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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of NGM282 Administered for 12 Weeks in Patients with Primary Sclerosing Cholangitis (PSC).

    Summary
    EudraCT number
    		 2015-003392-30
    Trial protocol
    		 GB   NL  
    Global end of trial date
    07 Jun 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Jul 2018
    First version publication date
    15 Jul 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    15-0106
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    NGM Biopharmaceuticals, Inc.
    Sponsor organisation address
    333 Oyster Point Boulevard, South San Francisco, United States, CA 94080
    Public contact
    Clinical Operations , NGM Biopharmaceuticals, Inc., 001 6502435555, clinical@ngmbio.com
    Scientific contact
    Clinical Operations , NGM Biopharmaceuticals, Inc., 001 6502435555, clinical@ngmbio.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
    25 Apr 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    07 Jun 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Jun 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate the treatment effect of NGM282 as measured by the mean change in alkaline phosphatase (ALP) from Baseline to Week 12 in patients with Primary Sclerosing Cholangitis (PSC).
    Protection of trial subjects
    This study was conducted under the auspices of a data monitoring committee (DMC) to protect subject welfare, preserve study integrity, and provide recommendations as needed regarding study conduct. This study was conducted in compliance with ICH E6 GCP: Consolidated Guidelines pertaining to informed consent.
    Background therapy
    		 Subjects taking medications for IBD (Inflammatory bowel disease - frequently associated with PSC) must have been on a stable regimen of these medications for at least 12 weeks before Day 1 and were to maintain, if possible, a stable dose during the study period. Subjects taking UDCA (ursodeoxycholic acid) were eligible but must have been on stable doses of <27 mg/kg/day for at least 12 weeks before their screening. No significant dosage changes were to be made during 8 weeks prior to screening and a minimum 8-week washout period was to occur before screening if UDCA was stopped. Subjects not taking UDCA were not to start it during the study period.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    23 Feb 2016
    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
    Netherlands: 13
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    France: 6
    Country: Number of subjects enrolled
    United States: 36
    Worldwide total number of subjects
    62
    EEA total number of subjects
    26
    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)
    57
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects studied had to have confirmed PSC as defined by an elevated ALP and either liver histology or cholangiography consistent with PSC. The presence of IBD was allowed, as well as treatment with a stable regimen of biologic, immunosuppressant, or systemic corticosteroid therapy.

    Pre-assignment
    Screening details
    		 A total of 95 subjects were assessed for eligibility after giving informed consent and 62 subjects were randomly assigned to treatment, including 21 subjects to 1-mg NGM282, 21 subjects to 3-mg NGM282, and 20 subjects to placebo.

    Period 1
    Period 1 title
    Period 1 (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor
    Blinding implementation details
    Syringes containing solutions of NGM282 and placebo were identical in packaging, appearance, and volume of solution. Study drug was coded to preserve blinding.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 1-mg NGM282
    Arm description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    NGM282
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    On Day 1 of the study, subjects were trained in study drug administration and were observed self-administering study drug in the clinic by clinic staff. On Weeks 1, 2, 4, 8, and 12, self-administration also occurred in the clinic under observation by clinic staff. All other doses through Week 12 were self-administered at home. Written instructions for study drug preparation and self-administration were provided to each subject and retraining was provided as required at clinic visits. Subjects were instructed to bring study drug syringes to room temperature before use. Subjects were required to complete a daily study drug administration diary and return previously dispensed kits of study drug at each clinic visit during the study period after Day 1.

    Arm title
    		 3-mg NGM282
    Arm description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    NGM282
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    On Day 1 of the study, subjects were trained in study drug administration and were observed self-administering study drug in the clinic by clinic staff. On Weeks 1, 2, 4, 8, and 12, self-administration also occurred in the clinic under observation by clinic staff. All other doses through Week 12 were self-administered at home. Written instructions for study drug preparation and self-administration were provided to each subject and retraining was provided as required at clinic visits. Subjects were instructed to bring study drug syringes to room temperature before use. Subjects were required to complete a daily study drug administration diary and return previously dispensed kits of study drug at each clinic visit during the study period after Day 1.

    Arm title
    		 Placebo
    Arm description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Study drug was provided as a sterile solution for injection in single-use prefilled syringes for SC administration of 1-mg NGM282, 3-mg NGM282, or placebo. On Day 1 of the study, subjects were trained in study drug administration and were observed self-administering study drug in the clinic by clinic staff. On Weeks 1, 2, 4, 8, and 12, self-administration also occurred in the clinic under observation by clinic staff. All other doses through Week 12 were self-administered at home. Written instructions for study drug preparation and self-administration were provided to each subject and retraining was provided as required at clinic visits. Subjects were instructed to bring study drug syringes to room temperature

    Number of subjects in period 1
    		1-mg NGM282 3-mg NGM282 Placebo
    Started
    21
    21
    20
    Completed
    19
    18
    19
    Not completed
    2
    3
    1
         Other
    -
    1
    1
         Adverse event
    1
    1
    -
         Noncompliance with study drug
    1
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    1-mg NGM282
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Reporting group title
    3-mg NGM282
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Reporting group title
    Placebo
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Reporting group values
    		 1-mg NGM282 3-mg NGM282 Placebo Total
    Number of subjects
    		 21 21 20 62
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0
        Adults (18-64 years)
    		 18 21 19 58
        From 65-84 years
    		 3 0 1 4
        85 years and over
    		 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 7 9 8 24
        Male
    		 14 12 12 38
    Subject analysis sets

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Analyses on the per protocol (PP) set were used as a supplement to the FAS analyses and were performed for all efficacy endpoints. The PP set included all subjects in the FAS who had at least 1 valid, non-missing post dose ALP measurement and excluded the following subjects: - FAS subjects who deviated from the conduct of the study, as adjudicated by the sponsor’s medical monitor - FAS subjects who had an AE deemed by the medical monitor to be impactful on the primary endpoint In associated analyses, subjects were grouped according to actual treatment received, even if it differed from the assigned treatment.

    Subject analysis set title
    Full Analysis
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All randomized subjects who received at least 1 dose (full or partial) of study drug and had at least 1 valid, non-missing post dose efficacy parameter value were included in the full analysis set (FAS). This was the set for the primary analyses of efficacy endpoints. In the FAS analyses, subjects were grouped according to the assigned treatment if this differed from the actual treatment received.

    Subject analysis sets values
    		 Per Protocol Set Full Analysis
    Number of subjects
    48
    62
    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)
    57
    44
        From 65-84 years
    5
    4
        85 years and over
    0
    0
    Age continuous
    Units:
        
    ( )
    ( )
    Gender categorical
    Units: Subjects
        Female
    20
    24
        Male
    28
    38

    End points

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    End points reporting groups
    Reporting group title
    1-mg NGM282
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Reporting group title
    3-mg NGM282
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Reporting group title
    Placebo
    Reporting group description
    		 Subjects self-administered study drug (1-mg NGM282, 3-mg NGM282, or matching placebo) as a subcutaneous injection to the abdomen daily, at approximately the same time each morning, over the 12-week treatment period.

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Analyses on the per protocol (PP) set were used as a supplement to the FAS analyses and were performed for all efficacy endpoints. The PP set included all subjects in the FAS who had at least 1 valid, non-missing post dose ALP measurement and excluded the following subjects: - FAS subjects who deviated from the conduct of the study, as adjudicated by the sponsor’s medical monitor - FAS subjects who had an AE deemed by the medical monitor to be impactful on the primary endpoint In associated analyses, subjects were grouped according to actual treatment received, even if it differed from the assigned treatment.

    Subject analysis set title
    Full Analysis
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All randomized subjects who received at least 1 dose (full or partial) of study drug and had at least 1 valid, non-missing post dose efficacy parameter value were included in the full analysis set (FAS). This was the set for the primary analyses of efficacy endpoints. In the FAS analyses, subjects were grouped according to the assigned treatment if this differed from the actual treatment received.

    Primary: The mean change in ALP from Baseline at Week 12

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    End point title
    		 The mean change in ALP from Baseline at Week 12
    End point description
    The primary analysis was performed to evaluate the treatment effect of NGM282. The mean change from Baseline at Week 12 in ALP was compared between each of the 2 active treatment groups (1-mg or 3-mg NGM282) and the placebo group using the Wilcoxon Rank Sum test. Sensitivity analyses for the primary endpoint used a mixed-effect model repeated measures (MMRM) analysis of covariance (ANCOVA).
    End point type
    Primary
    End point timeframe
    The mean change in ALP from Baseline at Week 12 will be compared between each of the two treatment groups (NGM282 1 mg or 3 mg, as applicable) and the placebo group.
    End point values
    		 1-mg NGM282 3-mg NGM282 Placebo
    Number of subjects analysed
    21
    21
    20
    Units: Mean change in ALP
        number (confidence interval 95%)
    21 (-21.1 to 62)
    21 (-56 to 26.8)
    20 (-38.3 to 48)
    Statistical analysis title
    		 SAP, dated 26 May 2017
    Statistical analysis description
    Mixed-effect model repeated measures (MMRM) analysis of covariance (ANCOVA) of mean change in ALP from Baseline at Week 12 will be used to compare the difference between treatment groups. The ANCOVA on the FAS was consistent with the Wilcoxon Rank Sum test and the MMRM analysis.
    Comparison groups
    1-mg NGM282 v Placebo v 3-mg NGM282
    Number of subjects included in analysis
    62
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority [1]
    P-value
    		 = 0.511 [2]
    Method
    		 ANCOVA/Wilcoxon Rank Sum/MMRM
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -8.7
         upper limit
    		 17.27
    Variability estimate
    Standard deviation
    Notes
    [1] - There was no significant mean percent change from Baseline in ALP in any treatment group at Week 12. There was no significant difference between either the 1-mg or 3-mg dose of NGM282 and placebo, and there was also no significant difference in efficacy between the 1-mg and the 3-mg doses.
    [2] - The difference between groups was estimated using the Hodges-Lehmann estimate and confidence interval, and the P-value was computed from the normal approximation of the Wilcoxon Rank Sum test.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    A TEAE was defined as an AE that began on or after the first dose of study drug and before the stop of study drug +30 days.
    Adverse event reporting additional description
    A TEAE was defined as an AE that met any of the following conditions: - Was completely missing a start date and end date - Was completely missing a start date and the end date was on or after the first dose of study drug
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    1-mg NGM282
    Reporting group description
    		 -

    Reporting group title
    3-mg NGM282
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 1-mg NGM282 3-mg NGM282 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 20 (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
    Gastrointestinal disorders
    Bowel obstruction
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (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
    Hepatobiliary disorders
    Sclerosing cholangitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 20 (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
    Infections and infestations
    Intervertebral discitis
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 1-mg NGM282 3-mg NGM282 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 21 (80.95%)
    20 / 21 (95.24%)
    18 / 20 (90.00%)
    Cardiac disorders
    ANGINA PECTORIS
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Nervous system disorders
    BURNING SENSATION
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    DIZZINESS
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    1
    0
    2
    HEADACHE
         subjects affected / exposed
    0 / 21 (0.00%)
    4 / 21 (19.05%)
    3 / 20 (15.00%)
         occurrences all number
    0
    4
    3
    PARAESTHESIA
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    CHEST PAIN
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    FATIGUE
         subjects affected / exposed
    1 / 21 (4.76%)
    3 / 21 (14.29%)
    3 / 20 (15.00%)
         occurrences all number
    1
    3
    3
    INJECTION SITE ERYTHEMA
         subjects affected / exposed
    2 / 21 (9.52%)
    11 / 21 (52.38%)
    1 / 20 (5.00%)
         occurrences all number
    2
    11
    1
    INJECTION SITE PAIN
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
         occurrences all number
    1
    1
    2
    MALAISE
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    PYREXIA
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Immune system disorders
    SARCOIDOSIS
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Eye disorders
    DRY EYE
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    ABDOMINAL CRAMPS
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    1
    0
    2
    ABDOMINAL DISTENSION
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    DIARRHEA
         subjects affected / exposed
    5 / 21 (23.81%)
    4 / 21 (19.05%)
    0 / 20 (0.00%)
         occurrences all number
    5
    4
    0
    DRY MOUTH
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    0
    DYSPEPSIA AGGRAVATED
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    0
    Frequent bowel movements
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    INCREASED STOOL FREQUENCY
         subjects affected / exposed
    1 / 21 (4.76%)
    3 / 21 (14.29%)
    0 / 20 (0.00%)
         occurrences all number
    1
    3
    0
    LOOSE STOOLS
         subjects affected / exposed
    4 / 21 (19.05%)
    2 / 21 (9.52%)
    1 / 20 (5.00%)
         occurrences all number
    4
    2
    1
    NAUSEA
         subjects affected / exposed
    2 / 21 (9.52%)
    2 / 21 (9.52%)
    3 / 20 (15.00%)
         occurrences all number
    2
    2
    3
    NAUSEA AGGRAVATED
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    1
    UMBILICAL HERNIA
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    VOMITING
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    1
    Respiratory, thoracic and mediastinal disorders
    DYSPNOEA
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    NASAL CONGESTION
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    0
    2
    OROPHARYNGEAL PAIN
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
         occurrences all number
    1
    1
    2
    Skin and subcutaneous tissue disorders
    PRURITUS
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
         occurrences all number
    1
    1
    2
    PRURITUS GENERALISED
         subjects affected / exposed
    2 / 21 (9.52%)
    2 / 21 (9.52%)
    2 / 20 (10.00%)
         occurrences all number
    2
    2
    2
    RASH MACULAR
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    SKIN IRRITATION
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    INSOMNIA
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    IRRITABILITY
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    INFLUENZA
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    1
    NASOPHARYNGITIS
         subjects affected / exposed
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    4 / 20 (20.00%)
         occurrences all number
    1
    2
    4
    RHINITIS
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    URINARY TRACT INFECTION
         subjects affected / exposed
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    Metabolism and nutrition disorders
    INCREASED APPETITE
         subjects affected / exposed
    4 / 21 (19.05%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
         occurrences all number
    4
    1
    0
    VITAMIN D DEFICIENCY
         subjects affected / exposed
    3 / 21 (14.29%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
         occurrences all number
    3
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Nov 2015
    The following is a summary of the major changes addressed with Protocol Amendment 1, dated 05 Nov 2015: - Calprotectin analysis was added as a study objective, replacing “intestinal inflammatory biomarkers.” - Total cholesterol, HDL cholesterol, LDL cholesterol, and triglycerides were added as secondary efficacy endpoints. - References to transient elastography were removed. - Various laboratory parameter range inclusion criteria were updated. - The inclusion/exclusion criteria for UDCA use and MRCP findings were updated. - The exclusion criteria were updated to permit subjects to enter the study who were taking antibiotics for the prevention or presumptive treatment of cholangitis and who had compensated cirrhosis. - Clarified that a colonoscopy was to be performed only in subjects with concomitant IBD who did not have a colonoscopy available within 12 months before screening. - Criteria for discontinuation of study treatment for an individual subject were updated, updating threshold levels for ALT, ALP, and total bilirubin. - Additional time points were added for assessment of itch, fatigue, antidrug antibodies, and neutralizing antibodies.
    27 May 2016
    The following is a summary of the major changes addressed with Protocol Amendment 2, dated 27 May 2016: - “Exploratory markers of fibrogenesis” was added as an exploratory objective. - Inclusion criteria were updated, including the criteria related to the confirmed diagnosis of PSC, carbohydrate antigen 19-9 (CA19-9), UDCA treatment use, concomitant IBD, and platelet levels. - Exclusion criteria were updated, including criteria related to liver disease, MRCP findings, bile duct stenting or drains, acute cholangitis, and decompensated cirrhosis. - Study procedures were updated. - Reporting of local injection-site symptom assessments was clarified. - The interim analysis (IA) procedure was clarified, as was the timing of the EOS visit.

    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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