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    Clinical Trial Results:
    Open-label, multicenter, single arm, phase III study to collect additional safety and efficacy data with deferasirox film-coated tablets in patients completing study CICL670F2201

    Summary
    EudraCT number
    		 2016-000186-23
    Trial protocol
    		 AT   GR   IT  
    Global end of trial date
    23 Jul 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Feb 2020
    First version publication date
    07 Feb 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670AIC04
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02720536
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Study Director, Novartis Pharma AG, 41 613241111, Novartis.email@novartis.com
    Scientific contact
    Study Director, 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
    23 Jul 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the overall safety of deferasirox FCT formulation in patients with transfusion dependent thalassemia or MDS at very low, low or intermediate risk
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Aug 2016
    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
    Austria: 3
    Country: Number of subjects enrolled
    Greece: 10
    Country: Number of subjects enrolled
    Italy: 40
    Worldwide total number of subjects
    53
    EEA total number of subjects
    53
    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)
    3
    Adults (18-64 years)
    47
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 All patients completed study CICL670F2201 prior to entry into this study

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

    Arms
    Arm title
    		 Deferasirox
    Arm description
    		 Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient’s actual body weight.
    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 was supplied as film-coated tablets of 90 mg, 180 mg and 360 mg dose strengths

    Number of subjects in period 1
    		Deferasirox
    Started
    53
    Completed
    34
    Not completed
    19
         PR- unwilling to comply with procedures
    1
         PR - unsatisfactory therapeutic effect
    2
         PR - abnormal lab value(s)
    1
         PR - pregnancy
    2
         Primary reason (PR) - subject withdrawal
    11
         PR - death
    1
         PR - adverse event
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient’s actual body weight.

    Reporting group values
    		 Deferasirox Total
    Number of subjects
    		 53 53
    Age Categorical
    Units: Participants
        <18
    		 3 3
        ≥18 - <50
    		 46 46
        ≥ 50 - <65
    		 1 1
        ≥ 65
    		 3 3
    Sex: Female, Male
    Units:
        Female
    		 35 35
        Male
    		 18 18
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian
    		 50 50
        Asian
    		 1 1
        Other
    		 2 2
    Main underlying disease
    Participants with myelodysplastic syndrome (MDS) and transfusion-dependent thalassemia. The very low, low or intermediate risk MDS was to be determined by the Revised International Prognostic Scoring System (IPSS-R) and IPSS-R was to be confirmed by a bone marrow examination within 6 months prior to study entry. MDS with INT risk =MDS with intermediate risk
    Units: Subjects
        MDS with very low risk as per the IPSS - R
    		 2 2
        MDS with low risk as per the IPSS - R
    		 1 1
        MDS with INT risk as per the IPSS - R
    		 1 1
        Transfusion-dependent thalassemia
    		 49 49

    End points

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    End points reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient’s actual body weight.

    Subject analysis set title
    Month 12
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 12

    Subject analysis set title
    Baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Serum ferritin value at baseline

    Subject analysis set title
    Month 6
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Serum ferritin value at baseline and at month 6

    Subject analysis set title
    Month 12
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Serum ferritin value at baseline and at month 12

    Subject analysis set title
    Baseline
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Baseline of laboratory value

    Subject analysis set title
    Month 6
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 6

    Subject analysis set title
    Month 6
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 6

    Subject analysis set title
    Month 6
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 6

    Subject analysis set title
    Month 6
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 6

    Subject analysis set title
    Baseline
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Baseline of laboratory value

    Subject analysis set title
    Month 12
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Change from baseline at month 12

    Primary: Overview of number of participants with adverse events

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    End point title
    		 Overview of number of participants with adverse events [1]
    End point description
    Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study
    End point type
    Primary
    End point timeframe
    Baseline up to approximately 25 months
    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: No analysis was done
    End point values
    		 Deferasirox
    Number of subjects analysed
    53
    Units: number of participants
        Adverse events (AEs)
    52
        Treatment related AEs
    20
        Severe adverse events
    14
        Treatment related severe adverse events
    2
        Serious adverse events (SAEs)
    13
        Treatment related SAEs
    0
        Fatal SAEs
    1
        Treatment related fatal SAEs
    0
        AEs leading to discontinuation
    4
        Treatment related AEs leading to discontinuation
    2
        AEs leading to dose adjust/interruption
    33
        AEs requiring additional therapy
    4
    No statistical analyses for this end point

    Primary: Change from baseline red blood cells (RBC) (10**12/L) at Month 6 and Month 12 (Safety set)

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    End point title
    		 Change from baseline red blood cells (RBC) (10**12/L) at Month 6 and Month 12 (Safety set) [2]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [2] - 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: No analysis was done
    End point values
    		 Month 12 Baseline Month 6
    Number of subjects analysed
    40
    53
    42
    Units: 10**12/L
    arithmetic mean (standard deviation)
        Baseline
    3.746 ( 0.5071 )
    3.753 ( 0.4909 )
    3.733 ( 0.5016 )
        Post
    3.607 ( 0.5423 )
    999.9 ( 999.9 )
    3.610 ( 0.5153 )
        Change
    -0.139 ( 0.4330 )
    999.9 ( 999.9 )
    -0.124 ( 0.3892 )
        Percentage relative change
    -3.281 ( 11.7360 )
    999.9 ( 999.9 )
    -2.887 ( 10.3411 )
    No statistical analyses for this end point

    Primary: Change from baseline white blood cells (WBC) (10**9/L) at Month 6 and Month 12

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    End point title
    		 Change from baseline white blood cells (WBC) (10**9/L) at Month 6 and Month 12 [3]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [3] - 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: No analysis was done
    End point values
    		 Month 12 Baseline Month 6
    Number of subjects analysed
    40
    53
    42
    Units: 10**9/L
    arithmetic mean (standard deviation)
        Baseline
    9.100 ( 5.4667 )
    9.336 ( 5.1383 )
    9.559 ( 5.6650 )
        Post
    9.007 ( 4.4385 )
    999.9 ( 999.9 )
    9.090 ( 4.3712 )
        Change
    -0.093 ( 4.5792 )
    999.9 ( 999.9 )
    -0.469 ( 4.0727 )
        Percentage relative change
    9.537 ( 47.2220 )
    999.9 ( 999.9 )
    3.788 ( 33.4252 )
    No statistical analyses for this end point

    Primary: Change from baseline platelets (10**9/L) at Month 6 and Month 12

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    End point title
    		 Change from baseline platelets (10**9/L) at Month 6 and Month 12 [4]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [4] - 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: No analysis was done
    End point values
    		 Month 12 Baseline Month 6
    Number of subjects analysed
    40
    53
    42
    Units: 10**9/L
    arithmetic mean (standard deviation)
        Baseline
    339.4 ( 196.41 )
    330.1 ( 188.81 )
    336.5 ( 195.97 )
        Post
    361.9 ( 165.91 )
    999.9 ( 999.9 )
    344.2 ( 190.43 )
        Change
    22.4 ( 100.72 )
    999.9 ( 999.9 )
    7.7 ( 112.98 )
        Percentage relative change
    14.9 ( 27.98 )
    999.9 ( 999.9 )
    10.3 ( 40.78 )
    No statistical analyses for this end point

    Primary: Change from baseline serum creatinine (umol/L) at Month 6 and Month 12

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    End point title
    		 Change from baseline serum creatinine (umol/L) at Month 6 and Month 12 [5]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [5] - 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: No analysis was done
    End point values
    		 Month 12 Baseline Month 6
    Number of subjects analysed
    40
    53
    43
    Units: umol/L
    arithmetic mean (standard deviation)
        Baseline
    57.4 ( 16.91 )
    55.1 ( 16.16 )
    54.4 ( 16.34 )
        Post
    63.5 ( 16.78 )
    999.9 ( 999.9 )
    62.0 ( 16.17 )
        Change
    6.1 ( 11.09 )
    999.9 ( 999.9 )
    7.6 ( 9.51 )
        Percentage relative change
    12.7 ( 18.66 )
    999.9 ( 999.9 )
    17.1 ( 21.54 )
    No statistical analyses for this end point

    Primary: Change from baseline creatinine clearance (mL/min) at Month 6 and Month 12

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    End point title
    		 Change from baseline creatinine clearance (mL/min) at Month 6 and Month 12 [6]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [6] - 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: No analysis was done
    End point values
    		 Month 12 Month 6 Baseline
    Number of subjects analysed
    40
    41
    52
    Units: mL/min
    arithmetic mean (standard deviation)
        Baseline
    129.6 ( 50.30 )
    131.8 ( 56.98 )
    131.9 ( 51.32 )
        Post
    119.8 ( 49.34 )
    116.0 ( 48.71 )
    999.9 ( 999.9 )
        Change
    -9.8 ( 32.74 )
    -15.8 ( 35.12 )
    999.9 ( 999.9 )
        Percentage relative change
    -4.5 ( 31.37 )
    -7.9 ( 30.68 )
    999.9 ( 999.9 )
    No statistical analyses for this end point

    Primary: Change from baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12

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    End point title
    		 Change from baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 [7]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [7] - 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: No analysis was done
    End point values
    		 Month 12 Baseline Month 6
    Number of subjects analysed
    40
    53
    44
    Units: U/L
    arithmetic mean (standard deviation)
        Baseline
    39.5 ( 33.32 )
    37.4 ( 31.47 )
    39.4 ( 33.51 )
        Post
    25.9 ( 19.08 )
    999.9 ( 999.9 )
    28.9 ( 21.12 )
        Change
    -13.6 ( 27.44 )
    999.9 ( 999.9 )
    -10.5 ( 29.16 )
        Percentage relative change
    -4.3 ( 68.59 )
    999.9 ( 999.9 )
    2.8 ( 83.58 )
    No statistical analyses for this end point

    Primary: Change from baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12

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    End point title
    		 Change from baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 [8]
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point
    End point type
    Primary
    End point timeframe
    Baseline, 6 and 12 months
    Notes
    [8] - 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: No analysis was done
    End point values
    		 Baseline Month 6 Month 12
    Number of subjects analysed
    53
    43
    39
    Units: U/L
    arithmetic mean (standard deviation)
        Baseline
    30.7 ( 24.40 )
    32.9 ( 26.48 )
    33.7 ( 26.67 )
        Post
    999.9 ( 999.9 )
    27.6 ( 18.81 )
    25.2 ( 12.78 )
        Change
    999.9 ( 999.9 )
    -5.3 ( 21.03 )
    -8.5 ( 19.18 )
        Percentage relative change
    999.9 ( 999.9 )
    2.9 ( 58.91 )
    -6.2 ( 46.20 )
    No statistical analyses for this end point

    Secondary: Absolute and relative change of serum ferritin level from baseline at month 6 and 12

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    End point title
    		 Absolute and relative change of serum ferritin level from baseline at month 6 and 12
    End point description
    The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative change from baseline is regarded as an improvement in this study
    End point type
    Secondary
    End point timeframe
    Baseline, 6 and 12 months
    End point values
    		 Baseline Month 6 Month 12
    Number of subjects analysed
    53
    44
    36
    Units: ug/l
    arithmetic mean (standard deviation)
        Baseline
    2523.51 ( 1746.087 )
    2614.12 ( 1781.287 )
    2542.76 ( 1904.087 )
        Post baseline
    999.9 ( 999.9 )
    2228.94 ( 1910.182 )
    1924.49 ( 1839.818 )
        Absolute change from baseline
    999.9 ( 999.9 )
    -385.18 ( 1038.789 )
    -618.26 ( 1054.150 )
        Percentage relative change from baseline
    999.9 ( 999.9 )
    -18.61 ( 32.969 )
    -29.08 ( 33.056 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
    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
    22.0
    Reporting groups
    Reporting group title
    DFXFCT
    Reporting group description
    		 DFXFCT

    Serious adverse events
    		 DFXFCT
    Total subjects affected by serious adverse events
         subjects affected / exposed
    13 / 53 (24.53%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Malignant melanoma
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Ovarian adenoma
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Papillary thyroid cancer
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Fracture
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Rib fracture
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Ulna fracture
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cardiac failure
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Pregnancy, puerperium and perinatal conditions
    Abortion spontaneous
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Hepatobiliary disorders
    Biliary colic
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cholecystitis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cholestasis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Hepatic failure
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Psychiatric disorders
    Panic attack
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Renal and urinary disorders
    Calculus urinary
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Hydronephrosis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Renal colic
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Ureterolithiasis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Endocrine disorders
    Goitre
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Infections and infestations
    Diverticulitis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Lymph gland infection
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Urosepsis
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Product issues
    Device failure
         subjects affected / exposed
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 DFXFCT
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    48 / 53 (90.57%)
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    4
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    8 / 53 (15.09%)
         occurrences all number
    19
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Nervous system disorders
    Headache
         subjects affected / exposed
    14 / 53 (26.42%)
         occurrences all number
    28
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    10 / 53 (18.87%)
         occurrences all number
    35
    Influenza like illness
         subjects affected / exposed
    5 / 53 (9.43%)
         occurrences all number
    6
    Pyrexia
         subjects affected / exposed
    13 / 53 (24.53%)
         occurrences all number
    21
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    10 / 53 (18.87%)
         occurrences all number
    18
    Abdominal pain upper
         subjects affected / exposed
    11 / 53 (20.75%)
         occurrences all number
    14
    Constipation
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Diarrhoea
         subjects affected / exposed
    14 / 53 (26.42%)
         occurrences all number
    25
    Dyspepsia
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    5
    Gastritis
         subjects affected / exposed
    5 / 53 (9.43%)
         occurrences all number
    6
    Nausea
         subjects affected / exposed
    12 / 53 (22.64%)
         occurrences all number
    14
    Vomiting
         subjects affected / exposed
    12 / 53 (22.64%)
         occurrences all number
    14
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    7
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    12 / 53 (22.64%)
         occurrences all number
    18
    Oropharyngeal pain
         subjects affected / exposed
    9 / 53 (16.98%)
         occurrences all number
    16
    Productive cough
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Glycosuria
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    4
    Proteinuria
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    5
    Renal colic
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    6
    Back pain
         subjects affected / exposed
    5 / 53 (9.43%)
         occurrences all number
    13
    Musculoskeletal pain
         subjects affected / exposed
    6 / 53 (11.32%)
         occurrences all number
    11
    Myalgia
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Neck pain
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    5 / 53 (9.43%)
         occurrences all number
    7
    Infections and infestations
    Ear infection
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    5
    Gastroenteritis
         subjects affected / exposed
    7 / 53 (13.21%)
         occurrences all number
    14
    Influenza
         subjects affected / exposed
    9 / 53 (16.98%)
         occurrences all number
    11
    Nasopharyngitis
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    7
    Pharyngitis
         subjects affected / exposed
    7 / 53 (13.21%)
         occurrences all number
    10
    Respiratory tract infection
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    5
    Rhinitis
         subjects affected / exposed
    8 / 53 (15.09%)
         occurrences all number
    9
    Sinusitis
         subjects affected / exposed
    3 / 53 (5.66%)
         occurrences all number
    4
    Tonsillitis
         subjects affected / exposed
    5 / 53 (9.43%)
         occurrences all number
    5
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 53 (7.55%)
         occurrences all number
    4
    Urinary tract infection
         subjects affected / exposed
    6 / 53 (11.32%)
         occurrences all number
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Oct 2017
    • The description related to study treatment duration was amended. • References to “Core and Extension” study were removed throughout the protocol. • Aligned protocol text with the Exjade prescribing information. • Corrected discrepancies on the description of the visit intervals. • Clarification was given on drug supply disposal and destruction and dosing regimen. • Corrected description for local clinical laboratory parameters collection plan. • Removed reference related to collection of pregnancy outcomes for the pregnant partners of male patients in accordance with Novartis’s pregnancy guidance working group. • Clarification was given about demographic and other baseline data to be summarized descriptively in line with actual eCRF design. • Corrected description for duration of treatment exposure, actual and planned daily dose. • Clarification about the descriptive statistical analyses of the primary objective. • Updated description for supportive sensitivity analyses • Added description for secondary efficacy objective analyses • Updated description for adverse events analyses

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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