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    Clinical Trial Results:
    What is the effect of intravenous iron supplementation on cardiopulmonary haemodynamics, exercise capacity and quality of life in patients with IPAH and iron deficiency?

    Summary
    EudraCT number
    2010-024585-22
    Trial protocol
    GB   DE  
    Global end of trial date
    22 Dec 2017

    Results information
    Results version number
    v1(current)
    This version publication date
    09 Oct 2019
    First version publication date
    09 Oct 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WILK3
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01447628
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Imperial College London
    Sponsor organisation address
    South Kensington Campus, London, United Kingdom,
    Public contact
    Luke Howard, Luke Howard, l.howard@imperial.ac.uk
    Scientific contact
    Luke Howard, Luke Howard, l.howard@imperial.ac.uk
    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
    12 Jul 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Dec 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Dec 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the clinical value of using intravenous iron (ferric carboxymaltose) infusion in iron deficient patients with idiopathic pulmonary arterial hypertension. The primary endpoint will be endurance time at the end of endurance bicycle cardiopulmonary exercise testing at 80% peak work rate determined from the baseline incremental exercise test, measured at 12 weeks after study treatment.
    Protection of trial subjects
    All adverse events were monitored and recorded throughout the trial. No other specific protection.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    29 Mar 2011
    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: 34
    Country: Number of subjects enrolled
    Germany: 5
    Worldwide total number of subjects
    39
    EEA total number of subjects
    39
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    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)
    34
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Patients participating in this study are adult males and females with symptomatic IPAH as defined by the eligibility criteria specified in the protocol.

    Pre-assignment
    Screening details
    Potential participants will be screened using data collected during their routine outpatient appointment at the Pulmonary Hypertension Service or PH Clinic. Any unavailable or missing data will be collected at the screening visit (week 0) once the patient has provided written informed consent.

    Pre-assignment period milestones
    Number of subjects started
    39
    Number of subjects completed
    39

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    All patients
    Arm description
    There are no products specified, as this is the baseline period, prior to randomisation.
    Arm type
    Baseline: no intervention

    Investigational medicinal product name
    Ferinject
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1000mg Ferinject, intravenous infusion

    Number of subjects in period 1
    All patients
    Started
    39
    Completed
    39
    Period 2
    Period 2 title
    Treatment
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Active treatment: Ferinject
    Arm description
    1000mg Ferinject infused over 15 minutes
    Arm type
    Experimental

    Investigational medicinal product name
    Ferinject
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1000mg Ferinject, intravenous infusion

    Arm title
    Placebo: Saline
    Arm description
    Saline infused over 15 minutes
    Arm type
    Placebo

    Investigational medicinal product name
    Saline
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1000ml saline for intravenous infusion

    Number of subjects in period 2
    Active treatment: Ferinject Placebo: Saline
    Started
    39
    39
    Completed
    39
    39

    Baseline characteristics

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

    Reporting group values
    Baseline Total
    Number of subjects
    39 39
    Age categorical
    All participants
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    0 0
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    34 34
        From 65-84 years
    5 5
        85 years and over
    0 0
    Gender categorical
    Units: Subjects
        Female
    29 29
        Male
    10 10
    Subject analysis sets

    Subject analysis set title
    Total population
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    All participants

    Subject analysis sets values
    Total population
    Number of subjects
    39
    Age categorical
    All participants
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        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)
    34
        From 65-84 years
    5
        85 years and over
    0
    Age continuous
    Units:
        
    ±
    Gender categorical
    Units: Subjects
        Female
    29
        Male
    10

    End points

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    End points reporting groups
    Reporting group title
    All patients
    Reporting group description
    There are no products specified, as this is the baseline period, prior to randomisation.
    Reporting group title
    Active treatment: Ferinject
    Reporting group description
    1000mg Ferinject infused over 15 minutes

    Reporting group title
    Placebo: Saline
    Reporting group description
    Saline infused over 15 minutes

    Subject analysis set title
    Total population
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    All participants

    Primary: Endurance time from start to finish of Cardio-pulmonary exercise test

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    End point title
    Endurance time from start to finish of Cardio-pulmonary exercise test
    End point description
    End point type
    Primary
    End point timeframe
    12 weeks post treatment
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: seconds
        arithmetic mean (confidence interval 95%)
    315.44 (274.68 to 356.20)
    302.89 (260.09 to 345.69)
    Statistical analysis title
    Linear mixed model
    Statistical analysis description
    All primary and secondary efficacy endpoints were analysed using linear mixed models appropriate for a crossover design. The linear models included administration sequence, period and treatment as fixed effects and subject as a random effect, and all models included relevant and statistically significant baseline covariates.
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.7109
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Peak VO2 Level at 12 Weeks After Study Treatment

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    End point title
    Peak VO2 Level at 12 Weeks After Study Treatment
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks post study treatment
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: litres per minute
        arithmetic mean (confidence interval 95%)
    1.17 (1.10 to 1.22)
    1.13 (1.08 to 1.21)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.6583
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VO2 at Metabolic Threshold

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    End point title
    VO2 at Metabolic Threshold
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks post study treatment
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: litres per minute
        arithmetic mean (confidence interval 95%)
    0.78 (0.73 to 0.82)
    0.77 (0.73 to 0.82)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.9773
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VE/VCO2 Slope

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    End point title
    VE/VCO2 Slope
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: interger
        arithmetic mean (confidence interval 95%)
    41.15 (39.43 to 42.43)
    42.35 (40.58 to 44.07)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.1815
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VO2 / WR Slope

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    End point title
    VO2 / WR Slope
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: integer
        arithmetic mean (confidence interval 95%)
    8.12 (7.50 to 8.71)
    8.04 (7.46 to 8.68)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.8751
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Peak O2 Pulse Rate

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    End point title
    Peak O2 Pulse Rate
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: interger
        arithmetic mean (confidence interval 95%)
    8.83 (8.36 to 9.28)
    8.62 (8.17 to 9.08)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.4813
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VO2 at the End of Endurance Cardio-pulmonary Exercise Test

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    End point title
    VO2 at the End of Endurance Cardio-pulmonary Exercise Test
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: litres per minute
        least squares mean (confidence interval 95%)
    4.25 (3.5 to 5.01)
    4.49 (3.71 to 5.28)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.2395
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VO2 at 3 mins

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    End point title
    VO2 at 3 mins
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: integer
        least squares mean (confidence interval 95%)
    1.29 (0.98 to 1.60)
    1.14 (0.81 to 1.48)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.4758
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Iron Indices: Serum Iron

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    End point title
    Iron Indices: Serum Iron
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: umol/l
        arithmetic mean (confidence interval 95%)
    17.15 (15.42 to 18.64)
    15.48 (13.94 to 17.17)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.2002
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Iron Indices: Transferrin Saturations

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    End point title
    Iron Indices: Transferrin Saturations
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: percentage
        arithmetic mean (confidence interval 95%)
    26.2 (22.96 to 28.89)
    22 (19.21 to 25.12)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0727
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Iron Indices: Ferritin

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    End point title
    Iron Indices: Ferritin
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: ug/L
        arithmetic mean (confidence interval 95%)
    150.32 (118.63 to 175.51)
    92.74 (66.08 to 122.88)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0003
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Iron Indices: sTfR

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    End point title
    Iron Indices: sTfR
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: nmol/L
        arithmetic mean (confidence interval 95%)
    28.37 (25.73 to 31.72)
    37.25 (34.02 to 40.0)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0001
    Method
    Mixed models analysis
    Confidence interval

    Secondary: 6 minute walk test: Distance Walked

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    End point title
    6 minute walk test: Distance Walked
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: metres
        arithmetic mean (confidence interval 95%)
    426.03 (411.57 to 440.20)
    424.3 (410.06 to 438.70)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.8093
    Method
    Mixed models analysis
    Confidence interval

    Secondary: 6 minute walk test: Borg dyspnoea score after test

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    End point title
    6 minute walk test: Borg dyspnoea score after test
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: Units on a scale
        arithmetic mean (confidence interval 95%)
    3.24 (2.75 to 3.75)
    3.68 (3.18 to 4.17)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Placebo: Saline v Active treatment: Ferinject
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0862
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Iron Indices: NT-pro-BNP

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    End point title
    Iron Indices: NT-pro-BNP
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: fmol/ml
        arithmetic mean (confidence interval 95%)
    307.64 (186.47 to 437.62)
    424.37 (297.52 to 548.39)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.1041
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Quality of Life: CAMPHOR Activity Score

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    End point title
    Quality of Life: CAMPHOR Activity Score
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: Units on a scale
        arithmetic mean (confidence interval 95%)
    9.12 (8.14 to 10.07)
    9.18 (8.25 to 10.16)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.8948
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Quality of Life: CAMPHOR Symptom Score

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    End point title
    Quality of Life: CAMPHOR Symptom Score
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: Items on a scale
        arithmetic mean (confidence interval 95%)
    7.94 (6.99 to 8.89)
    7.94 (7.02 to 8.89)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.9559
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Quality of Life: CAMPHOR QoL Score

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    End point title
    Quality of Life: CAMPHOR QoL Score
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: Items on a scale
        arithmetic mean (confidence interval 95%)
    7.58 (6.38 to 8.69)
    7 (5.91 to 8.16)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.2423
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Mean Right Atrial Pressure

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    End point title
    Mean Right Atrial Pressure
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: TBC
        arithmetic mean (confidence interval 95%)
    6.56 (5.40 to 9.23)
    10.12 (7.17 to 11.21)
    Statistical analysis title
    Linear mixed method
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.1221
    Method
    Mixed models analysis
    Confidence interval

    Secondary: Peak VO2 Level in ml/min/kg

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    End point title
    Peak VO2 Level in ml/min/kg
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: ml/min/kg
        arithmetic mean (confidence interval 95%)
    15.10 (14.10 to 15.58)
    14.67 (14.20 to 15.68)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.7625
    Method
    Mixed models analysis
    Confidence interval

    Secondary: VO2 at Metabolic Threshold measured during incremental CPET

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    End point title
    VO2 at Metabolic Threshold measured during incremental CPET
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks post study treatment
    End point values
    Active treatment: Ferinject Placebo: Saline
    Number of subjects analysed
    39
    39
    Units: ml/min/kg
        arithmetic mean (confidence interval 95%)
    10.01 (9.45 to 10.46)
    9.99 (9.52 to 10.53)
    Statistical analysis title
    Linear mixed model
    Comparison groups
    Active treatment: Ferinject v Placebo: Saline
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.7711
    Method
    Mixed models analysis
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From consent to 24 weeks
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    10
    Reporting groups
    Reporting group title
    All participants
    Reporting group description
    -

    Serious adverse events
    All participants
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 39 (20.51%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Hypophosphataemia
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Spinal haematoma
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Disease progression
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Haemorrhagic ovarian cyst
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Tonsilitis
         subjects affected / exposed
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    All participants
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    35 / 39 (89.74%)
    Investigations
    Hypophosphataemia
         subjects affected / exposed
    4 / 39 (10.26%)
         occurrences all number
    6
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    4 / 39 (10.26%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    3
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    7 / 39 (17.95%)
         occurrences all number
    10
    Headache
         subjects affected / exposed
    6 / 39 (15.38%)
         occurrences all number
    8
    Cough
         subjects affected / exposed
    3 / 39 (7.69%)
         occurrences all number
    3
    Abdominal pain
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    2
    Pyrexia
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 39 (10.26%)
         occurrences all number
    4
    Nausea
         subjects affected / exposed
    3 / 39 (7.69%)
         occurrences all number
    3
    Vomiting
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    4 / 39 (10.26%)
         occurrences all number
    4
    Infections and infestations
    Cold
         subjects affected / exposed
    8 / 39 (20.51%)
         occurrences all number
    11
    Respiratory Infection
         subjects affected / exposed
    3 / 39 (7.69%)
         occurrences all number
    3
    Flu symptoms
         subjects affected / exposed
    3 / 39 (7.69%)
         occurrences all number
    3
    Urinary tract infection
         subjects affected / exposed
    2 / 39 (5.13%)
         occurrences all number
    3

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Aug 2011
    Change to eligibility criteria, separate patient documents for participating sites.
    02 Jul 2012
    Change inclusion criterion 3, change of PI at one participating site.
    04 Dec 2012
    Addition of fasting glucose and insulin measurements.
    24 Sep 2013
    Change of PI at participating site
    20 Mar 2014
    Changes to eligibility, reduction in number of protocol clinic visits, removal of week 36 visit and addition of activity monitor.
    25 Jun 2015
    Changes to cardiac cath, activity monitor and replacement of visits 2 and 4 with telephone calls.
    14 Dec 2015
    Addition of German site.

    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.
    Pt 2003, 3004, 4002-05 had endurance CPETs performed at incorrect workloads, so those data were replaced by imputed values. Visit 5 CPETs for 1008, 1018 and 1019 were outside protocol window. 1014 received placebo at both treatment visits in error
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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