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    Clinical Trial Results:
    A single-arm interventional Phase IV, post-authorisation study evaluating the safety of pediatric patients with transfusional hemosiderosis treated with deferasirox crushed film coated tablets

    Summary
    EudraCT number
    		 2016-003482-25
    Trial protocol
    		 GB   IT  
    Global end of trial date
    05 Dec 2019

    Results information
    Results version number
    		 v2(current)
    This version publication date
    09 Aug 2020
    First version publication date
    21 Jun 2020
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670F2429
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03372083
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@novartis.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
    05 Dec 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Dec 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Dec 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders in pediatric patients aged ≥2 to < 6 years with transfusional iron overload up to 24 weeks including 30 days safety follow up.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Jan 2018
    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
    Oman: 15
    Country: Number of subjects enrolled
    Egypt: 12
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Thailand: 5
    Country: Number of subjects enrolled
    Lebanon: 3
    Country: Number of subjects enrolled
    United Arab Emirates: 2
    Country: Number of subjects enrolled
    United Kingdom: 2
    Worldwide total number of subjects
    44
    EEA total number of subjects
    7
    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)
    44
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was conducted in 10 sites in 7 countries (one in Egypt, three in Italy, one in Lebanon, one in Oman, two in Thailand, one in the United Arab Emirates, and one in the United Kingdom).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Deferasirox
    Arm description
    		 Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject’s weight.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    Other name
    ICL670
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deferosirox was provided in tablet forms of 90, 180 and 360 mg. Tablets were crushed in the home environment and administered by sprinkling the full dose on to soft food to be consumed immediately.

    Number of subjects in period 1
    		Deferasirox
    Started
    44
    Completed
    39
    Not completed
    5
         Physician decision
    1
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject’s weight.

    Reporting group values
    		 Deferasirox Total
    Number of subjects
    		 44 44
    Age categorical
    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)
    		 44 44
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 3.05 ( 1.056 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 17 17
        Male
    		 27 27
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian
    		 36 36
        Asian
    		 8 8
    Baseline iron chelation naive participants
    Units: Subjects
        Chelation naive = Yes
    		 10 10
        Chelation naive = No
    		 34 34
    Baseline Body Mass Index
    Units: kilogram per square metre (kg/m^2)
        arithmetic mean (standard deviation)
    		 16.40 ( 2.009 ) -

    End points

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    End points reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject’s weight.

    Primary: Number of Participants with selected gastrointestinal disorders up to 24 weeks

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    End point title
    		 Number of Participants with selected gastrointestinal disorders up to 24 weeks [1]
    End point description
    To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.
    End point type
    Primary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    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: Only descriptive analysis performed
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
        Oesophagitis
    0
        Barrett's esophagitis
    0
        Stomatitis
    0
        Mouth ulceration
    0
        Gastric ulcer
    0
        Gastrointestinal haemorrhage
    0
        Abdominal pain
    2
        Diarrhoea
    4
        Nausea
    0
        Vomiting
    2
    No statistical analyses for this end point

    Secondary: Adverse Events profile

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    End point title
    		 Adverse Events profile
    End point description
    Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
        On treatment-related AE
    22
        On treatment related SAE
    0
        On treatment Deaths
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Notable Changes in ECG values from baseline

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    End point title
    		 Number of Participants with Notable Changes in ECG values from baseline
    End point description
    Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
    0
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in serum ferritin (SF)

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    End point title
    		 Absolute change from baseline in serum ferritin (SF)
    End point description
    Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: ug/L
    arithmetic mean (standard deviation)
        Baseline (BL)
    2152.7 ( 1039.06 )
        Change from BL @ Week 4
    -118.4 ( 590.20 )
        Change from BL @ Week 8
    -17.5 ( 665.68 )
        Change from BL @ Week 12
    -52.8 ( 790.26 )
        Change from BL @ Week 16
    71.9 ( 988.30 )
        Change from BL @ Week 20
    -64.3 ( 791.92 )
        Change from BL @ EOT (Week 24)
    -140.7 ( 824.01 )
    No statistical analyses for this end point

    Secondary: Number of Participants with worst post-baseline values in selected Chemistry parameters

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    End point title
    		 Number of Participants with worst post-baseline values in selected Chemistry parameters
    End point description
    Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
        Alanine Aminotransferase|Normal
    16
        Alkaline Phosphatase|Normal
    25
        Aspartate Aminotransferase|Normal
    14
        Bilirubin|Normal
    16
        Creatinine|Normal
    6
        Direct Bilirubin|Normal
    16
        Protein/Creatinine|Normal
    20
        Alanine Aminotransferase|Low
    0
        Alkaline Phosphatase|Low
    2
        Aspartate Aminotransferase|Low
    0
        Bilirubin|Low
    0
        Creatinine|Low
    29
        Direct Bilirubin|Low
    0
        Protein/Creatinine|Low
    1
        Alanine Aminotransferase|High
    28
        Alkaline Phosphatase|High
    17
        Aspartate Aminotransferase|High
    30
        Bilirubin|High
    28
        Creatinine|High
    6
        Direct Bilirubin|High
    28
        Protein/Creatinine|High
    17
        Alanine Aminotransferase|High & Low
    0
        Alkaline Phosphatase|High & Low
    0
        Aspartate Aminotransferase|High & Low
    0
        Bilirubin|High & Low
    0
        Creatinine|High & Low
    3
        Direct Bilirubin|High & Low
    0
        Protein/Creatinine|High & Low
    6
        Alanine Aminotransferase|Missing
    0
        Alkaline Phosphatase|Missing
    0
        Aspartate Aminotransferase|Missing
    0
        Bilirubin|Missing
    0
        Creatinine|Missing
    0
        Direct Bilirubin|Missing
    0
        Protein/Creatinine|Missing
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant auditory assessments changes from baseline

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    End point title
    		 Number of Participants with clinically significant auditory assessments changes from baseline
    End point description
    Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant ocular assessments changes from baseline

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    End point title
    		 Number of Participants with clinically significant ocular assessments changes from baseline
    End point description
    Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Participants
    0
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in systolic and diastolic blood pressures (mmHg)

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    End point title
    		 Absolute change from baseline in systolic and diastolic blood pressures (mmHg)
    End point description
    Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: millimetre of mercury (mmHg)
    arithmetic mean (standard deviation)
        Systolic BP-Baseline (BL)
    100.0 ( 10.10 )
        Systolic BP-Change from BL @ Week 2
    -0.1 ( 11.27 )
        Systolic BP-Change from BL @ Week 3
    -2.3 ( 7.93 )
        Systolic BP-Change from BL @ Week 4
    -0.3 ( 12.56 )
        Systolic BP-Change from BL @ Week 8
    0.3 ( 8.53 )
        Systolic BP-Change from BL @ Week 12
    -1.4 ( 12.16 )
        Systolic BP-Change from BL @ Week 16
    -1.9 ( 11.76 )
        Systolic BP-Change from BL @ Week 20
    -0.6 ( 11.67 )
        Systolic BP-Change from BL @ EOT (Week 24)
    -2.6 ( 11.54 )
        Diastolic BP-Baseline (BL)
    60.0 ( 7.85 )
        Diastolic BP-Change from BL @ Week 2
    0.7 ( 6.13 )
        Diastolic BP-Change from BL @ Week 3
    -2.1 ( 9.60 )
        Diastolic BP-Change from BL @ Week 4
    1.0 ( 10.24 )
        Diastolic BP-Change from BL @ Week 8
    -0.6 ( 13.21 )
        Diastolic BP-Change from BL @ Week 12
    1.7 ( 11.42 )
        Diastolic BP-Change from BL @ Week 16
    -0.5 ( 10.28 )
        Diastolic BP-Change from BL @ Week 20
    -0.1 ( 11.70 )
        Diastolic BP-Change from BL @ EOT (Week 24)
    -0.4 ( 7.89 )
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in pulse rate (bpm)

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    End point title
    		 Absolute change from baseline in pulse rate (bpm)
    End point description
    Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: beats per minute (bpm)
    arithmetic mean (standard deviation)
        Baseline (BL)
    102.4 ( 10.73 )
        Change from BL @ Week 2
    -1.1 ( 10.51 )
        Change from BL @ Week 3
    -2.7 ( 16.18 )
        Change from BL @ Week 4
    -3.1 ( 12.85 )
        Change from BL @ Week 8
    -1.0 ( 15.47 )
        Change from BL @ Week 12
    -0.3 ( 12.69 )
        Change from BL @ Week 16
    -2.2 ( 8.99 )
        Change from BL @ Week 20
    0.8 ( 12.55 )
        Change from BL @ EOT (Week 24)
    1.1 ( 12.10 )
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in body temperature (°C)

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    End point title
    		 Absolute change from baseline in body temperature (°C)
    End point description
    Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: Celsius degree (°C)
    arithmetic mean (standard deviation)
        Baseline (BL)
    36.43 ( 0.416 )
        Change from BL @ Week 2
    -0.03 ( 0.285 )
        Change from BL @ Week 3
    -0.10 ( 0.353 )
        Change from BL @ Week 4
    0.00 ( 0.360 )
        Change from BL @ Week 8
    0.02 ( 0.336 )
        Change from BL @ Week 12
    -0.03 ( 0.413 )
        Change from BL @ Week 16
    0.10 ( 0.400 )
        Change from BL @ Week 20
    0.03 ( 0.372 )
        Change from BL @ EOT (Week 24)
    0.08 ( 0.453 )
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in body weight (kg)

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    End point title
    		 Absolute change from baseline in body weight (kg)
    End point description
    Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    44
    Units: kilogram (kg)
    arithmetic mean (standard deviation)
        Baseline (BL)
    15.26 ( 3.109 )
        Change from BL @ Week 4
    0.08 ( 0.290 )
        Change from BL @ Week 8
    0.19 ( 0.460 )
        Change from BL @ Week 12
    0.31 ( 0.482 )
        Change from BL @ Week 16
    0.25 ( 0.433 )
        Change from BL @ Week 20
    0.43 ( 0.532 )
        Change from BL @ EOT (Week 24)
    0.44 ( 0.600 )
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in participants pre-treated with deferasirox: Mean Change from Baseline in Adherence

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in participants pre-treated with deferasirox: Mean Change from Baseline in Adherence
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Score
    arithmetic mean (confidence interval 95%)
        Child's perspective overall score-Week 4
    -2.4 (-3.8 to -1.0)
        Child's perspective overall score-Week 12
    -2.6 (-4.5 to -0.7)
        Child's perspective overall score-EOT
    -1.9 (-3.5 to -0.2)
        Caregiver's perspective overall score-Week 4
    -1.4 (-2.9 to 0.1)
        Caregiver's perspective overall score-Week 12
    -1.4 (-3.3 to 0.4)
        Caregiver's perspective overall score-EOT
    -1.0 (-2.6 to 0.6)
    No statistical analyses for this end point

    Secondary: Modified SICT in participants pre-treated with deferasirox: Number of Participants with Type of Medicine child like scoring

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    End point title
    		 Modified SICT in participants pre-treated with deferasirox: Number of Participants with Type of Medicine child like scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Participants
        BL|Tablet to dissolve in liquid
    0
        Wk 4|Tablet to dissolve in liquid
    0
        Wk 12|Tablet to dissolve in liquid
    1
        EOT|Tablet to dissolve in liquid
    0
        BL|Tablet (taken once a day)
    4
        Wk 4|Tablet (taken once a day)
    1
        Wk 12|Tablet (taken once a day)
    1
        EOT|Tablet (taken once a day)
    4
        BL|Tablet (taken 3 times a day)
    0
        Wk 4|Tablet (taken 3 times a day)
    0
        Wk 12|Tablet (taken 3 times a day)
    0
        EOT|Tablet (taken 3 times a day)
    0
        BL|Tablet crushed
    11
        Wk 4|Tablet crushed
    14
        Wk 12|Tablet crushed
    13
        EOT|Tablet crushed
    9
        BL|Sprinkle powder on food
    14
        Wk 4|Sprinkle powder on food
    14
        Wk 12|Sprinkle powder on food
    12
        EOT|Sprinkle powder on food
    15
        BL|Injection
    0
        Wk 4|Injection
    0
        Wk 12|Injection
    0
        EOT|Injection
    0
        BL|I don't know
    3
        Wk 4|I don't know
    1
        Wk 12|I don't know
    0
        EOT|I don't know
    0
    No statistical analyses for this end point

    Secondary: Modified SICT in participants pre-treated with deferasirox: Number of Participants with Reasons child preferred crushed medicine scoring

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    End point title
    		 Modified SICT in participants pre-treated with deferasirox: Number of Participants with Reasons child preferred crushed medicine scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Participants
        BL|Taste
    6
        Wk 4|Taste
    0
        Wk 12|Taste
    0
        EOT|Taste
    0
        BL|Aftertaste
    5
        Wk 4|Aftertaste
    0
        Wk 12|Aftertaste
    0
        EOT|Aftertaste
    0
        BL|Convenience
    5
        Wk 4|Convenience
    4
        Wk 12|Convenience
    2
        EOT|Convenience
    0
        BL|Number of pills
    0
        Wk 4|Number of pills
    0
        Wk 12|Number of pills
    0
        EOT|Number of pills
    0
        BL|No/Less side effects
    0
        Wk 4|No/Less side effects
    0
        Wk 12|No/Less side effects
    0
        EOT|No/Less side effects
    0
        BL|Can correctly prepare the medicine
    0
        Wk 4|Can correctly prepare the medicine
    0
        Wk 12|Can correctly prepare the medicine
    0
        EOT|Can correctly prepare the medicine
    0
        BL|Easier to remember to take the medicine
    1
        Wk 4|Easier to remember to take the medicine
    0
        Wk 12|Easier to remember to take the medicine
    0
        EOT|Easier to remember to take the medicine
    0
        BL|No of times he/she has to take the medicine
    0
        Wk 4|No of times he/she has to take the medicine
    0
        Wk 12|No of times he/she has to take the medicine
    0
        EOT|No of times he/she has to take the medicine
    0
        BL|No/Less pain on the injection site
    0
        Wk 4|No/Less pain on the injection site
    0
        Wk 12|No/Less pain on the injection site
    0
        EOT|No/Less pain on the injection site
    0
        BL|Gain personal time with family&friends
    2
        Wk 4|Gain personal time with family&friends
    2
        Wk 12|Gain personal time with family&friends
    3
        EOT|Gain personal time with family&friends
    7
        BL|Other
    1
        Wk 4|Other
    8
        Wk 12|Other
    8
        EOT|Other
    3
    No statistical analyses for this end point

    Secondary: Modified SICT in participants pre-treated with deferasirox: Number of Participants with Rank based on child's preference scoring

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    End point title
    		 Modified SICT in participants pre-treated with deferasirox: Number of Participants with Rank based on child's preference scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count.
    End point type
    Secondary
    End point timeframe
    Baseline (BL), Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Participants
        BL -Tablet to dissolve in liquid|1
    0
        Week 4 -Tablet to dissolve in liquid|1
    0
        Week 12-Tablet to dissolve in liquid|1
    1
        EOT-Tablet to dissolve in liquid|1
    0
        BL-Tablet (taken once a day)|1
    6
        Week 4-Tablet (taken once a day)|1
    3
        Week 12-Tablet (taken once a day)|1
    1
        EOT-Tablet (taken once a day)|1
    4
        BL-Tablet (taken 3 times a day)|1
    0
        Week 4-Tablet (taken 3 times a day)|1
    0
        Week 12-Tablet (taken 3 times a day)|1
    0
        EOT-Tablet (taken 3 times a day)|1
    0
        BL-Tablet crushed|1
    12
        Week 4-Tablet crushed|1
    10
        Week 12-Tablet crushed|1
    8
        EOT-Tablet crushed|1
    8
        BL-Sprinkle powder on food|1
    16
        Week 4-Sprinkle powder on food|1
    19
        Week 12-Sprinkle powder on food|1
    15
        EOT-Sprinkle powder on food|1
    16
        BL-Injection|1
    0
        Week 4-Injection|1
    0
        Week 12-Injection|1
    1
        EOT-Injection|1
    0
        BL -Tablet to dissolve in liquid|2
    1
        Week 4 -Tablet to dissolve in liquid|2
    11
        Week 12-Tablet to dissolve in liquid|2
    7
        EOT-Tablet to dissolve in liquid|2
    8
        BL-Tablet (taken once a day)|2
    3
        Week 4-Tablet (taken once a day)|2
    0
        Week 12-Tablet (taken once a day)|2
    0
        EOT-Tablet (taken once a day)|2
    0
        BL-Tablet (taken 3 times a day)|2
    0
        Week 4-Tablet (taken 3 times a day)|2
    1
        Week 12-Tablet (taken 3 times a day)|2
    2
        EOT-Tablet (taken 3 times a day)|2
    1
        BL-Tablet crushed|2
    14
        Week 4-Tablet crushed|2
    9
        Week 12-Tablet crushed|2
    9
        EOT-Tablet crushed|2
    9
        BL-Sprinkle powder on food|2
    13
        Week 4-Sprinkle powder on food|2
    8
        Week 12-Sprinkle powder on food|2
    8
        EOT-Sprinkle powder on food|2
    10
        BL-Injection|2
    0
        Week 4-Injection|2
    0
        Week 12-Injection|2
    0
        EOT-Injection|2
    0
        BL -Tablet to dissolve in liquid|3
    12
        Week 4 -Tablet to dissolve in liquid|3
    8
        Week 12-Tablet to dissolve in liquid|3
    7
        EOT-Tablet to dissolve in liquid|3
    8
        BL-Tablet (taken once a day)|3
    15
        Week 4-Tablet (taken once a day)|3
    9
        Week 12-Tablet (taken once a day)|3
    11
        EOT-Tablet (taken once a day)|3
    11
        BL-Tablet (taken 3 times a day)|3
    0
        Week 4-Tablet (taken 3 times a day)|3
    1
        Week 12-Tablet (taken 3 times a day)|3
    1
        EOT-Tablet (taken 3 times a day)|3
    1
        BL-Tablet crushed|3
    3
        Week 4-Tablet crushed|3
    11
        Week 12-Tablet crushed|3
    6
        EOT-Tablet crushed|3
    8
        BL-Sprinkle powder on food|3
    1
        Week 4-Sprinkle powder on food|3
    0
        Week 12-Sprinkle powder on food|3
    1
        EOT-Sprinkle powder on food|3
    0
        BL-Injection|3
    0
        Week 4-Injection|3
    0
        Week 12-Injection|3
    0
        EOT-Injection|3
    0
        BL -Tablet to dissolve in liquid|4
    7
        Week 4 -Tablet to dissolve in liquid|4
    3
        Week 12-Tablet to dissolve in liquid|4
    4
        EOT-Tablet to dissolve in liquid|4
    7
        BL-Tablet (taken once a day)|4
    6
        Week 4-Tablet (taken once a day)|4
    18
        Week 12-Tablet (taken once a day)|4
    13
        EOT-Tablet (taken once a day)|4
    13
        BL-Tablet (taken 3 times a day)|4
    14
        Week 4-Tablet (taken 3 times a day)|4
    6
        Week 12-Tablet (taken 3 times a day)|4
    6
        EOT-Tablet (taken 3 times a day)|4
    3
        BL-Tablet crushed|4
    3
        Week 4-Tablet crushed|4
    0
        Week 12-Tablet crushed|4
    2
        EOT-Tablet crushed|4
    2
        BL-Sprinkle powder on food|4
    1
        Week 4-Sprinkle powder on food|4
    1
        Week 12-Sprinkle powder on food|4
    1
        EOT-Sprinkle powder on food|4
    2
        BL-Injection|4
    0
        Week 4-Injection|4
    1
        Week 12-Injection|4
    0
        EOT-Injection|4
    1
        BL -Tablet to dissolve in liquid|5
    11
        Week 4 -Tablet to dissolve in liquid|5
    5
        Week 12-Tablet to dissolve in liquid|5
    7
        EOT-Tablet to dissolve in liquid|5
    4
        BL-Tablet (taken once a day)|5
    2
        Week 4-Tablet (taken once a day)|5
    0
        Week 12-Tablet (taken once a day)|5
    1
        EOT-Tablet (taken once a day)|5
    0
        BL-Tablet (taken 3 times a day)|5
    17
        Week 4-Tablet (taken 3 times a day)|5
    21
        Week 12-Tablet (taken 3 times a day)|5
    17
        EOT-Tablet (taken 3 times a day)|5
    23
        BL-Tablet crushed|5
    0
        Week 4-Tablet crushed|5
    0
        Week 12-Tablet crushed|5
    0
        EOT-Tablet crushed|5
    1
        BL-Sprinkle powder on food|5
    1
        Week 4-Sprinkle powder on food|5
    2
        Week 12-Sprinkle powder on food|5
    1
        EOT-Sprinkle powder on food|5
    0
        BL-Injection|5
    0
        Week 4-Injection|5
    1
        Week 12-Injection|5
    0
        EOT-Injection|5
    0
        BL -Tablet to dissolve in liquid|6
    1
        Week 4 -Tablet to dissolve in liquid|6
    3
        Week 12-Tablet to dissolve in liquid|6
    0
        EOT-Tablet to dissolve in liquid|6
    1
        BL-Tablet (taken once a day)|6
    0
        Week 4-Tablet (taken once a day)|6
    0
        Week 12-Tablet (taken once a day)|6
    0
        EOT-Tablet (taken once a day)|6
    0
        BL-Tablet (taken 3 times a day)|6
    1
        Week 4-Tablet (taken 3 times a day)|6
    1
        Week 12-Tablet (taken 3 times a day)|6
    0
        EOT-Tablet (taken 3 times a day)|6
    0
        BL-Tablet crushed|6
    0
        Week 4-Tablet crushed|6
    0
        Week 12-Tablet crushed|6
    1
        EOT-Tablet crushed|6
    0
        BL-Sprinkle powder on food|6
    0
        Week 4-Sprinkle powder on food|6
    0
        Week 12-Sprinkle powder on food|6
    0
        EOT-Sprinkle powder on food|6
    0
        BL-Injection|6
    32
        Week 4-Injection|6
    28
        Week 12-Injection|6
    25
        EOT-Injection|6
    27
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in participants pre-treated with deferasirox: Mean Change from Baseline in Concerns

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in participants pre-treated with deferasirox: Mean Change from Baseline in Concerns
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child’s response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver’s responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Score
    arithmetic mean (confidence interval 95%)
        Child's perspective overall score-Week 4
    1.1 (0.6 to 1.7)
        Child's perspective overall score-Week 12
    1.2 (0.5 to 1.9)
        Child's perspective overall score-EOT
    0.8 (0.0 to 1.5)
        Caregiver's perspective overall score-Week 4
    0.4 (-0.1 to 0.8)
        Caregiver's perspective overall score-Week 12
    0.4 (-0.2 to 0.9)
        Caregiver's perspective overall score-EOT
    0.2 (-0.3 to 0.7)
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Mean Change from Baseline in Adherence

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Mean Change from Baseline in Adherence
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver’s perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Score
    arithmetic mean (standard deviation)
        Child's perspective overall score-Week 4
    10.4 ( 2.98 )
        Child's perspective overall score-Week 12
    9.8 ( 2.99 )
        Child's perspective overall score-EOT
    10.3 ( 3.43 )
        Caregiver's perspective overall score-Week 4
    9.0 ( 3.97 )
        Caregiver's perspective overall score-Week 12
    9.9 ( 5.30 )
        Caregiver's perspective overall score-EOT
    10.6 ( 5.03 )
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Type of Medicine child like scoring

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Type of Medicine child like scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Participants
        Wk 4|Tablet to dissolve in liquid
    0
        Wk 12|Tablet to dissolve in liquid
    1
        EOT|Tablet to dissolve in liquid
    4
        Wk 4|Tablet (taken once a day)
    1
        Wk 12|Tablet (taken once a day)
    0
        EOT|Tablet (taken once a day)
    0
        Wk 4|Tablet (taken 3 times a day)
    0
        Wk 12|Tablet (taken 3 times a day)
    0
        EOT|Tablet (taken 3 times a day)
    0
        Wk 4|Tablet crushed
    5
        Wk 12|Tablet crushed
    5
        EOT|Tablet crushed
    3
        Wk 4|Sprinkle powder on food
    3
        Wk 12|Sprinkle powder on food
    3
        EOT|Sprinkle powder on food
    2
        Wk 4|Injection
    0
        Wk 12|Injection
    0
        EOT|Injection
    0
        Wk 4|I don't know
    2
        Wk 12|I don't know
    0
        EOT|I don't know
    0
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Reasons child preferred crushed medicine scoring

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Reasons child preferred crushed medicine scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Participants
        Wk 4|Taste
    0
        Wk 12|Taste
    0
        EOT|Taste
    0
        Wk 4|Aftertaste
    1
        Wk 12|Aftertaste
    0
        EOT|Aftertaste
    0
        Wk 4|Convenience
    2
        Wk 12|Convenience
    2
        EOT|Convenience
    1
        Wk 4|Number of pills
    1
        Wk 12|Number of pills
    0
        EOT|Number of pills
    0
        Wk 4|No/Less side effects
    0
        Wk 12|No/Less side effects
    0
        EOT|No/Less side effects
    1
        Wk 4|Can correctly prepare the medicine
    1
        Wk 12|Can correctly prepare the medicine
    0
        EOT|Can correctly prepare the medicine
    1
        Wk 4|Easier to remember to take the medicine
    1
        Wk 12|Easier to remember to take the medicine
    0
        EOT|Easier to remember to take the medicine
    2
        Wk 4|No of times he/she has to take the medicine
    1
        Wk 12|No of times he/she has to take the medicine
    0
        EOT|No of times he/she has to take the medicine
    0
        Wk 4|No/Less pain on the injection site
    0
        Wk 12|No/Less pain on the injection site
    1
        EOT|No/Less pain on the injection site
    1
        Wk 4|Gain personal time with family&friends
    1
        Wk 12|Gain personal time with family&friends
    2
        EOT|Gain personal time with family&friends
    2
        Wk 4|Other
    1
        Wk 12|Other
    1
        EOT|Other
    0
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Rank based on child's preference scoring

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Number of Participants with Rank based on child's preference scoring
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Participants
        Wk 4 -Tablet to dissolve in liquid|1
    2
        Wk 12-Tablet to dissolve in liquid|1
    2
        EOT-Tablet to dissolve in liquid|1
    4
        Wk 4-Tablet (taken once a day)|1
    1
        Wk 12-Tablet (taken once a day)|1
    0
        EOT-Tablet (taken once a day)|1
    0
        Wk 4-Tablet (taken 3 times a day)|1
    1
        Wk 12-Tablet (taken 3 times a day)|1
    0
        EOT-Tablet (taken 3 times a day)|1
    0
        Wk 4-Tablet crushed|1
    1
        Wk 12-Tablet crushed|1
    1
        EOT-Tablet crushed|1
    1
        Wk 4-Sprinkle powder on food|1
    5
        Wk 12-Sprinkle powder on food|1
    5
        EOT-Sprinkle powder on food|1
    4
        Wk 4-Injection|1
    0
        Wk 12-Injection|1
    1
        EOT-Injection|1
    0
        Wk 4 -Tablet to dissolve in liquid|2
    1
        Wk 12-Tablet to dissolve in liquid|2
    3
        EOT-Tablet to dissolve in liquid|2
    2
        Wk 4-Tablet (taken once a day)|2
    3
        Wk 12-Tablet (taken once a day)|2
    0
        EOT-Tablet (taken once a day)|2
    1
        Wk 4-Tablet (taken 3 times a day)|2
    0
        Wk 12-Tablet (taken 3 times a day)|2
    1
        EOT-Tablet (taken 3 times a day)|2
    0
        Wk 4-Tablet crushed|2
    6
        Wk 12-Tablet crushed|2
    5
        EOT-Tablet crushed|2
    4
        Wk 4-Sprinkle powder on food|2
    0
        Wk 12-Sprinkle powder on food|2
    0
        EOT-Sprinkle powder on food|2
    2
        Wk 4-Injection|2
    0
        Wk 12-Injection|2
    0
        EOT-Injection|2
    0
        Wk 4 -Tablet to dissolve in liquid|3
    2
        Wk 12-Tablet to dissolve in liquid|3
    2
        EOT-Tablet to dissolve in liquid|3
    1
        Wk 4-Tablet (taken once a day)|3
    5
        Wk 12-Tablet (taken once a day)|3
    2
        EOT-Tablet (taken once a day)|3
    3
        Wk 4-Tablet (taken 3 times a day)|3
    1
        Wk 12-Tablet (taken 3 times a day)|3
    2
        EOT-Tablet (taken 3 times a day)|3
    0
        Wk 4-Tablet crushed|3
    1
        Wk 12-Tablet crushed|3
    2
        EOT-Tablet crushed|3
    4
        Wk 4-Sprinkle powder on food|3
    0
        Wk 12-Sprinkle powder on food|3
    1
        EOT-Sprinkle powder on food|3
    1
        Wk 4-Injection|3
    1
        Wk 12-Injection|3
    0
        EOT-Injection|3
    0
        Wk 4 -Tablet to dissolve in liquid|4
    4
        Wk 12-Tablet to dissolve in liquid|4
    0
        EOT-Tablet to dissolve in liquid|4
    2
        Wk 4-Tablet (taken once a day)|4
    1
        Wk 12-Tablet (taken once a day)|4
    6
        EOT-Tablet (taken once a day)|4
    5
        Wk 4-Tablet (taken 3 times a day)|4
    3
        Wk 12-Tablet (taken 3 times a day)|4
    1
        EOT-Tablet (taken 3 times a day)|4
    0
        Wk 4-Tablet crushed|4
    1
        Wk 12-Tablet crushed|4
    0
        EOT-Tablet crushed|4
    0
        Wk 4-Sprinkle powder on food|4
    1
        Wk 12-Sprinkle powder on food|4
    2
        EOT-Sprinkle powder on food|4
    2
        Wk 4-Injection|4
    0
        Wk 12-Injection|4
    0
        EOT-Injection|4
    0
        Wk 4 -Tablet to dissolve in liquid|5
    1
        Wk 12-Tablet to dissolve in liquid|5
    2
        EOT-Tablet to dissolve in liquid|5
    0
        Wk 4-Tablet (taken once a day)|5
    0
        Wk 12-Tablet (taken once a day)|5
    1
        EOT-Tablet (taken once a day)|5
    0
        Wk 4-Tablet (taken 3 times a day)|5
    4
        Wk 12-Tablet (taken 3 times a day)|5
    5
        EOT-Tablet (taken 3 times a day)|5
    9
        Wk 4-Tablet crushed|5
    1
        Wk 12-Tablet crushed|5
    0
        EOT-Tablet crushed|5
    0
        Wk 4-Sprinkle powder on food|5
    3
        Wk 12-Sprinkle powder on food|5
    1
        EOT-Sprinkle powder on food|5
    0
        Wk 4-Injection|5
    1
        Wk 12-Injection|5
    0
        EOT-Injection|5
    0
        Wk 4 -Tablet to dissolve in liquid|6
    0
        Wk 12-Tablet to dissolve in liquid|6
    0
        EOT-Tablet to dissolve in liquid|6
    0
        Wk 4-Tablet (taken once a day)|6
    0
        Wk 12-Tablet (taken once a day)|6
    0
        EOT-Tablet (taken once a day)|6
    0
        Wk 4-Tablet (taken 3 times a day)|6
    1
        Wk 12-Tablet (taken 3 times a day)|6
    0
        EOT-Tablet (taken 3 times a day)|6
    0
        Wk 4-Tablet crushed|6
    0
        Wk 12-Tablet crushed|6
    1
        EOT-Tablet crushed|6
    0
        Wk 4-Sprinkle powder on food|6
    1
        Wk 12-Sprinkle powder on food|6
    0
        EOT-Sprinkle powder on food|6
    0
        Wk 4-Injection|6
    8
        Wk 12-Injection|6
    8
        EOT-Injection|6
    9
    No statistical analyses for this end point

    Secondary: Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Mean Change from Baseline in Concerns

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    End point title
    		 Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation naive participants: Mean Change from Baseline in Concerns
    End point description
    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child’s response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver’s responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Score
    arithmetic mean (standard deviation)
        Child's perspective overall score-Week 4
    9.7 ( 0.65 )
        Child's perspective overall score-Week 12
    9.4 ( 0.88 )
        Child's perspective overall score-EOT
    8.9 ( 1.36 )
        Caregiver's perspective overall score-Week 4
    3.7 ( 1.42 )
        Caregiver's perspective overall score-Week 12
    3.9 ( 1.76 )
        Caregiver's perspective overall score-EOT
    3.4 ( 1.59 )
    No statistical analyses for this end point

    Secondary: Palatability score in Chelation naive participants

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    End point title
    		 Palatability score in Chelation naive participants
    End point description
    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Score
    arithmetic mean (standard deviation)
        Week 4
    10.4 ( 0.97 )
        Week 12
    10.9 ( 0.33 )
        EOT (Week 24)
    10.8 ( 0.67 )
    No statistical analyses for this end point

    Secondary: Number of chelation naive Participants with Palatability After taste item scoring

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    End point title
    		 Number of chelation naive Participants with Palatability After taste item scoring
    End point description
    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Participants
        Wk 4|Very Good
    3
        Wk 12|Very Good
    2
        EOT|Very Good
    1
        Wk 4|Good
    4
        Wk 12|Good
    5
        EOT|Good
    4
        Wk 4|Neither good nor bad
    3
        Wk 12|Neither good nor bad
    2
        EOT|Neither good nor bad
    3
        Wk 4|Bad
    1
        Wk 12|Bad
    0
        EOT|Bad
    1
        Wk 4|Very bad
    0
        Wk 12|Very bad
    0
        EOT|Very bad
    0
    No statistical analyses for this end point

    Secondary: Palatability score in participants pre-treated with deferasirox

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    End point title
    		 Palatability score in participants pre-treated with deferasirox
    End point description
    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
    End point type
    Secondary
    End point timeframe
    Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Score
    arithmetic mean (standard deviation)
        Week 4
    10.7 ( 1.29 )
        Week 12
    10.3 ( 2.13 )
        EOT (Week 24)
    10.6 ( 1.57 )
    No statistical analyses for this end point

    Secondary: Number of Participants pre-treated with deferasirox with Palatability After taste item scoring

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    End point title
    		 Number of Participants pre-treated with deferasirox with Palatability After taste item scoring
    End point description
    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4, Week 12, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Participants
        BL|Very Good
    5
        Wk 4|Very Good
    1
        Wk 12|Very Good
    4
        EOT|Very Good
    0
        BL|Good
    5
        Wk 4|Good
    13
        Wk 12|Good
    9
        EOT|Good
    11
        BL|Neither good nor bad
    15
        Wk 4|Neither good nor bad
    16
        Wk 12|Neither good nor bad
    12
        EOT|Neither good nor bad
    14
        BL|Bad
    7
        Wk 4|Bad
    0
        Wk 12|Bad
    2
        EOT|Bad
    3
        BL|Very bad
    0
        Wk 4|Very bad
    0
        Wk 12|Very bad
    0
        EOT|Very bad
    0
    No statistical analyses for this end point

    Secondary: GI Symptom Score in Chelation naive participants

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    End point title
    		 GI Symptom Score in Chelation naive participants
    End point description
    The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
    End point type
    Secondary
    End point timeframe
    Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Score
    arithmetic mean (standard deviation)
        Week 2
    6.8 ( 2.82 )
        Week 3
    7.1 ( 2.85 )
        Week 4
    7.0 ( 3.52 )
        Week 8
    6.8 ( 2.64 )
        Week 12
    6.2 ( 1.79 )
        Week 16
    7.0 ( 2.18 )
        Week 20
    5.9 ( 1.29 )
        EOT (Week 24)
    6.8 ( 1.92 )
    No statistical analyses for this end point

    Secondary: Number of Participants with GI Bowel Movements item scoring in Chelation naive participants

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    End point title
    		 Number of Participants with GI Bowel Movements item scoring in Chelation naive participants
    End point description
    The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 (“None”), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = “5 – 10” and 6 = “11 or more”. The GI bowel movements item score was presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    12
    Units: Participants
        Week 2|3
    1
        Week 3|3
    1
        Week 4|3
    0
        Week 8|3
    0
        Week 12|3
    0
        Week 16|3
    0
        Week 20|3
    0
        EOT (Week 24)|3
    2
        Week 2|4
    1
        Week 3|4
    0
        Week 4|4
    1
        Week 8|4
    3
        Week 12|4
    2
        Week 16|4
    2
        Week 20|4
    1
        EOT (Week 24)|4
    0
        Week 2|5 to 10
    8
        Week 3|5 to 10
    7
        Week 4|5 to 10
    8
        Week 8|5 to 10
    6
        Week 12|5 to 10
    6
        Week 16|5 to 10
    5
        Week 20|5 to 10
    7
        EOT (Week 24)|5 to 10
    6
        Week 2|11 or more
    2
        Week 3|11 or more
    2
        Week 4|11 or more
    2
        Week 8|11 or more
    2
        Week 12|11 or more
    1
        Week 16|11 or more
    2
        Week 20|11 or more
    2
        EOT (Week 24)|11 or more
    1
    No statistical analyses for this end point

    Secondary: GI Symptom Score in Participants pre-treated with deferasirox

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    End point title
    		 GI Symptom Score in Participants pre-treated with deferasirox
    End point description
    The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Score
    arithmetic mean (standard deviation)
        Week 2
    6.3 ( 1.91 )
        Week 3
    6.0 ( 1.63 )
        Week 4
    6.4 ( 1.81 )
        Week 8
    6.4 ( 2.34 )
        Week 12
    6.5 ( 1.83 )
        Week 16
    6.7 ( 2.10 )
        Week 20
    7.3 ( 2.37 )
        EOT (Week 24)
    7.6 ( 2.95 )
    No statistical analyses for this end point

    Secondary: Number of Participants with GI Bowel Movements item scoring in Participants pre-treated with deferasirox

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    End point title
    		 Number of Participants with GI Bowel Movements item scoring in Participants pre-treated with deferasirox
    End point description
    The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 (“None”), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = “5 – 10” and 6 = “11 or more”. The GI bowel movements item score was presented descriptively using frequency counts.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
    End point values
    		 Deferasirox
    Number of subjects analysed
    32
    Units: Participants
        Baseline (BL)|3
    0
        Week 2|3
    0
        Week 3|3
    0
        Week 4|3
    0
        Week 8|3
    1
        Week 12|3
    1
        Week 16|3
    0
        Week 20|3
    0
        EOT (Week 24)|3
    0
        Baseline (BL)|4
    1
        Week 2|4
    1
        Week 3|4
    1
        Week 4|4
    1
        Week 8|4
    1
        Week 12|4
    0
        Week 16|4
    1
        Week 20|4
    1
        EOT (Week 24)|4
    1
        Baseline (BL)|5 to 10
    30
        Week 2|5 to 10
    29
        Week 3|5 to 10
    26
        Week 4|5 to 10
    28
        Week 8|5 to 10
    29
        Week 12|5 to 10
    26
        Week 16|5 to 10
    29
        Week 20|5 to 10
    28
        EOT (Week 24)|5 to 10
    25
        Baseline (BL)|11 or more
    1
        Week 2|11 or more
    2
        Week 3|11 or more
    1
        Week 4|11 or more
    1
        Week 8|11 or more
    1
        Week 12|11 or more
    0
        Week 16|11 or more
    1
        Week 20|11 or more
    2
        EOT (Week 24)|11 or more
    2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment-emergent adverse events (TEAEs) were collected from first dose of study treatment until end of study treatment plus 30 days post-treatment, assessed for approximately 28 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    All Patients
    Reporting group description
    		 All Patients

    Serious adverse events
    		 All Patients
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 44 (4.55%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 44 (2.27%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 44 (2.27%)
         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 Patients
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    29 / 44 (65.91%)
    Investigations
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    12 / 44 (27.27%)
         occurrences all number
    14
    Blood creatinine increased
         subjects affected / exposed
    9 / 44 (20.45%)
         occurrences all number
    12
    Alanine aminotransferase increased
         subjects affected / exposed
    7 / 44 (15.91%)
         occurrences all number
    11
    Bilirubin conjugated increased
         subjects affected / exposed
    6 / 44 (13.64%)
         occurrences all number
    6
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    5 / 44 (11.36%)
         occurrences all number
    5
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 44 (9.09%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    6 / 44 (13.64%)
         occurrences all number
    8
    Rhinorrhoea
         subjects affected / exposed
    3 / 44 (6.82%)
         occurrences all number
    5
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    4 / 44 (9.09%)
         occurrences all number
    5
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 44 (22.73%)
         occurrences all number
    13

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Apr 2018
    Deferasirox FCT 90 mg, 180 mg, and 360 mg (Deferasirox FCT), was commercially available in some participating countries but not in all countries. The purpose of this amendment was to enable Novartis Drug Supply Management to supply labeled study drug to countries where Deferasirox FCT was not commercially available and also to enable countries where Deferasirox FCT was commercially available to follow their local processes for using commercial products in clinical studies. Two guidances were provided related to collection of Observer Reported Outcomes (ObsROs). In addition, some updates were made to provide further clarification on some aspects of the study.

    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
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