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    Clinical Trial Results:
    A FIRST IN HUMAN CLINICAL STUDY ON THE SAFETY AND TOLERABILITY OF TWO ESCALATING SINGLE DOSES OF CHF 5633 (SYNTHETIC SURFACTANT) IN PRETERM NEONATES WITH RESPIRATORY DISTRESS SYNDROME

    Summary
    EudraCT number
    		 2011-001331-22
    Trial protocol
    		 DE   GB   CZ  
    Global end of trial date
    23 Jan 2015

    Results information
    Results version number
    		 v2(current)
    This version publication date
    10 Apr 2019
    First version publication date
    27 Jul 2016
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    To report the results of the stand-alone clinical assessment performed to check the neurological status of the preterm born children at 24 months (±3 months) corrected age (CA), previously treated with CHF5633 within 48 hours of life for Respiratory Distress Syndrome (RDS) in the above clinical study.
    Summary report(s)
    		 CSR ADDENDUM FIH CHF5633 - RESULTS FOLLOW-UP 24MONTHS

    Trial information

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    Trial identification
    Sponsor protocol code
    CCD-1011-PR-0059
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01651637
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Chiesi Farmaceutici S.p.A.
    Sponsor organisation address
    Via Palermo, 26/A, Parma, Italy, 43122
    Public contact
    Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., ClinicalTrials_info@chiesi.com
    Scientific contact
    Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., ClinicalTrials_info@chiesi.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
    23 Jan 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jan 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jan 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the safety and tolerability of intratracheal administration of two different single doses of CHF 5633 in preterm neonates with respiratory distress syndrome (RDS) in terms of adverse events (AEs), adverse drug reactions (ADRs), haematology and blood chemistry values, and incidence of major neonatal morbidities and mortality.
    Protection of trial subjects
    The study was conducted in accordance with the Declaration of Helsinki, Good Clinical Practice (GCP) guidelines and local law requirements. Other than routine care, no specific measures for protection of trial subjects were implemented.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    03 Oct 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 21
    Country: Number of subjects enrolled
    Czech Republic: 11
    Country: Number of subjects enrolled
    Germany: 8
    Worldwide total number of subjects
    40
    EEA total number of subjects
    40
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    40
    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
    		 In both study cohorts, neonates were recruited/enrolled in a stepwise fashion, based on a safety evaluation following the administration of CHF 5633. If no major safety concerns were raised for each member of the first 4-neonate group and for the group overall, recruitment proceeded to the next 4-neonate group until 20 neonates were enrolled.

    Pre-assignment
    Screening details
    		 Seventy-five neonates were screened for the FIH study; 35 failed the screening, based on inclusion/exclusion criteria (n=33), investigator decision (n=1) or consent withdrawal (n=1)

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Not applicable; this was an open-label study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort A
    Arm description
    		 Neonates in cohort A received a single dose of CHF 5633 (100 mg/kg birth weight) administered intratracheally within 48 hours from birth.
    Arm type
    		 Experimental

    Investigational medicinal product name
    CHF 5633
    Investigational medicinal product code
    Other name
    SP B analogue (CHF 5736.03) + SP-C analogue (CHF 4902.03) + dipalmitoylphosphatidylcholine + 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol sodium salt
    Pharmaceutical forms
    Endotracheopulmonary instillation, suspension
    Routes of administration
    Intratracheal use
    Dosage and administration details
    CHF 5633 (100 mg/kg birth weight) as a single intratracheal dose within 48 hours from birth.

    Arm title
    		 Cohort B
    Arm description
    		 After completion of treatment in Cohort A neonates in cohort B received a single dose of CHF 5633 (200 mg/kg birth weight) administered intratracheally within 48 hours from birth.
    Arm type
    		 Experimental

    Investigational medicinal product name
    CHF 5633
    Investigational medicinal product code
    Other name
    SP B analogue (CHF 5736.03) + SP-C analogue (CHF 4902.03) + dipalmitoylphosphatidylcholine + 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol sodium salt
    Pharmaceutical forms
    Endotracheopulmonary instillation, suspension
    Routes of administration
    Intratracheal use
    Dosage and administration details
    CHF 5633 (200 mg/kg birth weight) as a single intratracheal dose within 48 hours from birth.

    Number of subjects in period 1
    		Cohort A Cohort B
    Started
    20
    20
    Completed
    20
    19
    Not completed
    0
    1
         Adverse event, serious fatal
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 40 40
    Age categorical
    Units: Subjects
        Newborns (0-27 days)
    		 40 40
    Age continuous
    Age is gestational age.
    Units: weeks
        arithmetic mean (standard deviation)
    		 29.6 ( 1.93 ) -
    Gender categorical
    Units: Subjects
        Female
    		 19 19
        Male
    		 21 21
    Subject analysis sets

    Subject analysis set title
    Cohort A - Safety/Efficacy analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All neonates who received any dose level of IMPCHF5633. This population was used for analysis of safety and efficacy analyses assessments

    Subject analysis set title
    Cohort B - Safety/Efficacy analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All neonates who received any dose level of IMPCHF5633. This population was used for analysis of safety and efficacy analyses assessments

    Subject analysis set title
    Cohort A - Systemic absorption analysis
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All neonates in the safety population with valid systemic absorption measurements. This population was used for analysis of SP-B and SP-C analogues.

    Subject analysis set title
    Cohort B - Systemic absorption analysis
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All neonates in the safety population with valid systemic absorption measurements. This population was used for analysis of SP-B and SP-C analogues.

    Subject analysis sets values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis Cohort A - Systemic absorption analysis Cohort B - Systemic absorption analysis
    Number of subjects
    20
    20
    20
    20
    Age categorical
    Units: Subjects
        Newborns (0-27 days)
    20
    20
    20
    20
    Age continuous
    Age is gestational age.
    Units: weeks
        arithmetic mean (standard deviation)
    29.6 ( 2.04 )
    29.6 ( 1.88 )
    29.6 ( 2.04 )
    29.6 ( 1.88 )
    Gender categorical
    Units: Subjects
        Female
    9
    10
    9
    10
        Male
    11
    10
    11
    10

    End points

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    End points reporting groups
    Reporting group title
    Cohort A
    Reporting group description
    		 Neonates in cohort A received a single dose of CHF 5633 (100 mg/kg birth weight) administered intratracheally within 48 hours from birth.

    Reporting group title
    Cohort B
    Reporting group description
    		 After completion of treatment in Cohort A neonates in cohort B received a single dose of CHF 5633 (200 mg/kg birth weight) administered intratracheally within 48 hours from birth.

    Subject analysis set title
    Cohort A - Safety/Efficacy analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All neonates who received any dose level of IMPCHF5633. This population was used for analysis of safety and efficacy analyses assessments

    Subject analysis set title
    Cohort B - Safety/Efficacy analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All neonates who received any dose level of IMPCHF5633. This population was used for analysis of safety and efficacy analyses assessments

    Subject analysis set title
    Cohort A - Systemic absorption analysis
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All neonates in the safety population with valid systemic absorption measurements. This population was used for analysis of SP-B and SP-C analogues.

    Subject analysis set title
    Cohort B - Systemic absorption analysis
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All neonates in the safety population with valid systemic absorption measurements. This population was used for analysis of SP-B and SP-C analogues.

    Primary: Systolic blood pressure

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    End point title
    		 Systolic blood pressure [1]
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Primary
    End point timeframe
    Observed and change from baseline values of vital signs by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    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: No statistical analys was performed.
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    18
    20
    Units: mmHg
        arithmetic mean (standard deviation)
    3.8 ( 9.05 )
    4.7 ( 7.15 )
    No statistical analyses for this end point

    Primary: Diastolic blood pressure

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    End point title
    		 Diastolic blood pressure [2]
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Primary
    End point timeframe
    Observed and change from baseline values of vital signs by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    Notes
    [2] - 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: No statistical analys was performed.
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    18
    20
    Units: mmHg
        arithmetic mean (standard deviation)
    3.6 ( 8.54 )
    5.8 ( 7.92 )
    No statistical analyses for this end point

    Primary: Heart rate

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    End point title
    		 Heart rate [3]
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Primary
    End point timeframe
    Observed and change from baseline values of vital signs by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    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: No statistical analys was performed.
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    20
    20
    Units: bpm
        arithmetic mean (standard deviation)
    -0.4 ( 22.36 )
    -0.7 ( 11.17 )
    No statistical analyses for this end point

    Secondary: SpO2

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    End point title
    		 SpO2
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here
    End point type
    Secondary
    End point timeframe
    Observed and change from baseline values by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    20
    20
    Units: percent
        arithmetic mean (standard deviation)
    2.3 ( 3.93 )
    2.6 ( 2.91 )
    No statistical analyses for this end point

    Secondary: FiO2

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    End point title
    		 FiO2
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Secondary
    End point timeframe
    Observed and change from baseline values by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    16
    19
    Units: percent
        arithmetic mean (standard deviation)
    -23.4 ( 19.09 )
    -29.4 ( 13.68 )
    No statistical analyses for this end point

    Secondary: FiO2 AUC0-6

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    End point title
    		 FiO2 AUC0-6
    End point description
    Only data for the two cohorts separately are reported here.
    End point type
    Secondary
    End point timeframe
    FiO2 AUC0-6 was assessed for the two cohorts and for the overall population based on the measurements performed at 30 min and 1, 3 and 6 hours following CHF 5633 administration.
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    19
    20
    Units: digit
        arithmetic mean (standard deviation)
    160.3 ( 29.94 )
    159 ( 39.38 )
    No statistical analyses for this end point

    Secondary: FiO2 AUC0-12

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    End point title
    		 FiO2 AUC0-12
    End point description
    Only data for the two cohorts separately are reported here.
    End point type
    Secondary
    End point timeframe
    FiO2 AUC0-12 was assessed for the two cohorts and for the overall population based on the measurements performed at 30 min and 1, 3, 6 and 12 hours following CHF 5633 administration.
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    19
    20
    Units: digit
        arithmetic mean (standard deviation)
    306.8 ( 55.34 )
    159 ( 39.38 )
    No statistical analyses for this end point

    Secondary: Mean airway pressure (MAP)

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    End point title
    		 Mean airway pressure (MAP)
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Secondary
    End point timeframe
    Observed and change from baseline values by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    5
    2
    Units: cmH2O
        arithmetic mean (standard deviation)
    -1 ( 1.17 )
    -3.1 ( 4.03 )
    No statistical analyses for this end point

    Secondary: Peak inspiratory pressure (PIP)

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    End point title
    		 Peak inspiratory pressure (PIP)
    End point description
    Only changes from baseline at 24 hrs for cohort A are reported here. Values for Cohort B were not estimable.
    End point type
    Secondary
    End point timeframe
    Observed and change from baseline values by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis
    Number of subjects analysed
    2
    Units: cmH2O
        arithmetic mean (standard deviation)
    -6 ( 0 )
    No statistical analyses for this end point

    Secondary: Positive end-expiratory pressure (PEEP)

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    End point title
    		 Positive end-expiratory pressure (PEEP)
    End point description
    Only changes from baseline at 24 hrs for the two cohorts separately are reported here.
    End point type
    Secondary
    End point timeframe
    Observed and change from baseline values by cohort and overall were assessed following CHF 5633 administration at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    15
    16
    Units: cmH2O
        arithmetic mean (standard deviation)
    -0.3 ( 1.37 )
    0.3 ( 1.32 )
    No statistical analyses for this end point

    Secondary: Duration of invasive ventilation

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    End point title
    		 Duration of invasive ventilation
    End point description
    End point type
    Secondary
    End point timeframe
    Assessments concerning ventilation following CHF 5633 administration were performed at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    16
    14
    Units: days
        arithmetic mean (standard deviation)
    1.5 ( 2.56 )
    1.3 ( 3.04 )
    No statistical analyses for this end point

    Secondary: Duration of non-invasive ventilation

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    End point title
    		 Duration of non-invasive ventilation
    End point description
    End point type
    Secondary
    End point timeframe
    Assessments concerning ventilation following CHF 5633 administration were performed at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    18
    20
    Units: days
        arithmetic mean (standard deviation)
    17.6 ( 14.2 )
    10.7 ( 10.79 )
    No statistical analyses for this end point

    Secondary: Duration of nasal continuous positive airway pressure (nCPAP)

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    End point title
    		 Duration of nasal continuous positive airway pressure (nCPAP)
    End point description
    End point type
    Secondary
    End point timeframe
    Assessments concerning nCPAP following CHF 5633 administration were performed at: - 30 minutes - 1, 3, 6, 12, 24 hours - 2, 3, 5, 7 days - in the follow-up period
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    18
    20
    Units: days
        arithmetic mean (standard deviation)
    17.5 ( 14.13 )
    10.7 ( 10.8 )
    No statistical analyses for this end point

    Secondary: Number of non-responders receiving rescue treatment

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    End point title
    		 Number of non-responders receiving rescue treatment
    End point description
    End point type
    Secondary
    End point timeframe
    Number of non-responders was assessed based on the administration of rescue treatment (poractant alfa) at 1, 3, 6, 12 and 24 hours as needed
    End point values
    		 Cohort A - Safety/Efficacy analysis Cohort B - Safety/Efficacy analysis
    Number of subjects analysed
    20
    20
    Units: integer
    1
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were monitored on a continual basis for the first 7 days following treatment. Adverse events that were ongoing at Day 7, together with any new AEs were evaluated at Days 10 and 28, at discharge home, and the Week 36 PMA.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    Cohort A - Safety population
    Reporting group description
    		 -

    Reporting group title
    Cohort B - Safety population
    Reporting group description
    		 -

    Serious adverse events
    		 Cohort A - Safety population Cohort B - Safety population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    0
    0
    Gastrointestinal disorders
    Necrotising enterocolitis neonatal
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Infections and infestations
    Bronchiolitis
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort A - Safety population Cohort B - Safety population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    19 / 20 (95.00%)
    19 / 20 (95.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Pregnancy, puerperium and perinatal conditions
    Jaundice neonatal
         subjects affected / exposed
    6 / 20 (30.00%)
    2 / 20 (10.00%)
         occurrences all number
    7
    3
    General disorders and administration site conditions
    Oedema
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Bronchopulmonary dysplasia
         subjects affected / exposed
    2 / 20 (10.00%)
    2 / 20 (10.00%)
         occurrences all number
    2
    2
    Infantile apnoeic attack
         subjects affected / exposed
    2 / 20 (10.00%)
    5 / 20 (25.00%)
         occurrences all number
    2
    5
    Neonatal respiratory distress syndrome
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Pneumothorax
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    Pulmonary hypertension
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Pulmonary interstitial emphysema syndrome
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Pulmonary oedema
         subjects affected / exposed
    3 / 20 (15.00%)
    1 / 20 (5.00%)
         occurrences all number
    3
    1
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    2
    2
    Blood phosphorus decreased
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Cardiac murmur
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Chest X-ray abnormal
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Staphylococcal identification test positive
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Endotracheal intubation complication
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    Congenital, familial and genetic disorders
    Atrial septal defect
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    2
    Patent ductus arteriosus
         subjects affected / exposed
    4 / 20 (20.00%)
    2 / 20 (10.00%)
         occurrences all number
    4
    2
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Cerebral haemorrage
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Intraventricular haemorrhage neonatal
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Periventricular leukomalacia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Anemia neonatal
         subjects affected / exposed
    2 / 20 (10.00%)
    2 / 20 (10.00%)
         occurrences all number
    3
    2
    Leukocytosis
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Neutrophilia
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    4 / 20 (20.00%)
    2 / 20 (10.00%)
         occurrences all number
    4
    2
    Flatulence
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Necrotising colitis
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Hyperbilirubinaemia
         subjects affected / exposed
    3 / 20 (15.00%)
    10 / 20 (50.00%)
         occurrences all number
    3
    10
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Osteopenia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Bacterial infection
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    Bacterial sepsis
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Enterococcal sepsis
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Eye infection bacterial
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    2
    Eye infection staphylococcal
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    Sepsis
         subjects affected / exposed
    1 / 20 (5.00%)
    2 / 20 (10.00%)
         occurrences all number
    1
    2
    Staphylococcal infection
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    Hypernatraemia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    3
    0
    Hypokalaemia
         subjects affected / exposed
    3 / 20 (15.00%)
    0 / 20 (0.00%)
         occurrences all number
    3
    0
    Hyponatraemia
         subjects affected / exposed
    8 / 20 (40.00%)
    3 / 20 (15.00%)
         occurrences all number
    9
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Apr 2012
    • Added central laboratory in charge of immunogenicity • Added role of clinical pharmacologist • Provided details and purpose of 24-month assessments • Clarified “original value” for blood chemistry results • Updated planned study start and end • Clarified the characteristics and role of the independent member of the Safety Monitoring Board • Clarified the stopping rules of the study and the processes for managing risk and escalating the dose • Added dedicated section for immunogenicity assessment • Added the Contract Research Organization safety contact • Clarified the serious adverse event reporting procedure • Added Safety Monitoring Board charter as protocol appendix
    01 Jul 2013
    • Increased number of sites from 6 to 12 to speed up enrolment • Extended period for IMP administration from within 24 hours to within 48 hours from birth to allow Investigators more time to speak with parents and explain procedures better. • Extended in agreement with the Central Laboratory the window to take the blood sample for immunogenicity assessments in serum from 4 to 12 weeks to 3 to 12 weeks to allow a more flexible interval to the Investigators. • Included the possibility that neonates might have been transferred to a continuing care site and added instructions for continued safety monitoring when this occurred • Allowed the use of a less invasive surfactant administration technique with rapid extubation (InSurE) • Redefined the end of the trial as the date of discharge home to take the continuing care sites into account • Extended the recruitment period

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    No limitations or caveats are applicable to this summary of the results.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28465315
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