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    Clinical Trial Results:
    A Multicenter, Multinational, Randomized, Active-Controlled Study to Investigate the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients with Iron Deficiency Anemia

    Summary
    EudraCT number
    		 2018-002078-27
    Trial protocol
    		 PL  
    Global end of trial date
    22 Jun 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Nov 2021
    First version publication date
    14 Nov 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    1VIT17044
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03523117
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    American Regent, Inc.
    Sponsor organisation address
    800 Adams Avenue, Suite 200, Norristown, United States, PA 19403
    Public contact
    Clinical Trial Information, American Regent, Inc., +1 610 650 4200,
    Scientific contact
    Mark Falone, MD, American Regent, Inc., +1 6317723500,
    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
    25 Jan 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Jun 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Jun 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To demonstrate the efficacy and safety of intravenous (IV) ferric carboxymaltose (FCM), compared to oral iron,
    Protection of trial subjects
    The parent(s)/guardian(s) and the minors, if appropriate for age, were informed by the Investigator about the nature of the study, along with the aims, methods, anticipated benefits, potential hazards, and discomfort that participation may have entailed. Written informed consent and assent were obtained from the parent(s)/guardian(s) and the minors, if appropriate for age. The study protocol and the informed consent form were submitted to the Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. The study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    31 Jan 2019
    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
    Poland: 9
    Country: Number of subjects enrolled
    Ukraine: 35
    Country: Number of subjects enrolled
    United States: 35
    Worldwide total number of subjects
    79
    EEA total number of subjects
    9
    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)
    5
    Children (2-11 years)
    13
    Adolescents (12-17 years)
    61
    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
    		 The study was conducted at 30 sites in four countries - Canada, Poland, Ukraine, and US. No subjects were enrolled at the Canadian sites.

    Pre-assignment
    Screening details
    		 The screening period, starting at Day -7 (+1) and following obtainment of informed consent/assent, was of maximum 8 days to allow for all screening results to be obtained and validated.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1
    Arm description
    		 Subjects assigned to Cohort 1 received two doses (on Day 0 and Day 7) of Ferric Carboxymaltose (FCM) at 15 mg/kg to a maximum single dose of 750 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ferric Carboxymaltose
    Investigational medicinal product code
    FCM
    Other name
    Injectafer®
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    15 mg/kg to a maximum single dose of 750 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

    Arm title
    		 Cohort 2
    Arm description
    		 Subjects assigned to Cohort 2 received an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age received 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 received 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) received oral ferrous sulfate drops, while children (ages ≥4 to <12 years) received oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) received an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants was 130 mg of elemental iron.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Ferrous sulate monohydrate
    Investigational medicinal product code
    Ferrous Sulfate Lomapharm
    Other name
    Pharmaceutical forms
    Film-coated tablet, Oral drops, Oral liquid
    Routes of administration
    Oral use
    Dosage and administration details
    Age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age received 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 received 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) received oral ferrous sulfate drops, while children (ages ≥4 to <12 years) received oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) received an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants was 130 mg of elemental iron.

    Number of subjects in period 1
    		Cohort 1 Cohort 2
    Started
    40
    39
    Completed
    40
    39

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Subjects assigned to Cohort 1 received two doses (on Day 0 and Day 7) of Ferric Carboxymaltose (FCM) at 15 mg/kg to a maximum single dose of 750 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Subjects assigned to Cohort 2 received an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age received 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 received 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) received oral ferrous sulfate drops, while children (ages ≥4 to <12 years) received oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) received an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants was 130 mg of elemental iron.

    Reporting group values
    		 Cohort 1 Cohort 2 Total
    Number of subjects
    		 40 39 79
    Age categorical
    Mean age of subjects assigned to Cohort 1 (FCM) was 12.5 years. Mean age of subjects assigned to cohort 2 (Oral ferric sulfate) was 12.8 years.
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 3 2 5
        Children (2-11 years)
    		 7 6 13
        Adolescents (12-17 years)
    		 30 31 61
        Adults (18-64 years)
    		 0 0 0
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 12.5 ( 4.84 ) 12.8 ( 4.35 ) -
    Gender categorical
    Units: Subjects
        Female
    		 33 30 63
        Male
    		 7 9 16

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Subjects assigned to Cohort 1 received two doses (on Day 0 and Day 7) of Ferric Carboxymaltose (FCM) at 15 mg/kg to a maximum single dose of 750 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Subjects assigned to Cohort 2 received an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age received 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 received 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) received oral ferrous sulfate drops, while children (ages ≥4 to <12 years) received oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) received an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants was 130 mg of elemental iron.

    Primary: Hemoglobin

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    End point title
    		 Hemoglobin
    End point description
    End point type
    Primary
    End point timeframe
    Baseline Day 35
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    40
    39
    Units: g/dL
        least squares mean (standard error)
    2.22 ( 0.266 )
    1.92 ( 0.244 )
    Statistical analysis title
    		 Hemoglobin
    Comparison groups
    Cohort 1 v Cohort 2
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.28
         upper limit
    		 0.88
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.293

    Secondary: Ferritin

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    End point title
    		 Ferritin
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline Day 35
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    40
    39
    Units: ng/mL
        least squares mean (standard error)
    132.1 ( 13.38 )
    11.01 ( 13.368 )
    Statistical analysis title
    		 Ferritin
    Comparison groups
    Cohort 1 v Cohort 2
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (final values)
    Point estimate
    121.08
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 88.93
         upper limit
    		 153.24
    Variability estimate
    Standard error of the mean
    Dispersion value
    16.137

    Secondary: Transferrin Saturation

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    End point title
    		 Transferrin Saturation
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline Day 35
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    40
    39
    Units: %
        least squares mean (standard error)
    24.3 ( 2.563 )
    8.66 ( 2.491 )
    Statistical analysis title
    		 Transferrin Saturation
    Comparison groups
    Cohort 1 v Cohort 2
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (final values)
    Point estimate
    15.64
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 9.59
         upper limit
    		 21.69
    Variability estimate
    Standard error of the mean
    Dispersion value
    3.037

    Secondary: Reticulocyte Hemoglobin Content

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    End point title
    		 Reticulocyte Hemoglobin Content
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline Day 35
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    35
    36
    Units: pg
        least squares mean (standard error)
    6.95 ( 0.374 )
    4.9 ( 0.377 )
    Statistical analysis title
    		 Reticulocyte Hemoglobin Content
    Comparison groups
    Cohort 1 v Cohort 2
    Number of subjects included in analysis
    71
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 > 0.0002
    Method
    		 mixed Mixed Model Repeated Measures
    Parameter type
    		 Mean difference (final values)
    Point estimate
    2.06
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1
         upper limit
    		 3.12
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.533

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 0 to Day 35
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Subjects assigned to Cohort 1 received two doses (on Day 0 and Day 7) of Ferric Carboxymaltose (FCM) at 15 mg/kg to a maximum single dose of 750 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Subjects assigned to Cohort 2 received an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age received 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 received 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) received oral ferrous sulfate drops, while children (ages ≥4 to <12 years) received oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) received an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants was 130 mg of elemental iron.

    Serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 40 (0.00%)
    0 / 38 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 40 (35.00%)
    10 / 38 (26.32%)
    Investigations
    Investigations
         subjects affected / exposed
    3 / 40 (7.50%)
    1 / 38 (2.63%)
         occurrences all number
    3
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 40 (5.00%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    General disorders and administration site conditions
    General disorders and administration site conditions
         subjects affected / exposed
    3 / 40 (7.50%)
    0 / 38 (0.00%)
         occurrences all number
    3
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    2 / 40 (5.00%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    Constipation
         subjects affected / exposed
    0 / 40 (0.00%)
    5 / 38 (13.16%)
         occurrences all number
    0
    5
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    2 / 40 (5.00%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 40 (2.50%)
    2 / 38 (5.26%)
         occurrences all number
    1
    2
    Metabolism and nutrition disorders
    Hypophosphatemia
         subjects affected / exposed
    4 / 40 (10.00%)
    0 / 38 (0.00%)
         occurrences all number
    4
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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