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    Clinical Trial Results:
    A phase II pilot study to assess the presence of molecular factors predictive for hematologic response in myelodysplastic syndrome patients receiving deferasirox therapy in hematological centers in Belgium using gene expressing profiling from baseline bone marrow.

    Summary
    EudraCT number
    		 2015-003775-30
    Trial protocol
    		 BE  
    Global end of trial date
    01 Jul 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Oct 2017
    First version publication date
    14 Oct 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670ABE04
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02663752
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharmaceuticals AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Novartis Pharmaceuticals AG, Novartis Pharmaceuticals AG, 41 613241111,
    Scientific contact
    Novartis Pharmaceuticals AG, Novartis Pharmaceuticals 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
    01 Jul 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Jul 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To identify differentially expressed genes in baseline bone marrow samples of low and intermediate-1 risk MDS patients with a hematologic response vs non-responder patients based on NGS of the whole transcriptome to search for a predictive gene signature.
    Protection of trial subjects
    Retrospective design was selected because, by comparison with classical investigational design, it does not require delay in treatment initiation. In addition, this allows the study to be shorter as we do not have to wait for a hematological response to develop in the study population. This specific design is made possible due to the storage and availability of most baseline bone marrow aspirates taken at the time of MDS diagnosis in Belgian hospitals and preserved under the right circumstances to perform RNA sequencing. Patients
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 May 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
    Belgium: 1
    Worldwide total number of subjects
    1
    EEA total number of subjects
    1
    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)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Due to low recruitment (1 patient after 12 months of recruitment) the study has been cancelled.

    Pre-assignment
    Screening details
    		 Only 1 patient was recruited.

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

    Arms
    Arm title
    		 Deferasirox
    Arm description
    		 All patients are already on commercial deferasirox before entering the study.
    Arm type
    		 Deferasirox was prerequisite for trial entry

    Investigational medicinal product name
    Deferasirox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    The patient continued taking the same dose as he was used to before study entry and when deferasirox is provided as IMP. There was no treatment administration specific to this study. However, eligibility criteria include the need for patients to be treated with deferasirox.

    Number of subjects in period 1
    		Deferasirox
    Started
    1
    Completed
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Period
    Reporting group description
    		 -

    Reporting group values
    		 Overall Period Total
    Number of subjects
    		 1 1
    Age categorical
    Units: Subjects
        From 65-84 years
    		 1 1
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 All patients are already on commercial deferasirox before entering the study.

    Primary: Fold increase/decrease in gene transcription from baseline bone marrow aspirate of responders versus nonresponders’

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    End point title
    		 Fold increase/decrease in gene transcription from baseline bone marrow aspirate of responders versus nonresponders’ [1]
    End point description
    Using next-generation sequencing, gene expression profiling in responder and non-responder patients were to be performed on existing bone marrow aspirate samples. Gene transcription were then to be compared between the two groups and the fold increase/decrease in differentially expressed genes were to be calculated.
    End point type
    Primary
    End point timeframe
    18 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: Since the study has been cancelled and only 1 patient has been included, no statistics has been performed.
    End point values
    		 Deferasirox
    Number of subjects analysed
    0 [2]
    Units: Number
    Notes
    [2] - Patient was randomized but did not complete trial.
    No statistical analyses for this end point

    Secondary: Time to response

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    End point title
    		 Time to response
    End point description
    The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group.
    End point type
    Secondary
    End point timeframe
    18 months
    End point values
    		 Deferasirox
    Number of subjects analysed
    0 [3]
    Units: Number
    Notes
    [3] - Patient was randomized but did not complete trial.
    No statistical analyses for this end point

    Secondary: Changes in serum ferritin levels

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    End point title
    		 Changes in serum ferritin levels
    End point description
    From baseline to time of response (responder group) or time to last follow up (non-responders).
    End point type
    Secondary
    End point timeframe
    Baseline, 18 months
    End point values
    		 Deferasirox
    Number of subjects analysed
    0 [4]
    Units: Number
    Notes
    [4] - Patient was randomized but did not complete trial.
    No statistical analyses for this end point

    Secondary: Deferasirox dose used

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    End point title
    		 Deferasirox dose used
    End point description
    Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the nonresponder group.
    End point type
    Secondary
    End point timeframe
    18 months
    End point values
    		 Deferasirox
    Number of subjects analysed
    0 [5]
    Units: Number
    Notes
    [5] - Patient was randomized but did not complete trial.
    No statistical analyses for this end point

    Secondary: Changes in serum transferrin levels

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    End point title
    		 Changes in serum transferrin levels
    End point description
    From baseline to time of response (responder group) or time to last follow up (non-responders).
    End point type
    Secondary
    End point timeframe
    Baseline, 18 months
    End point values
    		 Deferasirox
    Number of subjects analysed
    0 [6]
    Units: Number
    Notes
    [6] - Patient was randomized but did not complete trial.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    Deferasirox
    Reporting group description
    		 All patients are already on commercial deferasirox before entering the study.

    Serious adverse events
    		 Deferasirox
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Deferasirox
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 1 (0.00%)
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: The patient did not experience any Adverse Events during the trial.

    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.
    The trial was terminated due to low enrollment.
    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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