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    Clinical Trial Results:
    Pre-Clinical Phase 0 Microdose Study to evaluate the effect of Melphalan, Bortezomib and Dexamethasone on cellular gene-expression.

    Summary
    EudraCT number
    		 2011-003791-37
    Trial protocol
    		 DK  
    Global end of trial date
    31 Dec 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Oct 2017
    First version publication date
    14 Oct 2017
    Other versions
    Summary report(s)
    		 Phase 0 study on microdose melphalan in multiple myeloma

    Trial information

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    Trial identification
    Sponsor protocol code
    KFE2011.06
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Aalborg University Hospital
    Sponsor organisation address
    Mølleparkvej 4, Aalborg, Denmark, 9000
    Public contact
    Clinical Research Unit, Henrik Gregersen, Department of Haematology, Aalborg University Hospital, 45 99326320, lit@rn.dk
    Scientific contact
    Clinical Research Unit, Henrik Gregersen, Department of Haematology, Aalborg University Hospital, 45 99326320, lit@rn.dk
    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
    09 Sep 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Dec 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Dec 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To identify the specific genes that is up- or downregulated in patiaents who receive a microdose of either Melphalan (Alkeran)
    Protection of trial subjects
    Use of antiemetic
    Background therapy
    		 Four series of VCD
    Evidence for comparator
    		 No
    Actual start date of recruitment
    01 Oct 2013
    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
    Denmark: 6
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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)
    2
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Six patients with newly diagnosed multiple myeloma were included in the study

    Pre-assignment
    Screening details
    		 Treatment demanding multiple myeloma according to the IMWW criteria in patients eligible for high-dose melphalan

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

    Arms
    Arm title
    		 Microdose melphalan
    Arm description
    		 All patient received micro-dose melphalan
    Arm type
    		 Experimental

    Investigational medicinal product name
    melphalan
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous bolus use
    Dosage and administration details
    2 mg/sqm

    Number of subjects in period 1
    		Microdose melphalan
    Started
    6
    Completed
    6

    Baseline characteristics

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

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 6 6
    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)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 2 2
        From 65-84 years
    		 4 4
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 2 2
        Male
    		 4 4

    End points

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    End points reporting groups
    Reporting group title
    Microdose melphalan
    Reporting group description
    		 All patient received micro-dose melphalan

    Subject analysis set title
    study is to identify specific genes
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    study is to identify specific genes

    Primary: was to identify specific genes that might be up- or downregulated in the mononuclear cells (MNC) in the peripheral blood in multiple myeloma patients who receive a microdose of melphalan.

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    End point title
    		 was to identify specific genes that might be up- or downregulated in the mononuclear cells (MNC) in the peripheral blood in multiple myeloma patients who receive a microdose of melphalan. [1]
    End point description
    Results: No genes showed significant changes during the microdosis time period, when analyzed using multiple test correction. However, the genes showing most significance using un-adjusted p-values showed a small but systematic changes in a three dimensional PCA plot illustrating that the total composition of MNCs experience changes in immediate response to melphalan and that the effect is gradually lost after 120 minutes post microdose injection. However, one should be cautious interpreting these results, due to the pre-selection of significant genes. There were no clear patterns using SOMs. Finally, an analysis restricted to REGS genes and genes commonly associated with sleep patterns were conducted2. None of these showed significant changes over time and did not cluster the data patient or time wise.
    End point type
    Primary
    End point timeframe
    Two hours after infusion of microdose melphalan
    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: . Detection of the genes with significant change over time was conducted by linear models, where significance was determined on the basis of un-adjusted p-values and p-values adjusted for multiple testing. The analysis on the significant genes included unsupervised clustering and it was assessed whether clustering based on time or patient occurred. Detection of patterns across time by inspecting the PCA trajectories over time of all the significant genes, in both 2 and 3-dimensions were conduct
    End point values
    		 Microdose melphalan
    Number of subjects analysed
    6
    Units: Genes
    6
    Attachments
    		 abstract
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    No adverse events reported
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 SNOMED CT
    Dictionary version
    Sep 2015
    Reporting groups
    Reporting group title
    Any adverse event
    Reporting group description
    		 Any adverse event

    Serious adverse events
    		 Any adverse event
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (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
    		 Any adverse event
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 6 (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: No adverse or serious adverse events were observed in any patient in the study

    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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