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    Clinical Trial Results:
    Randomised, open-label, active-controlled, multicentre, comparative study to evaluate the safety and efficacy of ferric maltol (iron(III)-maltol complex) (ST10) oral suspension compared to ferrous sulfate oral liquid in children and adolescents aged 2 to 17 years with iron-deficiency anaemia, incorporating a single arm study in infants aged 1 month to less than 2 years

    Summary
    EudraCT number
    		 2018-000078-31
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    09 Jun 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Jul 2025
    First version publication date
    27 Jul 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ST10-01-305
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05126901
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 USA IND: 114832
    Sponsors
    Sponsor organisation name
    Shield TX (UK) Ltd.
    Sponsor organisation address
    Northern Design Centre, Baltic Business Quarter, Gateshead, United Kingdom, NE8 3DF
    Public contact
    Clinical Operations, Shield TX (UK) Ltd., info@shieldtherapeutics.com
    Scientific contact
    Clinical Operations, Shield TX (UK) Ltd., info@shieldtherapeutics.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001195-PIP01-11
    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
    09 Jun 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Jun 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    1. To compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period; and 2. To assess the effect on Hb in children and adolescents aged 1 month to 17 years after BID ferric maltol oral suspension administration for 12 weeks.
    Protection of trial subjects
    The study was conducted in accordance with the Declaration of Helsinki and with all applicable laws and regulations of the locales and countries where the study was conducted, and in compliance with Good Clinical Practice Guidelines.
    Background therapy
    		 -
    Evidence for comparator
    		 The mainstay of treatment of iron deficiency anemia is oral iron supplements. Ferrous compounds (sulfate, fumarate, and gluconate), which are available both in solid and liquid forms, are the most common due to the extremely low bioavailability of conventional ferric preparations.
    Actual start date of recruitment
    03 Nov 2021
    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 States: 52
    Country: Number of subjects enrolled
    United Kingdom: 13
    Worldwide total number of subjects
    65
    EEA total number of subjects
    0
    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)
    4
    Children (2-11 years)
    21
    Adolescents (12-17 years)
    40
    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
    		 This was a multicenter study with a total of 23 clinical sites located in the United States(including Puerto Rico) and United Kingdom. A total of 65 patients were enrolled in the study.

    Pre-assignment
    Screening details
    		 All eligible subjects aged 1 month to less than 2 years entered a Pre-assignment phase, 1-day Pharmacokinetic assessment day following a single dose of ferric maltol oral suspension.

    Period 1
    Period 1 title
    Treatment Period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ferrous Sulfate Randomized
    Arm description
    		 Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Ferrous sulfate
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral liquid
    Routes of administration
    Oral use
    Dosage and administration details
    Ferrous sulfate oral liquid 125 mg/mL (25 mg/mL elemental iron) or equivalent concentration was used for all children/adolescents. To maximize the iron replenishment, patients aged 2 to 17 years were dosed with 0.24 mL (6 mg elemental iron) per kg body weight, up to a maximum of 8 mL given daily in 2 divided doses.

    Arm title
    		 Ferric Maltol Randomized
    Arm description
    		 Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days. Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12. Ferrous maltol: 2-11 years 15 mg elemental iron (2.5mL) BID; 12-17 years 30mg elemental iron (5mL) BID
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ferric maltol
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral liquid
    Routes of administration
    Oral use
    Dosage and administration details
    The dose of ferric maltol oral suspension administered for children aged 2 to - 11 yrs was 2.5 ml BID and for children aged 12-17 yrs: 5 ml BID.

    Arm title
    		 Ferric Maltol Assigned
    Arm description
    		 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ferric maltol
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral liquid
    Routes of administration
    Oral use
    Dosage and administration details
    The dose of ferric maltol oral suspension that was administered for children aged 1 month to < 2 yrs is 0.1 ml/kg BID

    Number of subjects in period 1
    		Ferrous Sulfate Randomized Ferric Maltol Randomized Ferric Maltol Assigned
    Started
    30
    31
    4
    Completed
    25
    28
    3
    Not completed
    5
    3
    1
         Consent withdrawn by subject
    4
    1
    1
         Adverse event, non-fatal
    1
    -
    -
         Lost to follow-up
    -
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ferrous Sulfate Randomized
    Reporting group description
    		 Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.

    Reporting group title
    Ferric Maltol Randomized
    Reporting group description
    		 Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days. Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12. Ferrous maltol: 2-11 years 15 mg elemental iron (2.5mL) BID; 12-17 years 30mg elemental iron (5mL) BID

    Reporting group title
    Ferric Maltol Assigned
    Reporting group description
    		 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years

    Reporting group values
    		 Ferrous Sulfate Randomized Ferric Maltol Randomized Ferric Maltol Assigned Total
    Number of subjects
    		 30 31 4 65
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 4 4
        Children (2-11 years)
    		 11 10 0 21
        Adolescents (12-17 years)
    		 19 21 0 40
        Adults (18-64 years)
    		 0 0 0 0
        From 65-84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 22 23 3 48
        Male
    		 8 8 1 17

    End points

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    End points reporting groups
    Reporting group title
    Ferrous Sulfate Randomized
    Reporting group description
    		 Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.

    Reporting group title
    Ferric Maltol Randomized
    Reporting group description
    		 Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days. Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12. Ferrous maltol: 2-11 years 15 mg elemental iron (2.5mL) BID; 12-17 years 30mg elemental iron (5mL) BID

    Reporting group title
    Ferric Maltol Assigned
    Reporting group description
    		 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years

    Subject analysis set title
    mITT Ferrous Sulfate
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.

    Subject analysis set title
    mITT Ferric Maltol
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.

    Subject analysis set title
    mITT Ferric Maltol Assigned
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.

    Subject analysis set title
    Safety Population Ferrous Sulfate
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The Safety Population is defined as all randomized/assigned patients who received at least 1 dose of study drug.

    Subject analysis set title
    Safety Population Ferric Maltol
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The Safety Population is defined as all randomized/assigned patients who received at least 1 dose of study drug.

    Subject analysis set title
    Safety Population Ferric Maltol Assigned
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The Safety Population is defined as all randomized/assigned patients who received at least 1 dose of study drug.

    Subject analysis set title
    PK Population Ferric Maltol 15mg (2-9 y) Day 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Subject analysis set title
    PK Population Ferric Maltol 15mg (10-17 y) Day 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Subject analysis set title
    PK Population Ferric Maltol 30mg (10-17 y) Day 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Subject analysis set title
    PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Subject analysis set title
    PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Subject analysis set title
    PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample (applicable only for the Ferric Maltol group). A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.

    Primary: Change in hemoglobin concentration

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    End point title
    		 Change in hemoglobin concentration [1]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to week 12
    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: The change in Hb concentration from baseline to Week 12 is summarised based on the mITT Population for each treatment group using descriptive statistics summarised by mean, standard deviation, median, and range (minimum and maximum).
    End point values
    		 mITT Ferrous Sulfate mITT Ferric Maltol mITT Ferric Maltol Assigned
    Number of subjects analysed
    Units: g/L
        arithmetic mean (standard deviation)
    11.5 ( 13.97 )
    12.5 ( 13.89 )
    17.7 ( 13.61 )
    No statistical analyses for this end point

    Secondary: Changes in ferritin concentration

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    End point title
    		 Changes in ferritin concentration
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 12
    End point values
    		 mITT Ferrous Sulfate mITT Ferric Maltol mITT Ferric Maltol Assigned
    Number of subjects analysed
    28
    31
    3
    Units: µg/L
        arithmetic mean (standard deviation)
    20.6 ( 30.97 )
    8.1 ( 11.06 )
    6.3 ( 8.74 )
    No statistical analyses for this end point

    Secondary: Change in Iron concentration

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    End point title
    		 Change in Iron concentration
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 12.
    End point values
    		 mITT Ferrous Sulfate mITT Ferric Maltol mITT Ferric Maltol Assigned
    Number of subjects analysed
    28
    31
    3
    Units: µmol/L
        arithmetic mean (standard deviation)
    3.64 ( 8.911 )
    5.77 ( 8.518 )
    1.07 ( 0.577 )
    No statistical analyses for this end point

    Secondary: Change in transferrin saturation

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    End point title
    		 Change in transferrin saturation
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 12.
    End point values
    		 mITT Ferrous Sulfate mITT Ferric Maltol mITT Ferric Maltol Assigned
    Number of subjects analysed
    28
    31
    2
    Units: %
        arithmetic mean (standard deviation)
    6.5 ( 12.39 )
    7.7 ( 10.14 )
    2.0 ( 0.00 )
    No statistical analyses for this end point

    Secondary: Cmax for plasma maltol glucuronide

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    End point title
    		 Cmax for plasma maltol glucuronide
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: ng/mL
        number (not applicable)
    4350
    6810
    6420
    4250
    5940
    8160
    No statistical analyses for this end point

    Secondary: Tmax for plasma maltol glucuronide

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    End point title
    		 Tmax for plasma maltol glucuronide
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: h
        number (not applicable)
    1.00
    2.13
    0.52
    0.98
    2.78
    3.63
    No statistical analyses for this end point

    Secondary: AUC0-t for plasma maltol glucuronide

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    End point title
    		 AUC0-t for plasma maltol glucuronide
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: hxng/mL
        number (not applicable)
    4670
    12000
    14600
    5900
    8070
    16700
    No statistical analyses for this end point

    Secondary: Cmax for baseline corrected serum iron

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    End point title
    		 Cmax for baseline corrected serum iron
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: µg/dL
        number (not applicable)
    87.0
    40.0
    119
    96.5
    21.0
    364
    No statistical analyses for this end point

    Secondary: Tmax for baseline corrected serum iron

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    End point title
    		 Tmax for baseline corrected serum iron
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: h
        number (not applicable)
    2.37
    2.13
    2.00
    2.10
    2.78
    6.63
    No statistical analyses for this end point

    Secondary: AUC0-t for baseline corrected serum iron

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    End point title
    		 AUC0-t for baseline corrected serum iron
    End point description
    After a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose.
    End point type
    Secondary
    End point timeframe
    PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3)
    End point values
    		 PK Population Ferric Maltol 15mg (2-9 y) Day 1 PK Population Ferric Maltol 15mg (10-17 y) Day 1 PK Population Ferric Maltol 30mg (10-17 y) Day 1 PK Population Ferric Maltol 15mg (2-9 y) Day 7-10 PK Population Ferric Maltol 15mg (10-17 y) Day 7-10 PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
    Number of subjects analysed
    7
    3
    10
    7
    3
    10
    Units: hxµg/dL
        number (not applicable)
    64.4
    60.1
    374
    100
    1.93
    1180
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Ferrous Sulfate Randomized
    Reporting group description
    		 Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.

    Reporting group title
    Ferric Maltol Randomized
    Reporting group description
    		 Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days. Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12. Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2 - 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.

    Reporting group title
    Ferric Maltol Assigned
    Reporting group description
    		 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years

    Serious adverse events
    		 Ferrous Sulfate Randomized Ferric Maltol Randomized Ferric Maltol Assigned
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 31 (0.00%)
    1 / 3 (33.33%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Wheezing
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 31 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Ferrous Sulfate Randomized Ferric Maltol Randomized Ferric Maltol Assigned
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 30 (10.00%)
    4 / 31 (12.90%)
    2 / 3 (66.67%)
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 31 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Pyrexia
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    2 / 31 (6.45%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Gastrointestinal disorders
    Diarrhoea
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 31 (3.23%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Teething
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 31 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Nausea
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    3 / 30 (10.00%)
    2 / 31 (6.45%)
    0 / 3 (0.00%)
         occurrences all number
    3
    3
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 31 (3.23%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Infections and infestations
    Conjunctivitis
    Additional description: Occurrences are provided for Treatment Emergent Adverse Events (TEAEs). TEAEs were defined as AEs that started after the first dose of study drug.
         subjects affected / exposed
    0 / 30 (0.00%)
    0 / 31 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 May 2021
    • Study design updated with addition of urine sample in the pre-assignment PK phase and assigned treatment phase. • Update of visit window for Visit 3.
    03 Nov 2022
    • Changes to PK sampling, blood volume and study design. • Changes to exclusion/discontinuation criteria and permitted medications. • Changes to the secondary endpoints and secondary endpoint analysis.
    14 Mar 2023
    • Changes to study design • Changes to the primary endpoints/primary objectives
    06 Jul 2023
    • Removal of ferrous sulfate sample size reduction • Removal of late timepoints in PK sampling for younger cohort (2-9 years) to match sampling for older age group (10-17 years) • Addition of potential interim analysis and associated stopping rules • Removal of blood draws during preassignment PK in infants • Inclusion of quantitative efficacy measure

    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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