) 5.8 (1 to 9) -
    Apgar Score at Five Minutes
    The Apgar Score measures a baby's health by assessing five characteristics (Appearance, Pulse, Grimace, Activity, and Respiration) . Each of the five characteristics are scored from 0 to 2, and the five scores are summed for the Apgar Score. Thus, the Apgar Score ranges from 0 (worst) to 10 (best), and scores over 7 indicate a baby in good health. Apgar Scores are performed at 1 minute and 5 minutes after birth.
    Units: Scores on a scale
        arithmetic mean (full range (min-max))
    		 7.8 (7 to 9) 6.4 (1 to 9) 8.1 (6 to 9) 7.9 (5 to 9) -

    End points

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    End points reporting groups
    Reporting group title
    50mg/kg
    Reporting group description
    		 Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met.

    Reporting group title
    75mg/kg
    Reporting group description
    		 Lucinactant inhalation 75 mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dose allowed if repeat dosing criteria are met

    Reporting group title
    100 mg/kg
    Reporting group description
    		 Lucinactant fot inhalation 100mg TPL/kg with nCPAP (drug-device combination product) 1 repeat dosing will be allowed if repeat criteria are met.

    Reporting group title
    nCPAP only
    Reporting group description
    		 nCPAP therapy alone

    Primary: Number of Participants With Peri-Dosing Adverse Events - Initial Dose

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    End point title
    		 Number of Participants With Peri-Dosing Adverse Events - Initial Dose [1]
    End point description
    Number of Participants with adverse events that were experienced during the initial study treatment Peri-dosing events are events that occur during study treatment. Since this was an open-label study, no peri-dosing events were recorded for nCPAP alone. Any adverse events that occurred during the corresponding time for nCPAP alone were recorded as adverse events but not peri-dosing events.
    End point type
    Primary
    End point timeframe
    Randomization to 24 Hours Post Randomization
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Because no a priori statistical analyses were conducted in this study, no statistical analysis have been specified
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24 [2]
    Units: Participants
    number (not applicable)
        Bradycardia
    0
    2
    1
    0
        Desaturation
    1
    2
    3
    0
        Gagging/regurgitation
    0
    0
    0
    0
        Apnea
    1
    1
    1
    0
        Pallor
    0
    0
    0
    0
    Notes
    [2] - There was no dosing in this group (no intervention, standard of care)
    No statistical analyses for this end point

    Primary: Number of Participants With Air Leak

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    End point title
    		 Number of Participants With Air Leak [3]
    End point description
    Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema) This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    End point type
    Primary
    End point timeframe
    7 days
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Because no a priori statistical analyses were conducted in this study, no statistical analysis have been specified
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
        number (not applicable)
    3
    0
    2
    4
    No statistical analyses for this end point

    Secondary: Number of Participants With Worsening of Respiratory Status Criteria

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    End point title
    		 Number of Participants With Worsening of Respiratory Status Criteria
    End point description
    Number of participants with worsening in one of 12 respiratory status criteria through 72 hours post randomization (need for additional surfactant therapy, desaturation < 80%, heart rate < 100 bpm, sustained fraction of inspired oxygen (FiO2) > 0.50, arterial carbon dioxide (PCO2) > 65 mmHg, sustained apnea, persistent arterial pH < 7.2, intubation for any reason, nCPAP > 7 cmH2O, initiation of intermittent positive pressure ventilation, death, principal investigator determination of worsening status). Intent-to-treat population. This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    End point type
    Secondary
    End point timeframe
    Randomization to 72 Hours Post Randomization
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
        number (not applicable)
    6
    7
    6
    18
    No statistical analyses for this end point

    Secondary: Bronchopulmonary Dysplasia

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    End point title
    		 Bronchopulmonary Dysplasia
    End point description
    Number of participants with bronchopulmonary dysplasia (BPD) and number of participants alive and without BPD at 36 weeks post-menstrual age (PMA) This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    End point type
    Secondary
    End point timeframe
    Randomization to 36 weeks PMA
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
    number (not applicable)
        BPD
    0
    0
    0
    6
        Alive and without BPD
    7
    7
    8
    18
    No statistical analyses for this end point

    Secondary: Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure

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    End point title
    		 Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure
    End point description
    Participants who required intubation for mechanical ventilation or surfactant administration were defined as having failed nCPAP. This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    End point type
    Secondary
    End point timeframe
    Randomization to 72 Hours Post Randomization
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
        number (not applicable)
    5
    7
    5
    16
    No statistical analyses for this end point

    Secondary: Death

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    End point title
    		 Death
    End point description
    Number of participants who died during the study. This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    End point type
    Secondary
    End point timeframe
    Randomization to 36 weeks PMA
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
        number (not applicable)
    1
    1
    0
    0
    No statistical analyses for this end point

    Secondary: FiO2

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    End point title
    		 FiO2
    End point description
    Observed and change from baseline measurements for fraction of inspired oxygen (FiO2). Values represent the amount (fraction) of oxygen in the air the participant inspires; the values themselves do not have units. The normal amount of oxygen in air ("room air") is 21%, or 0.21. This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg. Safety population
    End point type
    Secondary
    End point timeframe
    Randomization to 72 hours post randomization
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Fraction of oxygen in inspired air
    arithmetic mean (standard deviation)
        Baseline (Randomization)
    0.31 ( 0.058 )
    0.33 ( 0.071 )
    0.37 ( 0.072 )
    0.32 ( 0.061 )
        60 minutes post randomization - observed
    0.31 ( 0.064 )
    0.35 ( 0.063 )
    0.37 ( 0.090 )
    0.34 ( 0.159 )
        60 minutes post randomization - change from baseli
    0.01 ( 0.010 )
    0.02 ( 0.024 )
    0.01 ( 0.044 )
    0.01 ( 0.162 )
        3 hours post randomization - observed
    0.37 ( 0.191 )
    0.37 ( 0.120 )
    0.37 ( 0.107 )
    0.29 ( 0.082 )
        3 hours post randomization - change from baseline
    0.06 ( 0.154 )
    0.00 ( 0.066 )
    0.00 ( 0.047 )
    -0.03 ( 0.082 )
        12 hours post randomization - observed
    0.30 ( 0.114 )
    0.30 ( 0.117 )
    0.30 ( 0.068 )
    0.33 ( 0.181 )
        12 hours post randomization - change from baseline
    -0.02 ( 0.137 )
    -0.07 ( 0.074 )
    -0.08 ( 0.090 )
    0.00 ( 0.172 )
        24 hours post randomization - observed
    0.28 ( 0.093 )
    0.25 ( 0.044 )
    0.28 ( 0.077 )
    0.29 ( 0.143 )
        24 hours post randomization - change from baseline
    -0.03 ( 0.072 )
    -0.09 ( 0.046 )
    -0.08 ( 0.105 )
    -0.04 ( 0.140 )
        48 hours post randomization - observed
    0.28 ( 0.058 )
    0.24 ( 0.038 )
    0.23 ( 0.071 )
    0.31 ( 0.188 )
        48 hours post randomization - change from baseline
    -0.03 ( 0.083 )
    -0.09 ( 0.075 )
    -0.09 ( 0.091 )
    -0.02 ( 0.191 )
        72 hours post randomization - observed
    0.3 ( 0.105 )
    0.23 ( 0.023 )
    0.25 ( 0.050 )
    0.27 ( 0.119 )
        72 hours post randomisation - change from baseline
    -0.02 ( 0.133 )
    -0.10 ( 0.071 )
    -0.12 ( 0.066 )
    -0.05 ( 0.133 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Complications of Prematurity

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    End point title
    		 Number of Participants With Complications of Prematurity
    End point description
    Number of participants with pre-specified common complications of prematurity. The study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150mg/kg group or received a 150mg/kg dose. Safety population.
    End point type
    Secondary
    End point timeframe
    Randomization to 36 weeks PMA
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    8
    8
    8
    24
    Units: Participants
    number (not applicable)
        Subject with any complication
    8
    7
    7
    22
        Acquired sepsis
    2
    2
    2
    5
        Apnea
    8
    5
    6
    19
        Cystic Periventricular Leukomalcia
    0
    0
    0
    1
        Patent Ductus Arteriosus
    3
    3
    4
    10
        Pulmonary Hemorrhage
    1
    0
    1
    1
        Intraventicular Hemorrhage
    0
    1
    3
    4
        Necrotizing Enterocolitis
    1
    1
    1
    1
        Retinopathy of Prematurity
    2
    1
    1
    9
    No statistical analyses for this end point

    Other pre-specified: nCPAP Failure Without Treatment Interruptions

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    End point title
    		 nCPAP Failure Without Treatment Interruptions
    End point description
    Number of subjects requiring mechanical ventilation or surfactant administration (nCPAP failure) but did not have a treatment interruption The study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group.
    End point type
    Other pre-specified
    End point timeframe
    Randomization to 72 Hours Post Randomization
    End point values
    		 50mg/kg 75mg/kg 100 mg/kg nCPAP only
    Number of subjects analysed
    7
    3
    6
    24
    Units: Participants
        number (not applicable)
    4
    3
    3
    16
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From randomization to 36 weeks PMA
    Adverse event reporting additional description
    This dose-escalation study was terminated after completion of the 100 mg/kg group for administrative purposes. No participants were enrolled in the 150 mg/kg group or received 150 mg/kg.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    50 mg/kg
    Reporting group description
    		 Lucinactant for inhalation 50 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. Lucinactant for inhalation: Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

    Reporting group title
    75 mg/kg
    Reporting group description
    		 Lucinactant for inhalation 75 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. Lucinactant for inhalation: Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

    Reporting group title
    100 mg/kg
    Reporting group description
    		 Lucinactant for inhalation 100 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. Lucinactant for inhalation: Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

    Reporting group title
    nCPAP alone
    Reporting group description
    		 nCPAP therapy alone

    Serious adverse events
    		 50 mg/kg 75 mg/kg 100 mg/kg nCPAP alone
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 8 (50.00%)
    3 / 8 (37.50%)
    2 / 8 (25.00%)
    6 / 24 (25.00%)
         number of deaths (all causes)
    1
    1
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Hepatic haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Laryngeal injury
    Additional description: Laryngeal trauma due to intubation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Traumatic liver injury
    Additional description: Liver laceration
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Shock
    Additional description: refractory shock
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac arrest
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardio-respiratory arrest
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Intraventricular haemorrhage neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 8 (25.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Necrotising enterocolitis neonatal
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Neonatal respiratory distress syndrome
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neonatal respiratory failure
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastric perforation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Intestinal perforation
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumoperitoneum
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Apnoea neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neonatal respiratory failure
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumothorax
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    2 / 24 (8.33%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sepsis neonatal
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 50 mg/kg 75 mg/kg 100 mg/kg nCPAP alone
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 8 (100.00%)
    8 / 8 (100.00%)
    8 / 8 (100.00%)
    24 / 24 (100.00%)
    Vascular disorders
    Haemangioma
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Hypertension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Hypotension
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    4 / 24 (16.67%)
         occurrences all number
    1
    2
    0
    7
    Pregnancy, puerperium and perinatal conditions
    Agitation neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Bradycardia neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    5 / 8 (62.50%)
    3 / 8 (37.50%)
    9 / 24 (37.50%)
         occurrences all number
    0
    7
    5
    9
    Fixed bowel loop
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Intraventricular haemorrhage neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    1
    2
    Jaundice neonatal
         subjects affected / exposed
    5 / 8 (62.50%)
    3 / 8 (37.50%)
    1 / 8 (12.50%)
    11 / 24 (45.83%)
         occurrences all number
    5
    3
    1
    12
    Necrotising enterocolitis neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    1
    Neonatal respiratory distress syndrome
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 8 (25.00%)
    2 / 8 (25.00%)
    9 / 24 (37.50%)
         occurrences all number
    1
    2
    3
    12
    Periventricular haemorrhage neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Retinopathy of prematurity
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    9 / 24 (37.50%)
         occurrences all number
    2
    1
    1
    9
    Small for dates baby
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Endotracheal intubation complication
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Face oedema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Hypothermia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Oedema
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    3
    0
    0
    1
    Pain
    alternative assessment type: Systematic
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Apnoea neonatal
         subjects affected / exposed
    7 / 8 (87.50%)
    4 / 8 (50.00%)
    6 / 8 (75.00%)
    19 / 24 (79.17%)
         occurrences all number
    7
    5
    9
    21
    Bronchopulmonary dysplasia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    7 / 24 (29.17%)
         occurrences all number
    0
    0
    0
    8
    Chronic respiratory failure
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypercapnia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    0
    4
    Nasal discomfort
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Nasal inflammation
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    3
    0
    0
    2
    Nasal mucosal ulcer
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Neonatal respiratory failure
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Neonatal tachypnoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    2 / 24 (8.33%)
         occurrences all number
    1
    0
    1
    3
    Pneumothorax
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Pulmonary haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    1
    Pulmonary hypertension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Pulmonary interstitial emphysema syndrome
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    3 / 24 (12.50%)
         occurrences all number
    3
    0
    1
    3
    Pulmonary oedema neonatal
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    0
    2
    Respiratory acidosis
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    2
    0
    0
    2
    Respiratory alkalosis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    2
    Respiratory tract haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Investigations
    Anticonvulsant drug level above therapeutic
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Blood urea increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Cardiac murmur
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    2
    1
    0
    1
    Coagulation time prolonged
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Neutrophil count increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Oxygen saturation decreased
         subjects affected / exposed
    2 / 8 (25.00%)
    4 / 8 (50.00%)
    6 / 8 (75.00%)
    9 / 24 (37.50%)
         occurrences all number
    3
    6
    11
    11
    PO2 increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Reticulocyte count increased
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Blister
    alternative assessment type: Systematic
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Ear abrasion
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infusion site extravasation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Laceration
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin abrasion
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Tracheal injury
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Congenital, familial and genetic disorders
    Atrial septal defect
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Cardiac septal defect
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Hydrocele
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Patent ductus arteriosus
         subjects affected / exposed
    3 / 8 (37.50%)
    3 / 8 (37.50%)
    4 / 8 (50.00%)
    10 / 24 (41.67%)
         occurrences all number
    3
    3
    4
    10
    Cardiac disorders
    Arrhythmia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Tachycardia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Nervous system disorders
    Hypotonia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Lethargy
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Periventricular leukomalacia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Seizure
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    2
    Blood and lymphatic system disorders
    Anaemia neonatal
         subjects affected / exposed
    6 / 8 (75.00%)
    4 / 8 (50.00%)
    2 / 8 (25.00%)
    12 / 24 (50.00%)
         occurrences all number
    6
    6
    2
    16
    Bandaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Coagulopathy
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Leukocystosis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Leukopenia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Neutropenia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Thrombocytopenia
         subjects affected / exposed
    2 / 8 (25.00%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    4 / 24 (16.67%)
         occurrences all number
    2
    2
    0
    4
    Thrombocytosis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Abdominal pain
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Constipation
         subjects affected / exposed
    5 / 8 (62.50%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    2 / 24 (8.33%)
         occurrences all number
    13
    0
    1
    2
    Gastric haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastric hypomotility
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal hypomotility
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Haematemesis
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Inguinal hernia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Salivary hypersecretion
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Umbilical hernia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    0
    3
    Gallbladder disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis diaper
         subjects affected / exposed
    4 / 8 (50.00%)
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    1 / 24 (4.17%)
         occurrences all number
    5
    2
    1
    1
    Rash
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Renal and urinary disorders
    Azotaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Haematuria
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Oliguria
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    1
    0
    2
    Renal failure
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    3
    Endocrine disorders
    Adrenal insufficiency
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Bacterial disease carrier
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 8 (25.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Enterococcal sepsis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Fungal skin infection
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Nosocomial infection
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    1
    0
    3
    Pneumonia klebsiella
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Pneumonia staphylococcal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Sepsis neonatal
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    4 / 24 (16.67%)
         occurrences all number
    1
    1
    1
    4
    Septic shock
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    2
    Urinary tract infection enterococcal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Urosepsis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Electrolyte imbalance
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Feeding intolerance
         subjects affected / exposed
    3 / 8 (37.50%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    1 / 24 (4.17%)
         occurrences all number
    3
    1
    1
    1
    Fluid retention
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    0
    1
    Hypercalcaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    0
    1
    Hyperchloraemia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    1
    0
    0
    3
    Hyperglycaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    1
    2
    0
    5
    Hyperkalaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Hypermagnesaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Hypernatraemia
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    4 / 24 (16.67%)
         occurrences all number
    1
    2
    0
    5
    Hypertriglyceridaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    1
    1
    0
    3
    Hypoalbuminaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    0
    3
    Hypocalcaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Hypochloraemia
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    2
    1
    0
    4
    Hypokalaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    2
    0
    4
    Hypomagnesaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    0
    1
    Hyponatraemia
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    7 / 24 (29.17%)
         occurrences all number
    3
    0
    0
    7
    Hypophosphataemia
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    2
    0
    0
    1
    Hypovolaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Metabolic acidosis
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    5 / 24 (20.83%)
         occurrences all number
    3
    0
    0
    5
    Metabolic alkalosis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Osteopenia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    0
    2
    Vitamin D deficiency
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypoglycaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    0
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Sep 2015
    Rationale for protocol amendment - additional requirement of meeting criteria at treatment initiation added - additional clarification on repeat dose - typo corrections - clarification of "completion of study" - SRC approval not required to advance to next dosing level - repeat dosing criteria changed - concomitant medications - additional clarifications added - supplement to the instruction for ADP returns - clarification of when SRC meeting would be held following a death - clarification of peri-dosing events

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    09 Jul 2017
    The sponsor of study 03-CL-1401, Windtree Therapeutics, Inc. has made a decision to terminate the dose-escalation study early after the completion of enrollment into Dose Group 3 3. It was anticipated 64 subjects would be enrolled into the study in four dose groups; however, only 48 subjects were enrolled (Dose Groups 1,2,3; n=16 per group). The decision to terminate the study early was taken to incorporate learning from this study into a redesigned trial to continue studying neonates in the 26-28 week gestational age. To this end, sponsor planned to study the 26-28 week GA patients in the amendment (Amendment 2) to the 03-CL-1202 study. A preliminary review of the data through Dose Group 3 has shown no safety signal of concern and the overall safety and tolerability profile was generally similar to the control group treated with nCPAP alone. A maximum tolerated dose was not established, the overall objective of the study was met. Early termination of 03-CL-1401 study has no effect on the overall risk-benefit assessment of lucinactant for inhalation. Last subject randomized into the study was on 16 May 2017 and was followed through study completion, which is 36 weeks PMA (9 July 2017)
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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