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    Clinical Trial Results:
    A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)

    Summary
    EudraCT number
    		 2014-004856-68
    Trial protocol
    		 NO   NL   FR  
    Global end of trial date
    15 Feb 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Mar 2017
    First version publication date
    26 Mar 2017
    Other versions
    Summary report(s)
    		 Synopsis

    Trial information

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    Trial identification
    Sponsor protocol code
    CB8025-21427
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02472535
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    CymaBay Therapeutics, Inc
    Sponsor organisation address
    7999 Gateway Blvd, suite 130, Newark, CA, United States, 94560
    Public contact
    Pol Boudes, CymaBay Therapeutics, Inc., 001 5102938815, pboudes@cymabay.com
    Scientific contact
    Pol Boudes, CymaBay Therapeutics, Inc., 001 5102938815, pboudes@cymabay.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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    15 Feb 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    15 Feb 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Feb 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Primary: To evaluate the effect of MBX-8025 on Low Density Lipoprotein Cholesterol (LDL-C).
    Protection of trial subjects
    If AEs were severe and drug related, the subject stopped treatment and entered the follow-up period.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Apr 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
    Norway: 2
    Country: Number of subjects enrolled
    Canada: 5
    Country: Number of subjects enrolled
    Netherlands: 2
    Country: Number of subjects enrolled
    France: 4
    Worldwide total number of subjects
    13
    EEA total number of subjects
    8
    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)
    11
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The screening period was a maximum of 2 weeks. All subjects had to confirm eligibility on Visit 2 (Week 0) prior to entering the run-in period

    Period 1
    Period 1 title
    2 weeks screening period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 single-arm
    Arm description
    		 -
    Arm type
    		 Cohort

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		single-arm
    Started
    13
    Completed
    13
    Period 2
    Period 2 title
    Run-in period
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 2 weeks treatment with placebo
    Arm description
    		 2 weeks treatment with placebo
    Arm type
    		 Cohort

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The placebo treatment was administered PO once daily and only during the run-in period. The placebo was a gelatin capsule, containing all ingredients in the investigational product, except for the active pharmaceutical ingredient, MBX-8025. Only 1 batch of placebo was used: batch number 14G058.

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Period 2 is the Baseline period
    Number of subjects in period 2
    		2 weeks treatment with placebo
    Started
    13
    Completed
    13
    Period 3
    Period 3 title
    Treatment Phase 1
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 4 weeks treatment with 50 mg daily
    Arm description
    		 4 weeks treatment with 50 mg daily
    Arm type
    		 Cohort

    Investigational medicinal product name
    MBX-8025
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The test product was MBX-8025 capsules (50 mg). Dosing was oral (PO), once daily, in doses of 50 mg (first 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059.

    Number of subjects in period 3
    		4 weeks treatment with 50 mg daily
    Started
    13
    Completed
    13
    Period 4
    Period 4 title
    Treatment Phase 2
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 4 weeks treatment with 100 mg daily
    Arm description
    		 4 weeks treatment with 100 mg daily
    Arm type
    		 Cohort

    Investigational medicinal product name
    MBX-8025
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The test product was MBX-8025 capsules (50 or 100 mg). Dosing was oral (PO), once daily, in doses of 100 or 50 mg (second 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059, and the 100 mg MBX-8025 test product was batch number 14G060.

    Number of subjects in period 4
    		4 weeks treatment with 100 mg daily
    Started
    13
    Completed
    13
    Period 5
    Period 5 title
    Treatment Phase 3
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 4 weeks treatment with 200 mg daily
    Arm description
    		 4 weeks treatment with 200 mg daily
    Arm type
    		 Cohort

    Investigational medicinal product name
    MBX-8025
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The test product was MBX-8025 capsules (50 or 100 mg). Dosing was oral (PO), once daily, in doses of 200, 100, or 50 (third 4-week treatment period). The 50 mg MBX-8025 test product was batch number 14G059, and the 100 mg MBX-8025 test product was batch number 14G060.

    Number of subjects in period 5
    		4 weeks treatment with 200 mg daily
    Started
    13
    Completed
    13
    Period 6
    Period 6 title
    Follow-up period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 2 weeks follow-up
    Arm description
    		 2 weeks follow-up
    Arm type
    		 Cohort

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 6
    		2 weeks follow-up
    Started
    13
    Completed
    13

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Run-in period
    Reporting group description
    		 -

    Reporting group values
    		 Run-in period Total
    Number of subjects
    		 13 13
    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)
    		 11 11
        From 65-84 years
    		 2 2
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 41.2 (18 to 85) -
    Gender categorical
    Units: Subjects
        Female
    		 5 5
        Male
    		 8 8

    End points

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    End points reporting groups
    Reporting group title
    single-arm
    Reporting group description
    		 -
    Reporting group title
    2 weeks treatment with placebo
    Reporting group description
    		 2 weeks treatment with placebo
    Reporting group title
    4 weeks treatment with 50 mg daily
    Reporting group description
    		 4 weeks treatment with 50 mg daily
    Reporting group title
    4 weeks treatment with 100 mg daily
    Reporting group description
    		 4 weeks treatment with 100 mg daily
    Reporting group title
    4 weeks treatment with 200 mg daily
    Reporting group description
    		 4 weeks treatment with 200 mg daily
    Reporting group title
    2 weeks follow-up
    Reporting group description
    		 2 weeks follow-up

    Primary: Safety

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    End point title
    		 Safety [1]
    End point description
    End point type
    Primary
    End point timeframe
    4 weeks
    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: Please see attached synopsis
    End point values
    		 4 weeks treatment with 50 mg daily 4 weeks treatment with 100 mg daily 4 weeks treatment with 200 mg daily
    Number of subjects analysed
    13
    13
    13
    Units: Subjects with at least one TEAE
    7
    8
    5
    No statistical analyses for this end point

    Primary: Efficacy

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    End point title
    		 Efficacy [2]
    End point description
    End point type
    Primary
    End point timeframe
    4 weeks
    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: Please see attached synopsis
    End point values
    		 4 weeks treatment with 50 mg daily 4 weeks treatment with 100 mg daily 4 weeks treatment with 200 mg daily
    Number of subjects analysed
    13
    13
    13
    Units: % Mean Maximum Change in LDL-C
        number (not applicable)
    -13.2
    -10.5
    -10.7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    From baseline through week 16
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    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: Please see attached synopsis

    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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    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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