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    Clinical Trial Results:
    Open-label, multicenter, single arm, phase II study assessing treatment patient preference for new deferasirox formulation (film-coated tablet) compared to the reference deferasirox dispersible tablet formulation

    Summary
    EudraCT number
    		 2016-002282-61
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    11 Mar 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 Sep 2021
    First version publication date
    22 Sep 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670FIC05
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02993224
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    11 Mar 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Mar 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective was to evaluate patient preference between deferasirox film-coated tablets (FCTs) and deferasirox dispersible tablets (DTs)
    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
    27 Jul 2017
    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
    Turkey: 10
    Country: Number of subjects enrolled
    Egypt: 45
    Country: Number of subjects enrolled
    Lebanon: 5
    Country: Number of subjects enrolled
    Saudi Arabia: 17
    Country: Number of subjects enrolled
    Morocco: 8
    Country: Number of subjects enrolled
    Thailand: 51
    Country: Number of subjects enrolled
    Vietnam: 12
    Worldwide total number of subjects
    148
    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)
    0
    Children (2-11 years)
    73
    Adolescents (12-17 years)
    20
    Adults (18-64 years)
    54
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Participants took part in 17 investigative sites in 7 countries

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

    Arms
    Arm title
    		 Deferasirox DT followed by deferasirox FCT
    Arm description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Pharmaceutical forms
    Dispersible tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country. For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT. For iron chelation (deferoxamine and/or deferiprone) participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone). Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water.

    Number of subjects in period 1
    		Deferasirox DT followed by deferasirox FCT
    Started
    148
    Completed
    140
    Not completed
    8
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    4
         Lost to follow-up
    3
    Period 2
    Period 2 title
    Core Phase- Period 2
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Deferasirox DT followed by deferasirox FCT
    Arm description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT. Participants took deferasirox FCT once daily for 24 weeks during the core phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).

    Number of subjects in period 2
    		Deferasirox DT followed by deferasirox FCT
    Started
    140
    Completed
    136
    Not completed
    4
         Adverse event, non-fatal
    1
         Unwillingness To Comply With Protocol Procedures
    2
         Allogenic stem cell transplantation
    1
    Period 3
    Period 3 title
    Extension Phase
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Deferasirox DT followed by deferasirox FCT
    Arm description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. Participants took deferasirox FCT once daily up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).

    Number of subjects in period 3 [1]
    		Deferasirox DT followed by deferasirox FCT
    Started
    116
    Completed
    80
    Not completed
    36
         Consent withdrawn by subject
    3
         Adverse event, non-fatal
    3
         Unwillingness To Comply With Protocol Procedures
    1
         Deferasirox FCT locally reimbursed
    27
         Lost to follow-up
    2
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Not all participants who completed the core phase started the extension phase (optional)

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deferasirox DT followed by deferasirox FCT
    Reporting group description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT

    Reporting group values
    		 Deferasirox DT followed by deferasirox FCT Total
    Number of subjects
    		 148 148
    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)
    		 73 73
        Adolescents (12-17 years)
    		 20 20
        Adults (18-64 years)
    		 54 54
        From 65-84 years
    		 1 1
        85 years and over
    		 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 15.32 ± 13.824 -
    Sex: Female, Male
    Units: Participants
        Female
    		 82 82
        Male
    		 66 66
    Race/Ethnicity, Customized
    Units: Subjects
        Indian (Indian subcontinent)
    		 5 5
        Other
    		 143 143

    End points

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    End points reporting groups
    Reporting group title
    Deferasirox DT followed by deferasirox FCT
    Reporting group description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
    Reporting group title
    Deferasirox DT followed by deferasirox FCT
    Reporting group description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
    Reporting group title
    Deferasirox DT followed by deferasirox FCT
    Reporting group description
    		 Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT

    Subject analysis set title
    Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants to whom study treatment was assigned and who received at least one dose of each study treatment (deferasirox DT and FCT).

    Primary: Number of participants preferring deferasirox FCT or DT at Week 48 based on preference questionnaire (item 2)

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    End point title
    		 Number of participants preferring deferasirox FCT or DT at Week 48 based on preference questionnaire (item 2) [1]
    End point description
    Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient “like best”: “Tablet to dissolve in liquid” (=deferasirox DT), “Film coated tablet” (=deferasirox FCT), “Sprinkle powder on food” (=deferasirox FCT) and “I don’t know” (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.
    End point type
    Primary
    End point timeframe
    Week 48
    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: Due to EudraCT system limitations, no statistical analyses have been specified for the endpoints, as this is a single arm study and comparisons within an arm are not supported by the system
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    134
    Units: Participants
        Preference for deferasirox DT
    10
        Preference for deferasirox FCT
    121
        Preference for none of the above
    3
    No statistical analyses for this end point

    Secondary: Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy at Week 28 based on preference questionnaire (item 2)

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    End point title
    		 Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy at Week 28 based on preference questionnaire (item 2)
    End point description
    Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient “like best”: “Tablet to dissolve in liquid” (=deferasirox DT), “Film coated tablet (taken once a day)” (=deferasirox FCT), “Sprinkle powder on food” (=deferasirox FCT), “Tablet (taken 3 times a day)” (=previous iron chelation therapy), “Injection” (=previous iron chelation therapy) and “I don’t know” (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.
    End point type
    Secondary
    End point timeframe
    Week 28
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    69
    Units: Participants
        Preference for deferasirox DT
    6
        Preference for deferasirox FCT
    60
        Preference for iron chelation therapy
    3
    No statistical analyses for this end point

    Secondary: Number of participants preferring deferasirox DT or previous iron chelation therapy at Week 4 and Week 24 based on preference questionnaire (item 2)

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    End point title
    		 Number of participants preferring deferasirox DT or previous iron chelation therapy at Week 4 and Week 24 based on preference questionnaire (item 2)
    End point description
    Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient “like best”: “Tablet to dissolve in liquid” (=deferasirox DT), “Tablet (taken 3 times a day)” (=previous iron chelation therapy), “Injection” (=previous iron chelation therapy) and “I don’t know” (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire
    End point type
    Secondary
    End point timeframe
    Week 4 and Week 24
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    70
    Units: Participants
        Preference for deferasirox DT (Week 4)
    57
        Preference for iron chelation therapy (Week 4)
    11
        Preference for none of the above (Week 4)
    2
        Preference for deferasirox DT (Week 24)
    52
        Preference for iron chelation therapy (Week 24)
    11
        Preference for none of the above (Week 24)
    6
    No statistical analyses for this end point

    Secondary: Number of participants selecting each reason for treatment preference as assessed by the preference questionnaire at Week 28 and Week 48

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    End point title
    		 Number of participants selecting each reason for treatment preference as assessed by the preference questionnaire at Week 28 and Week 48
    End point description
    The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient “Like best”. Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.
    End point type
    Secondary
    End point timeframe
    Week 28 and Week 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Participants
        Aftertaste (Week 28)
    9
        Can correctly prepare the medicine (Week 28)
    35
        Convenience (Week 28)
    109
        Easier to remember to take the medicine (Week 28)
    57
        Gain personal time with family/friends (Week 28)
    35
        No/ less pain on the injection site (Week 28)
    31
        No/ less side effects (Week 28)
    50
        Number of pills (Week 28)
    46
        Number of times to take the medicine (Week 28)
    42
        Other (Week 28)
    5
        Taste (Week 28)
    30
        Aftertaste (Week 48)
    17
        Can correctly prepare the medicine (Week 48)
    48
        Convenience (Week 48)
    103
        Easier to remember to take the medicine (Week 48)
    61
        Gain personal time with family/friends (Week 48)
    36
        No/ less pain on the injection site (Week 48)
    37
        No/ less side effects (Week 48)
    48
        Number of pills (Week 48)
    41
        Number of times to take the medicine (Week 48)
    47
        Other (Week 48)
    0
        Taste (Week 48)
    51
    No statistical analyses for this end point

    Secondary: Percentage of consumed tablet counts during deferasirox DT and deferasirox FCT treatment periods

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    End point title
    		 Percentage of consumed tablet counts during deferasirox DT and deferasirox FCT treatment periods
    End point description
    The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.
    End point type
    Secondary
    End point timeframe
    Deferasirox DT: Baseline Day 1 to Week 24. Deferasirox FCT: Week 25 to Week 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Percentage of tablet counts
    arithmetic mean (standard deviation)
        Deferasirox DT (Baseline to Week 24) n=139
    98.68 ± 22.373
        Deferasirox FCT (Week 25 to Week 48) n=138
    95.07 ± 15.368
    No statistical analyses for this end point

    Secondary: Change over time in aftertaste score of palatability questionnaire

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    End point title
    		 Change over time in aftertaste score of palatability questionnaire
    End point description
    The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of “no aftertaste”. The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste. For participants less than (<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than (≥) 10 years old. Change in aftertaste score over time was assessed. Note: 9999 indicates value is not applicable.
    End point type
    Secondary
    End point timeframe
    Week 4, 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Week 4 - 24: treated with deferasirox DT (n=102)
    -0.1 ± 0.80
        Week 4 - 24: treated with deferasirox FCT (n=6)
    -0.5 ± 1.22
        Week 24 - 28: treated with deferasirox DT (n=0)
    9999 ± 9999
        Week 24 - 28: treated with deferasirox FCT (n=106)
    -0.5 ± 1.16
        Week 24 - 48: treated with deferasirox DT (n=0)
    9999 ± 9999
        Week 24 - 48: treated with deferasirox FCT (n=98)
    -0.5 ± 1.08
    No statistical analyses for this end point

    Secondary: Change over time in palatability score of palatability questionnaire

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    End point title
    		 Change over time in palatability score of palatability questionnaire
    End point description
    The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. For participants <10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed. Note: 9999 indicates value is not applicable
    End point type
    Secondary
    End point timeframe
    Week 4, 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Week 4 - 24: treated with deferasirox DT (n=131)
    -0.1 ± 2.46
        Week 4 - 24: treated with deferasirox FCT (n=7)
    0.0 ± 0.00
        Week 24 - 28: treated with deferasirox DT (n=0)
    9999 ± 9999
        Week 24 - 28: treated with deferasirox FCT (n=137)
    1.1 ± 2.77
        Week 24 - 48: treated with deferasirox DT (n=0)
    9999 ± 9999
        Week 24 - 48: treated with deferasirox FCT (n=134)
    1.3 ± 2.54
    No statistical analyses for this end point

    Secondary: Change from baseline in adherence domain score of modified satisfaction with iron chelation (mSICT) questionnaire

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    End point title
    		 Change from baseline in adherence domain score of modified satisfaction with iron chelation (mSICT) questionnaire
    End point description
    The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence. For participants <10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old. Note: 9999 indicates value is not applicable
    End point type
    Secondary
    End point timeframe
    Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Week 24-treated with deferasirox DT (PRO) (n=72)
    -0.3 ± 3.37
        Week 24 -treated with deferasirox FCT (PRO) (n=4)
    -1.0 ± 2.94
        Week 28 -treated with deferasirox DT (PRO) (n=0)
    9999 ± 9999
        Week 28 -treated with deferasirox FCT (PRO) (n=77)
    0.5 ± 3.59
        Week 48 -treated with deferasirox DT (PRO) (n=0)
    9999 ± 9999
        Week 48 -treated with deferasirox FCT (PRO) (n=75)
    0.5 ± 3.29
        Week 24-treated with deferasirox DT(ObsRO)(n=56)
    -0.2 ± 3.35
        Week 24-treated with deferasirox FCT(ObsRO)(n=3)
    -0.7 ± 4.16
        Week 28-treated with deferasirox DT (ObsRO)(n=0)
    9999 ± 9999
        Week 28-treated with deferasirox FCT(ObsRO)(n=58)
    0.8 ± 3.68
        Week 48-treated with deferasirox DT(ObsRO)(n=0)
    9999 ± 9999
        Week 48-treated with deferasirox FCT(ObsRO)(n=55)
    1.1 ± 3.79
    No statistical analyses for this end point

    Secondary: Change from baseline in preference domain score of modified satisfaction with iron chelation (mSICT) questionnaire

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    End point title
    		 Change from baseline in preference domain score of modified satisfaction with iron chelation (mSICT) questionnaire
    End point description
    The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction. For participants < 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old. Note: 9999 indicates value is not applicable.
    End point type
    Secondary
    End point timeframe
    Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Week 24-treated with deferasirox DT(PRO)(n=72)
    0.5 ± 1.21
        Week 24-treated with deferasirox FCT(PRO)(n=4)
    0.5 ± 3.70
        Week 28-treated with deferasirox DT(PRO)(n=0)
    9999 ± 9999
        Week 28-treated with deferasirox FCT(PRO)(n=77)
    -1.1 ± 1.83
        Week 48-treated with deferasirox DT(PRO)(n=0)
    9999 ± 9999
        Week 48-treated with deferasirox FCT(PRO)(n=75)
    -0.9 ± 2.02
        Week 24-treated with deferasirox DT(ObsRO)(n=55)
    0.0 ± 1.49
        Week 24-treated with deferasirox FCT(ObsRO)(n=3)
    0.0 ± 1.73
        Week 28-treated with deferasirox DT(ObsRO)(n=0)
    9999 ± 9999
        Week 28-treated with deferasirox FCT(ObsRO)(n=57)
    -0.8 ± 1.74
        Week 48-treated with deferasirox DT(ObsRO)(n=0)
    9999 ± 9999
        Week 48-treated with deferasirox FCT(ObsRO)(n=54)
    -0.9 ± 1.75
    No statistical analyses for this end point

    Secondary: Change from baseline in concerns domain score of modified satisfaction with iron chelation (mSICT) questionnaire

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    End point title
    		 Change from baseline in concerns domain score of modified satisfaction with iron chelation (mSICT) questionnaire
    End point description
    The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants < 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old. Note: 9999 indicates value is not applicable
    End point type
    Secondary
    End point timeframe
    Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Week 24-treated with deferasirox DT(PRO)(n=72)
    -0.1 ± 1.99
        Week 24-treated with deferasirox FCT(PRO)(n=4)
    -1.0 ± 1.15
        Week 24-treated with deferasirox DT (PRO)(n=0)
    9999 ± 9999
        Week 28-treated with deferasirox FCT(PRO)(n=77)
    0.3 ± 1.84
        Week 48-treated with deferasirox DT (PRO)(n=0)
    9999 ± 9999
        Week 48-treated with deferasirox FCT(PRO)(n=75)
    0.5 ± 1.80
        Week 24-treated with deferasirox DT (ObsRO)(n=55)
    -0.4 ± 1.33
        Week 24-treated with deferasirox FCT(ObsRO)(n=3)
    -0.7 ± 1.15
        Week 28-treated with deferasirox DT (ObsRO)(n=0)
    9999 ± 9999
        Week 28-treated with deferasirox FCT(ObsRO)(n=58)
    0.1 ± 2.05
        Week 48-treated with deferasirox DT(ObsRO)(n=0)
    9999 ± 9999
        Week 48-treated with deferasirox FCT(ObsRO)(n=54)
    0.1 ± 1.58
    No statistical analyses for this end point

    Secondary: Change from baseline in gastrointestinal (GI) symptom score based on GI questionnaire

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    End point title
    		 Change from baseline in gastrointestinal (GI) symptom score based on GI questionnaire
    End point description
    The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, 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. An observer GI symptom questionnaire was administered to those patients who were < 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old. Note: 9999 indicates value is not applicable
    End point type
    Secondary
    End point timeframe
    Baseline (week -1 or, if missing, week -2), week 24, 28 and 48
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Week 24 <10yrs treated with deferasirox DT(n=60)
    0.2 ± 2.78
        Week 24 <10yrs treated with deferasirox FCT(n=3)
    -2.0 ± 2.65
        Week 28 <10yrs treated with deferasirox DT(n=0)
    9999 ± 9999
        Week 28 <10yrs treated with deferasirox FCT(n=62)
    -0.5 ± 2.49
        Week 48 <10yrs treated with deferasirox DT(n=0)
    9999 ± 9999
        Week 48 <10yrs treated with deferasirox FCT(n=61)
    -0.6 ± 2.33
        Week 24 ≥10yrs treated with deferasirox DT(n=71)
    1.4 ± 5.53
        Week 24 ≥10yrs treated with deferasirox FCT(n=4)
    5.8 ± 12.82
        Week 28 ≥10yrs treated with deferasirox DT(n=0)
    9999 ± 9999
        Week 28 ≥10yrs treated with deferasirox FCT(n=75)
    -1.9 ± 4.33
        Week 48 ≥10yrs treated with deferasirox DT(n=0)
    9999 ± 9999
        Week 48 ≥10yrs treated with deferasirox FCT(n=75)
    -2.2 ± 4.74
    No statistical analyses for this end point

    Secondary: Change from baseline in serum ferritin levels

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    End point title
    		 Change from baseline in serum ferritin levels
    End point description
    Absolute change from baseline over time in serum ferritin levels
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) up to 96 weeks
    End point values
    		 Deferasirox DT followed by deferasirox FCT (Full Analysis Set)
    Number of subjects analysed
    139
    Units: microgram/liter (ug/L)
    arithmetic mean (standard deviation)
        Week 2 - treated with deferasirox DT(n=108)
    363.654 ± 323.3828
        Week 4 - treated with deferasirox DT (n=120)
    393.126 ± 457.8032
        Week 8 - treated with deferasirox DT (n=121)
    462.560 ± 427.7138
        Week 12 - treated with deferasirox DT (n=129)
    510.455 ± 548.6222
        Week 16 - treated with deferasirox DT (n=131)
    519.038 ± 508.7432
        Week 20 - treated with deferasirox DT (n=134)
    540.618 ± 548.2318
        Week 24 - treated with deferasirox DT (n=130)
    644.849 ± 629.6687
        Week 28 - treated with deferasirox FCT (n=114)
    679.610 ± 701.6219
        Week 32 - treated with deferasirox FCT (n=126)
    623.600 ± 629.5417
        Week 36 - treated with deferasirox FCT (n=127)
    679.011 ± 674.6716
        Week 40 - treated with deferasirox FCT (n=120)
    739.016 ± 659.0840
        Week 44 - treated with deferasirox FCT (n=120)
    748.063 ± 647.3000
        Week 48 - treated with deferasirox FCT (n=120)
    833.700 ± 788.0650
        Week 52 - treated with deferasirox FCT
    760.229 ± 798.0836
        Week 56 - treated with deferasirox FCT (n=113)
    903.622 ± 832.9872
        Week 60 - treated with deferasirox FCT (n=106)
    980.644 ± 994.4445
        Week 64 - treated with deferasirox FCT (n=102)
    863.579 ± 854.8592
        Week 68 - treated with deferasirox FCT (n=92)
    921.725 ± 860.8738
        Week 72 - treated with deferasirox FCT (n=91)
    971.112 ± 872.7306
        Week 76 - treated with deferasirox FCT (n=90)
    888.917 ± 869.0805
        Week 80 - treated with deferasirox FCT (n=77)
    1005.758 ± 868.4338
        Week 84 - treated with deferasirox FCT (n=87)
    1013.683 ± 924.5828
        Week 88 - treated with deferasirox FCT (n=80)
    1117.849 ± 985.0767
        Week 92 - treated with deferasirox FCT (n=74)
    1222.429 ± 1027.2565
        Week 96 - treated with deferasirox FCT (n=74)
    1137.347 ± 972.1286
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AEs) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approx. 30 weeks for deferasirox DT treatment, 39 weeks for deferasirox FCT treatment and 70 weeks for deferasirox FCT treatment
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Deferasirox DT (Core Phase)
    Reporting group description
    		 Participants who were treated with deferasirox DT once daily in the core phase

    Reporting group title
    Deferasirox FCT (Extension phase)
    Reporting group description
    		 Participants who were treated with deferasirox FCT once daily in the extension phase

    Reporting group title
    Deferasirox FCT (Core Phase)
    Reporting group description
    		 Participants who were treated with deferasirox FCT once daily in the core phase

    Serious adverse events
    		 Deferasirox DT (Core Phase) Deferasirox FCT (Extension phase) Deferasirox FCT (Core Phase)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    13 / 148 (8.78%)
    16 / 116 (13.79%)
    6 / 140 (4.29%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Cholangiocarcinoma
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 148 (0.68%)
    2 / 116 (1.72%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 148 (1.35%)
    1 / 116 (0.86%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    3 / 3
    1 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 148 (1.35%)
    0 / 116 (0.00%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood bilirubin increased
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Abdominal injury
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal injury
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Road traffic accident
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Splenic rupture
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ulna fracture
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure congestive
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac failure chronic
         subjects affected / exposed
    0 / 148 (0.00%)
    0 / 116 (0.00%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Polyneuropathy
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Leukopenia
         subjects affected / exposed
    2 / 148 (1.35%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Neutropenia
         subjects affected / exposed
    2 / 148 (1.35%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Optic nerve cupping
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholangitis acute
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Jaundice cholestatic
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 148 (0.68%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Proteinuria
         subjects affected / exposed
    1 / 148 (0.68%)
    1 / 116 (0.86%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 148 (0.00%)
    0 / 116 (0.00%)
    1 / 140 (0.71%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 148 (0.68%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Diarrhoea infectious
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ecthyma
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal infection
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 148 (0.68%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Otitis media
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    0 / 148 (0.00%)
    1 / 116 (0.86%)
    0 / 140 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         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
    		 Deferasirox DT (Core Phase) Deferasirox FCT (Extension phase) Deferasirox FCT (Core Phase)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    82 / 148 (55.41%)
    49 / 116 (42.24%)
    59 / 140 (42.14%)
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    5 / 148 (3.38%)
    6 / 116 (5.17%)
    4 / 140 (2.86%)
         occurrences all number
    7
    6
    6
    Serum ferritin increased
         subjects affected / exposed
    8 / 148 (5.41%)
    1 / 116 (0.86%)
    2 / 140 (1.43%)
         occurrences all number
    9
    1
    2
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    13 / 148 (8.78%)
    7 / 116 (6.03%)
    12 / 140 (8.57%)
         occurrences all number
    15
    8
    14
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    7 / 148 (4.73%)
    5 / 116 (4.31%)
    8 / 140 (5.71%)
         occurrences all number
    7
    10
    8
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    16 / 148 (10.81%)
    2 / 116 (1.72%)
    5 / 140 (3.57%)
         occurrences all number
    17
    3
    5
    Hepatobiliary disorders
    Hepatomegaly
         subjects affected / exposed
    8 / 148 (5.41%)
    0 / 116 (0.00%)
    0 / 140 (0.00%)
         occurrences all number
    8
    0
    0
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    8 / 148 (5.41%)
    12 / 116 (10.34%)
    9 / 140 (6.43%)
         occurrences all number
    8
    18
    10
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    6 / 148 (4.05%)
    3 / 116 (2.59%)
    7 / 140 (5.00%)
         occurrences all number
    6
    3
    9
    Pharyngitis
         subjects affected / exposed
    7 / 148 (4.73%)
    6 / 116 (5.17%)
    6 / 140 (4.29%)
         occurrences all number
    10
    7
    7
    Upper respiratory tract infection
         subjects affected / exposed
    8 / 148 (5.41%)
    4 / 116 (3.45%)
    8 / 140 (5.71%)
         occurrences all number
    9
    4
    8
    Urinary tract infection
         subjects affected / exposed
    9 / 148 (6.08%)
    3 / 116 (2.59%)
    3 / 140 (2.14%)
         occurrences all number
    9
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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Feb 2019
    The purpose of this amendment was to provide additional clarity to the study Investigators in relation to dosing of deferasirox for patients with non-transfusion dependent thalassemia (NTDT), those patients changing therapy from deferiprone, and those patients with body weight lower than 20 kg; to provide additional guidance in relation to the ocular and auditory assessments; to revise the underlying assumptions used to calculate the sample size of the study; to clarify the inclusion criterion that relates to prior therapy; to correct inconsistencies and typos; and to update withdrawal of consent language.

    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.
    Due to EudraCT system limitations, no statistical analyses have been specified for the endpoints, as this is a single arm study and comparisons within an arm are not supported by the system. Please use https://www.novctrd.com/ for complete results.
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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