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    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multiple-dose Phase 2 Study to Evaluate the Efficacy and Safety of BMS-986263 in Adults with Compensated Cirrhosis from Nonalcoholic Steatohepatitis (NASH)

    Summary
    EudraCT number
    		 2019-003932-22
    Trial protocol
    		 FR   BE   DE   IT   NL  
    Global end of trial date
    09 Feb 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Aug 2024
    First version publication date
    28 Aug 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    IM025-017
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bristol-Myers Squibb
    Sponsor organisation address
    Chaussée de la Hulpe 185, Brussels, Belgium, 1170
    Public contact
    EU Study Start-Up Unit, Bristol-Myers Squibb International Corporation, Clinical.Trials@bms.com
    Scientific contact
    Bristol-Myers Squibb Study Director, Bristol-Myers Squibb, Clinical.Trials@bms.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
    09 Feb 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Feb 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of BMS-986263 compared with placebo to improve liver fibrosis in participants with compensated cirrhosis due to NASH
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization Good Clinical Practice Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Feb 2021
    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
    Israel: 2
    Country: Number of subjects enrolled
    Japan: 18
    Country: Number of subjects enrolled
    Korea, Republic of: 4
    Country: Number of subjects enrolled
    Taiwan: 1
    Country: Number of subjects enrolled
    Belgium: 4
    Country: Number of subjects enrolled
    Germany: 9
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Spain: 7
    Country: Number of subjects enrolled
    Switzerland: 1
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Canada: 2
    Country: Number of subjects enrolled
    United States: 52
    Country: Number of subjects enrolled
    Argentina: 7
    Country: Number of subjects enrolled
    Brazil: 10
    Worldwide total number of subjects
    124
    EEA total number of subjects
    24
    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)
    88
    From 65 to 84 years
    36
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 124 Subjects Randomized 122 Treated

    Period 1
    Period 1 title
    Randomization
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    Placebo

    Arm title
    		 Treatment 1
    Arm description
    		 BMS-986263 45mg QW
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMS-986263
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    10 mg per vial; 3 mg/mL

    Arm title
    		 Treatment 2
    Arm description
    		 BMS-986263 90mg QW
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMS-986263
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    10 mg per vial; 3 mg/mL

    Number of subjects in period 1
    		Placebo Treatment 1 Treatment 2
    Started
    40
    42
    42
    Completed
    39
    41
    42
    Not completed
    1
    1
    0
         Adverse event, non-fatal
    1
    -
    -
         Administrative Reasons by Sponsor
    -
    1
    -
    Period 2
    Period 2 title
    Treatment Period
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    Placebo

    Arm title
    		 Treatment 1
    Arm description
    		 BMS-986263 45mg QW
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMS-986263
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    10 mg per vial; 3 mg/mL

    Arm title
    		 Treatment 2
    Arm description
    		 BMS-986263 90mg QW
    Arm type
    		 Experimental

    Investigational medicinal product name
    BMS-986263
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravascular use
    Dosage and administration details
    10 mg per vial; 3 mg/mL

    Number of subjects in period 2
    		Placebo Treatment 1 Treatment 2
    Started
    39
    41
    42
    Completed
    36
    35
    35
    Not completed
    3
    6
    7
         Adverse event, non-fatal
    -
    2
    3
         Participant Withdrew Consent
    -
    -
    1
         Administrative Reasons by Sponspor
    3
    4
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    Treatment 1
    Reporting group description
    		 BMS-986263 45mg QW

    Reporting group title
    Treatment 2
    Reporting group description
    		 BMS-986263 90mg QW

    Reporting group values
    		 Placebo Treatment 1 Treatment 2 Total
    Number of subjects
    		 40 42 42 124
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 31 32 25 88
        From 65-84 years
    		 9 10 17 36
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 58.9 ( 8.56 ) 58.9 ( 6.76 ) 60.0 ( 8.65 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 22 25 25 72
        Male
    		 18 17 17 52
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0
        Asian
    		 6 11 9 26
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Black or African American
    		 0 0 1 1
        White
    		 34 31 32 97
        More than one race
    		 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 22 13 18 53
        Not Hispanic or Latino
    		 7 18 17 42
        Unknown or Not Reported
    		 11 11 7 29

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    Treatment 1
    Reporting group description
    		 BMS-986263 45mg QW

    Reporting group title
    Treatment 2
    Reporting group description
    		 BMS-986263 90mg QW
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    Treatment 1
    Reporting group description
    		 BMS-986263 45mg QW

    Reporting group title
    Treatment 2
    Reporting group description
    		 BMS-986263 90mg QW

    Primary: Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score), as determined by liver biopsy after 12 weeks of treatment.

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    End point title
    		 Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score), as determined by liver biopsy after 12 weeks of treatment. [1]
    End point description
    Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score), as determined by liver biopsy after 12 weeks of treatment. For the NASH CRN Fibrosis Score, fibrosis is staged on a 0 to 4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
    End point type
    Primary
    End point timeframe
    12 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: No Statistical Analysis done for this endpoint
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Percentage
    number (not applicable)
        responders
    20.5
    12.2
    7.1
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment.

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    End point title
    		 Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment.
    End point description
    Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment. For the NASH CRN Fibrosis Score, fibrosis is staged on a 0 to 4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
    End point type
    Secondary
    End point timeframe
    12 Weeks
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Percentage
    number (not applicable)
        responders
    20.5
    12.2
    4.8
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment.

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    End point title
    		 Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment.
    End point description
    Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (NASH CRN Fibrosis Score) with no worsening of NASH after 12 weeks of treatment. For the NASH CRN Fibrosis Score, fibrosis is staged on a 0 to 4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
    End point type
    Secondary
    End point timeframe
    12 Weeks
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Percentage
    number (not applicable)
        responders
    2.6
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment.

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    End point title
    		 Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment.
    End point description
    Percentage of participants who achieve ≥ 1 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment. A modified Ishak scoring system (0 to 6 scale) was originally developed tograde portal-based liver fibrosis associated with viral hepatitis. The modified Ishak system has been adapted to grade central-based liver fibrosis associated with NASH, and it also uses a 0 to 6 scale: 0: No fibrosis 1: perisinusoidal or periportal fibrosis 2: perisinusoidal and portal/periportal fibrosis 3: bridging fibrosis with linkage of < 50% of vascular structures (portal and centrilobular) 4: bridging fibrosis with linkage of > 50% of vascular structures (portal and centrilobular) 5: early or incomplete cirrhosis 6: established or advanced cirrhosis
    End point type
    Secondary
    End point timeframe
    12 Weeks
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Percentage
    number (not applicable)
        Responders
    30.8
    26.8
    21.4
    No statistical analyses for this end point

    Secondary: Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment.

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    End point title
    		 Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment.
    End point description
    Percentage of participants who achieve ≥ 2 stage improvement in liver fibrosis (modified Ishak score) after 12 weeks of treatment. A modified Ishak scoring system (0 to 6 scale) was originally developed tograde portal-based liver fibrosis associated with viral hepatitis. The modified Ishak system has been adapted to grade central-based liver fibrosis associated with NASH, and it also uses a 0 to 6 scale: 0: No fibrosis 1: perisinusoidal or periportal fibrosis 2: perisinusoidal and portal/periportal fibrosis 3: bridging fibrosis with linkage of < 50% of vascular structures (portal and centrilobular) 4: bridging fibrosis with linkage of > 50% of vascular structures (portal and centrilobular) 5: early or incomplete cirrhosis 6: established or advanced cirrhosis
    End point type
    Secondary
    End point timeframe
    12 Weeks
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Percentage
    number (not applicable)
        responders
    10.3
    4.9
    4.8
    No statistical analyses for this end point

    Secondary: Mean change from baseline in CPA after 12 weeks of Treatment

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    End point title
    		 Mean change from baseline in CPA after 12 weeks of Treatment
    End point description
    Change from baseline in CPA after 12 weeks of treatment. Assessment of collagen proportionate area(CPA) is a method by which the amount (percentage) of collagen in stained tissue sections is analyzed using morphometric image analysis. This allows for a quantitative assessment of fibrosis. Percentage of fat in stained tissue sections is also analyzed using morphometric image analysis.
    End point type
    Secondary
    End point timeframe
    12 Weeks
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    35
    32
    27
    Units: Percentage
        arithmetic mean (standard error)
    -3.67 ( 1.861 )
    -0.35 ( 1.374 )
    -3.67 ( 1.973 )
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment Emergent Adverse Events (TEAE) and Treatment Emergent Serious Adverse Events (TESAE)

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    End point title
    		 Number of Participants with Treatment Emergent Adverse Events (TEAE) and Treatment Emergent Serious Adverse Events (TESAE)
    End point description
    An AE is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug and that does have a causal relationship with this treatment.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
        TEAE
    24
    33
    34
        TESAE
    1
    1
    2
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant changes in clinical laboratory values.

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    End point title
    		 Number of Participants with clinically significant changes in clinical laboratory values.
    End point description
    Investigators must document their review of each laboratory safety report. A central laboratory will perform the analyses and will provide reference ranges for these tests. clinical laboratory assessments analyzed: Hematology, Blood Chemistry, Urinalysis and a Metabolic Panel.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant changes in vitals signs.

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    End point title
    		 Number of Participants with clinically significant changes in vitals signs.
    End point description
    Includes body temperature, respiratory rate, blood pressure, and heart rate. Blood pressure and heart rate should be measured after the participant has been resting quietly for at least 5 minutes.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant changes in physical examination findings.

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    End point title
    		 Number of Participants with clinically significant changes in physical examination findings.
    End point description
    Physical examination includes body weight, height, and BMI (height and BMI calculation at screening only).
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant changes in electrocardiogram readings.

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    End point title
    		 Number of Participants with clinically significant changes in electrocardiogram readings.
    End point description
    Number of Participants with clinically significant changes in electrocardiogram readings.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with clinically significant changes in BMD.

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    End point title
    		 Number of Participants with clinically significant changes in BMD.
    End point description
    Bone Mineral Density(BMD) will be measured by a dual-energy X-ray absorptiometry (DXA) Scan.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    39
    41
    42
    Units: Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Plasma Concentration of BMS-986263 components at the end of 12 weeks.

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    End point title
    		 Plasma Concentration of BMS-986263 components at the end of 12 weeks.
    End point description
    Plasma concentrations of BMS-986263 components siRNA, DPD, HEDC, and S104.
    End point type
    Secondary
    End point timeframe
    From First Treatment to end of Follow up (36 weeks)
    End point values
    		 Placebo Treatment 1 Treatment 2
    Number of subjects analysed
    0 [2]
    42
    42
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        siRNA
    ( )
    38.9 ( 151 )
    72.9 ( 169 )
        DPD
    ( )
    519 ( 45.2 )
    1138 ( 80.7 )
        HEDC
    ( )
    25.1 ( 51.8 )
    59.0 ( 58.8 )
        S104
    ( )
    4.16 ( 141 )
    3.68 ( 50.7 )
    Notes
    [2] - Subjects in this arm not given treatment drug to have components
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events and Serious Adverse Events: (From first dose to last dose + 24 week follow up): Approximately 36 Weeks All-Cause mortality (From randomization to end of study): Approximately 42 Weeks.
    Adverse event reporting additional description
    The number at Risk for All-Cause Mortality represents all Randomized Participants. The number at Risk for Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    PLACEBO
    Reporting group description
    		 Placebo

    Reporting group title
    Treatment 2
    Reporting group description
    		 BMS-986263 90mg QW

    Reporting group title
    Treatment 1
    Reporting group description
    		 BMS-986263 45mg QW

    Serious adverse events
    		 PLACEBO Treatment 2 Treatment 1
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 39 (2.56%)
    2 / 42 (4.76%)
    1 / 41 (2.44%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Post procedural complication
         subjects affected / exposed
    1 / 39 (2.56%)
    0 / 42 (0.00%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Post procedural haematoma
         subjects affected / exposed
    0 / 39 (0.00%)
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Procedural complication
         subjects affected / exposed
    0 / 39 (0.00%)
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    0 / 39 (0.00%)
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Infected cyst
         subjects affected / exposed
    0 / 39 (0.00%)
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 PLACEBO Treatment 2 Treatment 1
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 39 (43.59%)
    29 / 42 (69.05%)
    24 / 41 (58.54%)
    Injury, poisoning and procedural complications
    Infusion related reaction
         subjects affected / exposed
    0 / 39 (0.00%)
    22 / 42 (52.38%)
    17 / 41 (41.46%)
         occurrences all number
    0
    70
    44
    Procedural pain
         subjects affected / exposed
    2 / 39 (5.13%)
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences all number
    2
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 39 (10.26%)
    6 / 42 (14.29%)
    4 / 41 (9.76%)
         occurrences all number
    6
    12
    13
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    2 / 39 (5.13%)
    0 / 42 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    2
    0
    0
    Fatigue
         subjects affected / exposed
    2 / 39 (5.13%)
    4 / 42 (9.52%)
    1 / 41 (2.44%)
         occurrences all number
    2
    15
    1
    Gastrointestinal disorders
    Gastrooesophageal reflux disease
         subjects affected / exposed
    2 / 39 (5.13%)
    0 / 42 (0.00%)
    0 / 41 (0.00%)
         occurrences all number
    5
    0
    0
    Nausea
         subjects affected / exposed
    3 / 39 (7.69%)
    1 / 42 (2.38%)
    2 / 41 (4.88%)
         occurrences all number
    5
    1
    3
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    2 / 39 (5.13%)
    1 / 42 (2.38%)
    1 / 41 (2.44%)
         occurrences all number
    2
    1
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 39 (5.13%)
    2 / 42 (4.76%)
    2 / 41 (4.88%)
         occurrences all number
    2
    2
    2
    Back pain
         subjects affected / exposed
    2 / 39 (5.13%)
    2 / 42 (4.76%)
    2 / 41 (4.88%)
         occurrences all number
    2
    3
    2
    Muscle spasms
         subjects affected / exposed
    3 / 39 (7.69%)
    1 / 42 (2.38%)
    1 / 41 (2.44%)
         occurrences all number
    3
    1
    1
    Myalgia
         subjects affected / exposed
    4 / 39 (10.26%)
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences all number
    7
    0
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    4 / 39 (10.26%)
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    4
    1
    4
    Urinary tract infection
         subjects affected / exposed
    2 / 39 (5.13%)
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences all number
    2
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Nov 2022
    The primary purpose of this Global Revised Protocol is to expand the patient population from Child-Pugh A5 to Child-Pugh A6, including corresponding laboratory exclusion criteria, based on preliminary results from hepatic impairment (HI) study IM025015. Key secondary revisions include the following:  Adding results from Part 1 of HI study IM025015, which supports the changes in Child- Pugh/laboratory exclusion criteria  Adjustment of step-wise infusion rates (decreased number of steps)  Adding a ({Bis[2-(tetradecanoyloxy)ethyl] carbamoyl}methyl)-(2- hydroxyethyl)dimethylazanium bromide (HEDC; lipid component) assay  Adding guidance and exploratory assessments related to the coronavirus disease 2019 (COVID-19) pandemic  Changing statistical sample size calculation, study stratification factors, and statistical analysis methodology for primary endpoint  Excluding participants from the study who are taking anticoagulants  Replacing liver ultrasound as a screening procedure for detection of hepatocellular carcinoma (HCC) with multiphasic liver computed tomography (CT)/magnetic resonance imaging (MRI)  Updating exclusion criterion related to history of weight gain/loss  Updating exclusion criterion related to history of illegal intravenous (IV) drug use  Adding option for participants who have a prolonged international normalized ratio (INR) and/or lower platelet count to potentially receive treatments for coagulation abnormalities and/or low platelet counts prior to liver biopsy

    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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