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    Clinical Trial Results:
    A phase II, multi-center, single-arm, prospective study to evaluate the safety and efficacy of deferasirox in beta-thalassemia major patients after hematopoietic stem cell transplantation

    Summary
    EudraCT number
    		 2016-001561-88
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    21 Oct 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Jun 2016
    First version publication date
    25 Jun 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670ATR04
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01610297
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    21 Oct 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Oct 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the present study was to determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period
    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
    19 Sep 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Turkey: 27
    Worldwide total number of subjects
    27
    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)
    20
    Adolescents (12-17 years)
    7
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The patients received oral deferasirox at an initial dose of 10 mg/kg/day and dose escalation was allowed up to 20 mg/kg daily for 12 months or until the serum ferritin level was below 500 μg/L. Dose titration was allowed in 3 months periods by 5 mg/kg/day at the discretion of the investigator

    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
    		 ICL670
    Arm description
    		 Oral dose of ICL670 at 10 mg/kg daily
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasinox
    Investigational medicinal product code
    ICL670
    Other name
    Pharmaceutical forms
    Dispersible tablet
    Routes of administration
    Oral use
    Dosage and administration details
    deferasirox (ICL670) 10 mg/kg, oral, daily

    Number of subjects in period 1
    		ICL670
    Started
    27
    Completed
    26
    Not completed
    1
         Lost to follow-up
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ICL670
    Reporting group description
    		 Oral dose of ICL670 at 10 mg/kg daily

    Reporting group values
    		 ICL670 Total
    Number of subjects
    		 27 27
    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)
    		 20 20
        Adolescents (12-17 years)
    		 7 7
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous |
    Units: years
        arithmetic mean (standard deviation)
    		 9.07 ( 3.81 ) -
    Gender, Male/Female
    Units: Participants
        Female
    		 8 8
        Male
    		 19 19

    End points

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    End points reporting groups
    Reporting group title
    ICL670
    Reporting group description
    		 Oral dose of ICL670 at 10 mg/kg daily

    Primary: Number of Participants with Adverse Events, Serious Adverse Events and Deaths as a measure of Safety and Tolerability

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    End point title
    		 Number of Participants with Adverse Events, Serious Adverse Events and Deaths as a measure of Safety and Tolerability [1]
    End point description
    To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period. No statistical analysis was planned for this primary outcome. The Safety Set (SS) includes all included patients who were included in the study.
    End point type
    Primary
    End point timeframe
    12 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: There was no planned analysis for this safety endpoint.
    End point values
    		 ICL670
    Number of subjects analysed
    27
    Units: Participants
        Adverse events
    25
        Serious adverse events
    3
        Death
    0
    No statistical analyses for this end point

    Secondary: Change in serum ferritin level

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    End point title
    		 Change in serum ferritin level
    End point description
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level. The The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
    End point type
    Secondary
    End point timeframe
    Baseline, 12 Months
    End point values
    		 ICL670
    Number of subjects analysed
    27
    Units: ng/mL
    arithmetic mean (standard deviation)
        Baseline
    1766.81 ( 599.64 )
        Month 12
    903.56 ( 596.62 )
    No statistical analyses for this end point

    Secondary: Change in the further parameters of iron overload (cardiac iron concentration by MR examination)

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    End point title
    		 Change in the further parameters of iron overload (cardiac iron concentration by MR examination)
    End point description
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the T2*MRI (Cardiac MRI). The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
    End point type
    Secondary
    End point timeframe
    Baseline, 12 month
    End point values
    		 ICL670
    Number of subjects analysed
    27
    Units: T2*MRI
    arithmetic mean (standard deviation)
        Baseline Cardiac MRI(n= 27)
    26.48 ( 7.49 )
        Week 52 Cardiac MRI (n=24)
    28.25 ( 5.53 )
    No statistical analyses for this end point

    Secondary: The percentage of patients reaching serum ferritin levels lower than 500 μg/L

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    End point title
    		 The percentage of patients reaching serum ferritin levels lower than 500 μg/L
    End point description
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level. The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
    End point type
    Secondary
    End point timeframe
    Week 28 and Week 52
    End point values
    		 ICL670
    Number of subjects analysed
    27
    Units: Patients
    number (not applicable)
        Week 28
    7.7
        Week 52
    33.3
    No statistical analyses for this end point

    Secondary: Change in the further parameters of iron overload (liver iron concentration by Magnetic resonance imaging (MRI examination)

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    End point title
    		 Change in the further parameters of iron overload (liver iron concentration by Magnetic resonance imaging (MRI examination)
    End point description
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the R2*MRI (Liver MRI).
    End point type
    Secondary
    End point timeframe
    Baseline, 12 Months
    End point values
    		 ICL670
    Number of subjects analysed
    27
    Units: mg\g dry weight
    arithmetic mean (standard deviation)
        Baseline Liver MRI (n=27)
    12.07 ( 9.42 )
        Week 52 Liver MRI (n=25)
    4.62 ( 2.85 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    ICL670
    Reporting group description
    		 Oral dose of ICL670 at 10 mg/kg daily

    Serious adverse events
    		 ICL670
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 27 (11.11%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Office visit
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Hepatitis B
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 ICL670
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 27 (85.19%)
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    6 / 27 (22.22%)
         occurrences all number
    1
    White blood cell count decreased
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    7 / 27 (25.93%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anemia
         subjects affected / exposed
    7 / 27 (25.93%)
         occurrences all number
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    7 / 27 (25.93%)
         occurrences all number
    1
    Influenza like illness
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    7 / 27 (25.93%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Rhinorrhea
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Infections and infestations
    Pharyngitis
         subjects affected / exposed
    6 / 27 (22.22%)
         occurrences all number
    1
    Haemophilus infection
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Herpes zoster
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Infection
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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