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    Clinical Trial Results:
    A study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in patients with Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or other anaemias treated with Exjade®(deferasirox). The MILE Study

    Summary
    EudraCT number
    		 2016-000246-62
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    08 Sep 2011

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Jan 2017
    First version publication date
    04 Jan 2017
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    CICL670AAU01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00673608
    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,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    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
    08 Sep 2011
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Sep 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the study was to evaluate the change in cardiac iron load and cardiac ejection fraction by Magnetic Resonance Imaging (MRI) after 53 weeks of deferasirox treatment in the following: 1. Transfused subjects with haemoglobinopathies (thalassaemia-major (Th-maj) and Sickle Cell Disease (SCD)) and a serum ferritin of > 500 microgram (µg)/liter (L). 2. Myelodysplastic Syndromes (MDS) and other rare anaemias (e.g. Myeloproliferative Disease (MPD), Diamond-blackfan anaemia [DBA]) subjects who demonstrate evidence of transfusional iron overload by a serum ferritin of > 1,000 µg/L.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Nov 2007
    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
    Australia: 53
    Worldwide total number of subjects
    53
    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)
    0
    Adolescents (12-17 years)
    5
    Adults (18-64 years)
    41
    From 65 to 84 years
    7
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 1 center in Australia.

    Pre-assignment
    Screening details
    		 A total of 53 subjects were enrolled and 43 completed the study.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    The study was open label study, hence no blinding was performed

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Th-maj and SCD: Deferasirox
    Arm description
    		 Subjects with haemoglobinopathy like Th-maj or SCD who required regular blood cell transfusions were administered with once daily (o.d.) on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 milligram (mg)/kilogram (kg)/day.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Exjade
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were orally administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day. Deferasirox was supplied as 125 mg, 250 mg and 500 mg tablets which were dispersed by stirring in water, orange or apple juice (tablets were dispersed in about 20 mL of water before dilution with the juice) until a fine suspension was obtained. After the suspension was swallowed, any residue was re-suspended in a small volume of water or juice and swallowed.

    Arm title
    		 MDS/anaemia: Deferasirox
    Arm description
    		 Subjects having other inherited or acquired anaemia like MDS, MPD, DBA and other rare anaemias who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Exjade
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were orally administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day. Deferasirox was supplied as 125 mg, 250 mg and 500 mg tablets which were dispersed by stirring in water, orange or apple juice (tablets were dispersed in about 20 mL of water before dilution with the juice) until a fine suspension was obtained. After the suspension was swallowed, any residue was re-suspended in a small volume of water or juice and swallowed.

    Arm title
    		 Bone marrow transplantation (BMT): Deferasirox
    Arm description
    		 Subjects who had a post BMT who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Exjade
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were orally administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day. Deferasirox was supplied as 125 mg, 250 mg and 500 mg tablets which were dispersed by stirring in water, orange or apple juice (tablets were dispersed in about 20 mL of water before dilution with the juice) until a fine suspension was obtained. After the suspension was swallowed, any residue was re-suspended in a small volume of water or juice and swallowed.

    Arm title
    		 DBA: Deferasirox
    Arm description
    		 Subjects having other inherited or acquired anaemia (MDS, MPD, DBA and other rare anaemias) who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    ICL670
    Other name
    Exjade
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were orally administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day. Deferasirox was supplied as 125 mg, 250 mg and 500 mg tablets which were dispersed by stirring in water, orange or apple juice (tablets were dispersed in about 20 mL of water before dilution with the juice) until a fine suspension was obtained. After the suspension was swallowed, any residue was re-suspended in a small volume of water or juice and swallowed.

    Number of subjects in period 1
    		Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Bone marrow transplantation (BMT): Deferasirox DBA: Deferasirox
    Started
    42
    9
    1
    1
    Completed
    37
    5
    1
    0
    Not completed
    5
    4
    0
    1
         Administrative reasons
    2
    -
    -
    -
         Adverse event(s)
    1
    3
    -
    -
         Lack of efficacy
    1
    -
    -
    -
         Protocol deviation
    1
    1
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Th-maj and SCD: Deferasirox
    Reporting group description
    		 Subjects with haemoglobinopathy like Th-maj or SCD who required regular blood cell transfusions were administered with once daily (o.d.) on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 milligram (mg)/kilogram (kg)/day.

    Reporting group title
    MDS/anaemia: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia like MDS, MPD, DBA and other rare anaemias who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    Bone marrow transplantation (BMT): Deferasirox
    Reporting group description
    		 Subjects who had a post BMT who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    DBA: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia (MDS, MPD, DBA and other rare anaemias) who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Bone marrow transplantation (BMT): Deferasirox DBA: Deferasirox Total
    Number of subjects
    		 42 9 1 1 53
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 29.6 ± 11.74 68 ± 8.54 55 ± 0 16 ± 0 -
    Gender categorical
    Units: Subjects
        Female
    		 26 5 1 0 32
        Male
    		 16 4 0 1 21

    End points

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    End points reporting groups
    Reporting group title
    Th-maj and SCD: Deferasirox
    Reporting group description
    		 Subjects with haemoglobinopathy like Th-maj or SCD who required regular blood cell transfusions were administered with once daily (o.d.) on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 milligram (mg)/kilogram (kg)/day.

    Reporting group title
    MDS/anaemia: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia like MDS, MPD, DBA and other rare anaemias who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    Bone marrow transplantation (BMT): Deferasirox
    Reporting group description
    		 Subjects who had a post BMT who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    DBA: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia (MDS, MPD, DBA and other rare anaemias) who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Subject analysis set title
    Full analysis set population
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Full analysis set population was defined as all subjects in the safety population (all subjects who received at least one dose of study drug) with a cardiac iron sensitive relaxation time (T2*) at baseline and one post-baseline efficacy assessment (cardiac iron load, liver iron concentration, serum ferritin, cardiac function assessment or blood magnetic susceptibility (BMS) assessment)).

    Primary: Change from baseline in cardiac iron load fraction of deferasirox treatment from baseline to 53 weeks

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    End point title
    		 Change from baseline in cardiac iron load fraction of deferasirox treatment from baseline to 53 weeks [1] [2]
    End point description
    Cardiac iron load was evaluated as log ratio of cardiac T2* at 53 weeks to baseline, ln(T2*53 /T2*0). The T2* relaxometry MRI method was used to evaluate cardiac iron load. A positive change from baseline indicated decrease in iron load. The analysis was performed in FAS population. The missing values were not imputed.
    End point type
    Primary
    End point timeframe
    From baseline to Week 53
    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: Least squares estimates for change in cardiac iron load from baseline to end of study was determined and has been reported relative change.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint was planned to evaluate for the specified arm only.
    End point values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Full analysis set population
    Number of subjects analysed
    42
    8
    52
    Units: Relative change in cardiac iron load
        number (confidence interval 95%)
    1.1 (1.03 to 1.17)
    1.12 (0.95 to 1.31)
    1.1 (1.04 to 1.16)
    No statistical analyses for this end point

    Secondary: Absolute change from baseline in Left ventricular ejection fraction (LVEF) values, left ventricular end-systolic (LVES) and end-diastolic (LVED) volume; left ventricular mass (LVM) from baseline values after 53 weeks of deferasirox treatment

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    End point title
    		 Absolute change from baseline in Left ventricular ejection fraction (LVEF) values, left ventricular end-systolic (LVES) and end-diastolic (LVED) volume; left ventricular mass (LVM) from baseline values after 53 weeks of deferasirox treatment [3]
    End point description
    LVEF was defined as the fraction of the end-diastolic volume that was ejected out of left ventricle with each contraction. LVES volume was defined as the volume of blood in a left ventricle at the end of contraction. LVED volume was defined as the volume of blood in a left ventricle immediately before a contraction. LVM increase was a measure of increase in wall thickness, an increase in cavity size, or both. Global cardiac functions: LVEF; LVES and LVED volume; and LVM were assessed by MRI. The absolute change from baseline was calculated as the value at week 53 minus the value at baseline. A negative change from baseline in LVEF indicates improvement in cardiac function while for other parameters a positive change from baseline indicates improvement in cardiac function. The analysis was performed in FAS population. The missing values were not imputed.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 53
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint was planned to evaluate for the specified arm only.
    End point values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Full analysis set population
    Number of subjects analysed
    42
    9
    52
    Units: Absolute change in cardiac function
    number (confidence interval 95%)
        Left Ventricular Ejection Fraction
    -1.4 (-2.83 to 0.02)
    0.11 (-3.91 to 4.13)
    -1.23 (-2.56 to 0.1)
        Left Ventricular End Systolic Volume
    3.02 (0.26 to 5.77)
    4.54 (-3.33 to 12.41)
    3.19 (0.62 to 5.75)
        Left Ventricular End Diastolic Volume
    2.99 (-1.62 to 7.6)
    14.11 (0.93 to 27.3)
    4.22 (-0.16 to 8.61)
        Left Ventricular Mass
    4.37 (1.92 to 6.82)
    -3.09 (-9.88 to 3.69)
    3.53 (1.15 to 5.91)
    No statistical analyses for this end point

    Secondary: Changes from baseline in serum ferritin from baseline values to 53 weeks

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    End point title
    		 Changes from baseline in serum ferritin from baseline values to 53 weeks [4]
    End point description
    Serum ferritin level in the blood directly relate to the amount of iron stored in the body. Serum ferritin levels are indicators for symptoms of anemia. A negative change from baseline indicated increase in iron load. The analysis was performed in FAS population. The missing values were not imputed.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 53
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint was planned to evaluate for the specified arm only.
    End point values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Full analysis set population
    Number of subjects analysed
    42
    9
    53
    Units: microgram(µg)/ Litre (L)
        number (confidence interval 95%)
    -2.56 (-8.88 to 3.77)
    -24.97 (-41.47 to -8.48)
    -5.84 (-11.56 to 0.61)
    No statistical analyses for this end point

    Secondary: Change from baseline in liver iron concentration (LIC) by MRI of the Proton Transverse Relaxation Rate (R2-MRI) from baseline values to 53 weeks

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    End point title
    		 Change from baseline in liver iron concentration (LIC) by MRI of the Proton Transverse Relaxation Rate (R2-MRI) from baseline values to 53 weeks [5]
    End point description
    Measurements of liver iron concentration (LIC) was an important predictors of iron burden, measured using relaxation rate [R2 = 1/relaxation time (T2)] magnetic resonance imaging (R2-MRI) technique at baseline and Week 52. R2-MRI scans using a specific sequence and raw image data were analysed centrally to determine the subject’s LIC value. The analysis was performed in FAS population. The missing values were not imputed.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 53
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint was planned to evaluate for the specified arm only.
    End point values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Full analysis set population
    Number of subjects analysed
    42
    9
    52
    Units: Relative change in LIC
        number (confidence interval 95%)
    -1.89 (-3.91 to 0.13)
    -3.88 (-10.72 to 2.96)
    -2.18 (-4.06 to -0.31)
    No statistical analyses for this end point

    Secondary: Number of subjects with Adverse Events (AEs), Serious Adverse Events (SAEs), AE leading to discontinuation and who died

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    End point title
    		 Number of subjects with Adverse Events (AEs), Serious Adverse Events (SAEs), AE leading to discontinuation and who died
    End point description
    AEs are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards. The analysis was performed on safety set population.
    End point type
    Secondary
    End point timeframe
    From Day 1 to Week 53
    End point values
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Bone marrow transplantation (BMT): Deferasirox DBA: Deferasirox
    Number of subjects analysed
    42
    9
    1
    1
    Units: Subjects
        AEs
    37
    9
    1
    0
        Deaths
    0
    0
    0
    0
        SAEs
    5
    4
    0
    0
        AEs leading to study drug discontinuation
    1
    2
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Serious Adverse Events are monitored from date of First Subject First Visit (FSFV) until Last Subject Last Visit (LSLV). All other adverse events are monitored from First Subject First Treatment until LSLV.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    Th-maj and SCD: Deferasirox
    Reporting group description
    		 Subjects with haemoglobinopathy like Th-maj or SCD who required regular blood cell transfusions were administered with o.d on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    MDS/anaemia: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia like MDS, MPD, DBA and other rare anaemias who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    Bone marrow transplantation (BMT): Deferasirox:
    Reporting group description
    		 Subjects who had a post BMT who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Reporting group title
    DBA: Deferasirox
    Reporting group description
    		 Subjects having other inherited or acquired anaemia (MDS, MPD, DBA and other rare anaemias) who required regular blood cell transfusions were administered with deferasirox o.d. on an empty stomach at least 30 minutes before food, preferably at the same time each day. Dose was up to 40 mg/kg/day.

    Serious adverse events
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Bone marrow transplantation (BMT): Deferasirox: DBA: Deferasirox
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 42 (9.52%)
    3 / 9 (33.33%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    WRIST FRACTURE
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Congenital, familial and genetic disorders
    SICKLE CELL ANAEMIA WITH CRISIS
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    CHEST PAIN
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    PYREXIA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    DIARRHOEA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    RECTAL HAEMORRHAGE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    VOMITING
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    MANIA
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    RENAL COLIC
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    BONE PAIN
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    MUSCULOSKELETAL PAIN
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    LOBAR PNEUMONIA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    NEUTROPENIC SEPSIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Th-maj and SCD: Deferasirox MDS/anaemia: Deferasirox Bone marrow transplantation (BMT): Deferasirox: DBA: Deferasirox
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    37 / 42 (88.10%)
    9 / 9 (100.00%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
    Investigations
    BLOOD CREATININE INCREASED
         subjects affected / exposed
    8 / 42 (19.05%)
    2 / 9 (22.22%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    8
    2
    1
    0
    ALANINE AMINOTRANSFERASE INCREASED
         subjects affected / exposed
    6 / 42 (14.29%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    6
    2
    0
    0
    ASPARTATE AMINOTRANSFERASE INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    BLOOD ALKALINE PHOSPHATASE INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    BLOOD BILIRUBIN INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    SERUM FERRITIN INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Basophil count increased
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    EOSINOPHIL COUNT INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    INTERNATIONAL NORMALISED RATIO INCREASED
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vascular disorders
    DEEP VEIN THROMBOSIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    PALLOR
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nervous system disorders
    HEADACHE
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    3
    2
    0
    0
    LETHARGY
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    DIZZINESS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    General disorders and administration site conditions
    PYREXIA
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    2
    0
    0
    FATIGUE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    CHILLS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    OEDEMA PERIPHERAL
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood and lymphatic system disorders
    ANAEMIA
         subjects affected / exposed
    0 / 42 (0.00%)
    0 / 9 (0.00%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    LYMPHADENOPATHY
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    NEUTROPENIA
         subjects affected / exposed
    0 / 42 (0.00%)
    0 / 9 (0.00%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eye disorders
    DRY EYE
         subjects affected / exposed
    0 / 42 (0.00%)
    0 / 9 (0.00%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    EYE HAEMORRHAGE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastrointestinal disorders
    DIARRHOEA
         subjects affected / exposed
    10 / 42 (23.81%)
    5 / 9 (55.56%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    10
    5
    0
    0
    NAUSEA
         subjects affected / exposed
    7 / 42 (16.67%)
    4 / 9 (44.44%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    7
    4
    0
    0
    ABDOMINAL PAIN
         subjects affected / exposed
    4 / 42 (9.52%)
    4 / 9 (44.44%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    4
    4
    0
    0
    ABDOMINAL PAIN UPPER
         subjects affected / exposed
    6 / 42 (14.29%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    6
    0
    0
    0
    CONSTIPATION
         subjects affected / exposed
    4 / 42 (9.52%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    4
    1
    0
    0
    VOMITING
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    3
    2
    0
    0
    ABDOMINAL DISTENSION
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    BOWEL MOVEMENT IRREGULARITY
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    DRY MOUTH
         subjects affected / exposed
    0 / 42 (0.00%)
    0 / 9 (0.00%)
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    MOUTH HAEMORRHAGE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    RECTAL HAEMORRHAGE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    DYSPNOEA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    COUGH
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    EPISTAXIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    RALES
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    RASH
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    3
    2
    0
    0
    ECCHYMOSIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    PETECHIAE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    BACK PAIN
         subjects affected / exposed
    7 / 42 (16.67%)
    0 / 9 (0.00%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    7
    0
    0
    0
    ARTHRALGIA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    JOINT SWELLING
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infections and infestations
    UPPER RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    13 / 42 (30.95%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    13
    1
    0
    0
    URINARY TRACT INFECTION
         subjects affected / exposed
    4 / 42 (9.52%)
    2 / 9 (22.22%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    4
    2
    0
    0
    NASOPHARYNGITIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    LOWER RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    VIRAL INFECTION
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    LOBAR PNEUMONIA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    LOCALISED INFECTION
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    NEUTROPENIC SEPSIS
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Metabolism and nutrition disorders
    DECREASED APPETITE
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    DEHYDRATION
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    GOUT
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    HYPOKALAEMIA
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 9 (11.11%)
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Oct 2010
    The amendment updated: the title of study; inclusion and exclusion criteria; subject population; starting dose of study drug; dose adjustments; cardiac assessments; restricted concomitant therapies; study visit window; subject numbers and statistical analysis.

    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
    For support, Contact us.
    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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