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    The EU Clinical Trials Register currently displays   42570   clinical trials with a EudraCT protocol, of which   7009   are clinical trials conducted with subjects less than 18 years old.
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    Clinical Trial Results:
    An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)

    Summary
    EudraCT number
    2015-000906-20
    Trial protocol
    GB  
    Global end of trial date
    24 Jul 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Nov 2019
    First version publication date
    27 Nov 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    LUM001-601
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Mirum Pharmaceuticals, Inc. (Transferred from Shire to Mirum)
    Sponsor organisation address
    950 Tower Lane, Foster City, United States, CA 94404
    Public contact
    Chief Scientific Officer, Mirum Pharmaceuticals, Inc., medinfo@mirumpharma.com
    Scientific contact
    Chief Scientific Officer, Mirum Pharmaceuticals, Inc., medinfo@mirumpharma.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
    24 Jul 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    24 Jul 2015
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of this trial is to evaluate the long-term safety of LUM001 in patients with ALGS or PFIC. Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered in Population of Trial Subjects section since ‘0’ could not be entered due to EudraCT system constraints.
    Protection of trial subjects
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered in Population of Trial Subjects section since ‘0’ could not be entered due to EudraCT system constraints.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    24 Jul 2015
    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
    United Kingdom: 999999
    Worldwide total number of subjects
    999999
    EEA total number of subjects
    999999
    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)
    999999
    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
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.

    Period 1
    Period 1 title
    Period 1 (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Arm title
    LUM001
    Arm description
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.
    Arm type
    Experimental

    Investigational medicinal product name
    LUM001
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.

    Number of subjects in period 1
    LUM001
    Started
    999999
    Completed
    0
    Not completed
    999999
         Study was withdrawn with 0 participants
    999999

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    LUM001
    Reporting group description
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.

    Reporting group values
    LUM001 Total
    Number of subjects
    999999 999999
    Age categorical
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.
    Units: Subjects
        Age Categorical
    999999 999999
    Gender categorical
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.
    Units: Subjects
        All
    999999 999999

    End points

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    End points reporting groups
    Reporting group title
    LUM001
    Reporting group description
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started. ‘999999’ was entered since ‘0’ could not be entered due to EudraCT system constraints.

    Primary: Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [1]
    End point description
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
    End point type
    Primary
    End point timeframe
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
    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: Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
    End point values
    LUM001
    Number of subjects analysed
    0 [2]
    Units: Number of participants
    Notes
    [2] - Study was withdrawn with 0 participants.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
    Adverse event reporting additional description
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    NA
    Frequency threshold for reporting non-serious adverse events: 5%
    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: Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.

    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.
    Study was withdrawn with 0 participants. There is still no EudraCT functionality to inform the public that recruitment never started.
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