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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Study to Investigate the Safety and Efficacy of MultiStem (PF-05285401) in Subjects With Moderate to Severe Ulcerative Colitis

    Due to the EudraCT – Results system being out of service between 31 July 2015 and 12 January 2016, these results have been published in compliance with revised timelines.
    Summary
    EudraCT number
    2010-022766-27
    Trial protocol
    SE   BE   HU   SK   IT  
    Global end of trial date
    19 Nov 2014

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Jul 2016
    First version publication date
    27 Jul 2016
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    B3041001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Pfizer, Inc.
    Sponsor organisation address
    235 E 42nd Street, New York, United States, 10017
    Public contact
    Pfizer, Inc., Pfizer ClinicalTrials.gov Call Center, 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.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
    08 Jul 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Nov 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Nov 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the trial was to evaluate the safety and tolerability of intravenous doses of MultiStem in moderate-to-severe ulcerative colitis.
    Protection of trial subjects
    The study used an independent External Data Monitoring Committee (EDMC), who was responsible for ongoing monitoring of the safety of subjects according to the Charter. The recommendations made by the EDMC to alter the conduct of the study was forwarded to Pfizer for final decision.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    28 Feb 2011
    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
    Slovakia: 5
    Country: Number of subjects enrolled
    Sweden: 9
    Country: Number of subjects enrolled
    United States: 63
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    Germany: 12
    Country: Number of subjects enrolled
    Hungary: 10
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    105
    EEA total number of subjects
    38
    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)
    95
    From 65 to 84 years
    10
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    There were 3 cohorts planned. Cohorts 1 and 2 each included 9 subjects. The size of Cohort 3 was reviewed as the study progressed, based purely upon emerging variability estimates of the primary endpoints.

    Period 1
    Period 1 title
    Overall Study
    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
    Cohort 1: Placebo, MultiStem 300
    Arm description
    Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    300

    Arm title
    Cohort 2: Placebo, MultiStem 750
    Arm description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    750

    Arm title
    Cohort 1: MultiStem 300, Placebo
    Arm description
    Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    300

    Arm title
    Cohort 1: MultiStem 300 (x3), Placebo
    Arm description
    Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    300

    Arm title
    Cohort 2: MultiStem 750, Placebo
    Arm description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    750

    Arm title
    Cohort 3: MultiStem 750, MultiStem 750
    Arm description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    750

    Arm title
    Cohort 3: MultiStem 750, Placebo
    Arm description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    750

    Arm title
    Cohort 3: Placebo, MultiStem 750
    Arm description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    750

    Arm title
    Cohort 3: Placebo, Placebo
    Arm description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    placebo

    Number of subjects in period 1
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Started
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Completed
    0
    2
    2
    1
    2
    13
    17
    15
    13
    Not completed
    2
    1
    0
    3
    4
    10
    8
    4
    8
         Insufficient Clinical Response
    -
    -
    -
    -
    1
    3
    6
    -
    3
         Adverse event, non-fatal
    -
    -
    -
    1
    -
    2
    -
    -
    1
         Unspecified
    -
    -
    -
    -
    2
    -
    -
    -
    -
         Consent withdrawn by subject
    2
    1
    -
    2
    -
    4
    2
    3
    3
         Lost to follow-up
    -
    -
    -
    -
    1
    1
    -
    1
    1
    Period 2
    Period 2 title
    Pooled Cohort 3
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Pooled MultiStem
    Arm description
    -
    Arm type
    Experimental

    Investigational medicinal product name
    MultiStem
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    350 M or 750 M

    Arm title
    Pooled Placebo
    Arm description
    -
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    placebo

    Number of subjects in period 2
    Pooled MultiStem Pooled Placebo
    Started
    48
    40
    Completed
    30
    28
    Not completed
    18
    12
         Insufficient Clinical Response
    9
    3
         Adverse event, non-fatal
    2
    1
         Consent withdrawn by subject
    6
    6
         Lost to follow-up
    1
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1: Placebo, MultiStem 300
    Reporting group description
    Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 2: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300 (x3), Placebo
    Reporting group description
    Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 2: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, MultiStem 750
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, Placebo
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo Total
    Number of subjects
    2 3 2 4 6 23 25 19 21 105
    Age categorical
    Units: Subjects
        In Utero
    0 0 0 0 0 0 0 0 0 0
        Pre-term newborn - gestational age <37 wk
    0 0 0 0 0 0 0 0 0 0
        Newborns (0-27 days)
    0 0 0 0 0 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0 0 0 0 0 0 0
        Children (2-11 years)
    0 0 0 0 0 0 0 0 0 0
        Adolescents (12-17 years)
    0 0 0 0 0 0 0 0 0 0
        Adults (18-64 years)
    1 3 1 4 6 21 23 18 18 95
        Elderly (From 65-84 years)
    1 0 1 0 0 2 2 1 3 10
        Elderly 85 years and over
    0 0 0 0 0 0 0 0 0 0
    Age Continuous |
    Units: years
        arithmetic mean (standard deviation)
    61 ± 7.1 52 ± 7 58 ± 19.8 45 ± 11.1 40 ± 14 43.4 ± 12.8 38.8 ± 13.4 39.8 ± 12.5 42.5 ± 15.7 -
    Gender, Male/Female
    Units: participants
        Male
    2 2 2 3 1 16 13 16 14 69
        Female
    0 1 0 1 5 7 12 3 7 36

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1: Placebo, MultiStem 300
    Reporting group description
    Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 2: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300 (x3), Placebo
    Reporting group description
    Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 2: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, MultiStem 750
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, Placebo
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.
    Reporting group title
    Pooled MultiStem
    Reporting group description
    -

    Reporting group title
    Pooled Placebo
    Reporting group description
    -

    Primary: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

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    End point title
    Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8
    End point description
    Modified Baron Score is an instrument designed to measure endoscopic activity of ulcerative colitis. It classifies the mucosal inflammation in 4 grades (0=normal, 1=granular mucosa with an abnormal vascular pattern, 2=friable mucosa, 3=microulceration with spontaneous bleeding, 4=gross ulceration with spontaneous bleeding).
    End point type
    Primary
    End point timeframe
    Baseline and Week 8
    End point values
    Pooled MultiStem Pooled Placebo
    Number of subjects analysed
    48
    40
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline
    3.13 ± 1.104
    3.1 ± 1.128
        Change at Week 8
    0.1 ± 1.134
    -0.3 ± 1.091
    Statistical analysis title
    Change From Baseline in Endoscopic Score (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Pooled MultiStem v Pooled Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.96 [1]
    Method
    ANCOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    0.4
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.12
         upper limit
    0.69
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.223
    Notes
    [1] - 1-sided p-value

    Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

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    End point title
    Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4
    End point description
    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.
    End point type
    Primary
    End point timeframe
    Baseline and Week 4
    End point values
    Pooled MultiStem Pooled Placebo
    Number of subjects analysed
    48
    40
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline
    1.42 ± 0.942
    1.23 ± 0.832
        Change at Week 4
    -0.44 ± 0.943
    -0.38 ± 0.705
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Pooled MultiStem v Pooled Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.54 [2]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.02
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.19
         upper limit
    0.22
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.161
    Notes
    [2] - 1-sided p-value

    Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

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    End point title
    Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8
    End point description
    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.
    End point type
    Primary
    End point timeframe
    Baseline and Week 8
    End point values
    Pooled MultiStem Pooled Placebo
    Number of subjects analysed
    48
    40
    Units: scores on a scale
        arithmetic mean (standard deviation)
    -0.46 ± 1.051
    -0.43 ± 0.874
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Pooled MultiStem v Pooled Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.67 [3]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.08
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.15
         upper limit
    0.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.174
    Notes
    [3] - 1-sided p-value

    Primary: Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [4]
    End point description
    An AE was any untoward medical occurrence in a subject who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    Notes
    [4] - 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 was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: subjects
        AEs
    2
    3
    0
    4
    5
    20
    18
    16
    19
        SAEs
    1
    0
    0
    3
    0
    7
    4
    5
    4
    No statistical analyses for this end point

    Primary: Incidence of Treatment-Emergent AEs by System Organ Class (SOC)

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    End point title
    Incidence of Treatment-Emergent AEs by System Organ Class (SOC) [5]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    Notes
    [5] - 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 was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: subjects
        BLOOD AND LYMPHATIC SYSTEM DISORDERS
    0
    0
    0
    0
    0
    5
    0
    3
    4
        CARDIAC DISORDERS
    0
    0
    0
    0
    0
    1
    0
    1
    1
        EAR AND LABYRINTH DISORDERS
    0
    0
    0
    0
    0
    1
    0
    1
    1
        ENDOCRINE DISORDERS
    0
    0
    0
    0
    0
    0
    1
    1
    0
        EYE DISORDERS
    0
    0
    0
    0
    0
    1
    0
    0
    3
        GASTROINTESTINAL DISORDERS
    2
    1
    0
    2
    4
    13
    10
    11
    12
        GENERAL DISORDERS
    2
    1
    0
    3
    1
    8
    5
    5
    6
        IMMUNE SYSTEM DISORDERS
    0
    0
    0
    1
    0
    0
    2
    0
    0
        INFECTIONS AND INFESTATIONS
    1
    1
    0
    1
    1
    10
    9
    9
    7
        INJURY, POISONING AND PROCEDURAL COMPLICATIONS
    0
    1
    0
    1
    0
    2
    2
    2
    1
        INVESTIGATIONS
    0
    1
    0
    0
    1
    4
    1
    5
    3
        METABOLISM AND NUTRITION DISORDERS
    0
    0
    0
    1
    0
    2
    0
    1
    1
        MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS
    0
    2
    0
    0
    2
    6
    5
    2
    1
        NEOPLASMS BENIGN, MALIGNANT AND UNSPECIFIED
    0
    0
    0
    0
    0
    1
    0
    0
    0
        NERVOUS SYSTEM DISORDERS
    0
    1
    0
    1
    1
    1
    3
    9
    6
        PSYCHIATRIC DISORDERS
    0
    0
    0
    0
    0
    1
    1
    3
    2
        RENAL AND URINARY DISORDERS
    0
    0
    0
    0
    0
    1
    1
    0
    0
        RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS
    1
    1
    0
    0
    0
    5
    5
    1
    4
        SKIN AND SUBCUTANEOUS TISSUE DISORDERS
    0
    0
    0
    1
    1
    4
    4
    3
    2
        SURGICAL AND MEDICAL PROCEDURES
    0
    0
    0
    0
    0
    1
    0
    1
    0
        VASCULAR DISORDERS
    0
    0
    0
    0
    2
    1
    1
    2
    1
    No statistical analyses for this end point

    Primary: Number of Treatment-Emergent AEs by Severity

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    End point title
    Number of Treatment-Emergent AEs by Severity [6]
    End point description
    The intensity grades were defined as follows: mild=does not interfere with subject's usual function; moderate=interferes to some extent with subject's usual function; severe=interferes significantly with subject's usual function.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    Notes
    [6] - 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 was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: adverse events
        Mild AEs
    6
    16
    0
    8
    13
    61
    35
    56
    48
        Moderate AEs
    4
    0
    0
    5
    2
    33
    31
    21
    17
        Severe AEs
    1
    0
    0
    4
    0
    13
    8
    4
    3
    No statistical analyses for this end point

    Secondary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

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    End point title
    Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16
    End point description
    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12, Week 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Change at Week 12 (n=0,0,0,0,0,22,25,17,19)
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    -0.77 ± 0.869
    -0.64 ± 1.114
    -0.53 ± 0.874
    -0.47 ± 0.905
        Change at Week 16 (n=2,3,2,4,5,21,24,17,19)
    -1.5 ± 0.707
    -1 ± 1
    0 ± 0
    0 ± 0.816
    -0.6 ± 1.14
    -0.57 ± 0.978
    -0.92 ± 1.1
    -0.47 ± 0.874
    -0.58 ± 0.838
    No statistical analyses for this end point

    Secondary: Number of Subjects With Laboratory Test Abnormalities

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    End point title
    Number of Subjects With Laboratory Test Abnormalities
    End point description
    The total number of subjects with laboratory test abnormalities with or without regard to baseline abnormality was assessed. Laboratory parameters included: hematology (hemoglobin, hematocrit, red blood cell [RBC] count, platelet count, white blood cell [WBC] count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes, erythrocyte sedimentation rate); chemistry (blood urea nitrogen and creatinine, glucose, calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase, alanine aminotransferase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein; urinalysis (pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, microscopy (only if urine dipstick was positive for blood, protein, nitrites or leukocyte esterase); other (follicle-stimulating hormone, human chorionic gonadotropin, stool microbiology, creatinine kinase, direct bilirubin, indirect bilirubin, gamma-glutamyl transferase, international normalized ratio.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 24
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: subjects
        Normal/Abnormal Baseline(n=2,3,2,4,6,23,25,19,21)
    2
    2
    1
    4
    4
    14
    20
    15
    17
        Normal Baseline (n=2,3,2,4,6,23,25,19,21)
    2
    2
    1
    3
    4
    9
    16
    12
    13
        Abnormal Baseline (n=2,3,2,2,3,22,19,15,18)
    1
    1
    1
    1
    2
    7
    4
    4
    6
    No statistical analyses for this end point

    Secondary: Number of Subjects With Potentially Clinically Significant Vital Signs Findings

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    End point title
    Number of Subjects With Potentially Clinically Significant Vital Signs Findings
    End point description
    Vital signs assessment included pulse rate, systolic blood pressure (SBP) and diastolic blood pressure (DBP). Criteria for vital sign values meeting potential clinical concern included: SBP <90 millimeters of mercury (mm Hg) and >=30 mm Hg increase/decrease from baseline, DBP <50 mm Hg and >=20 mm Hg increase/decrease from baseline, pulse rate <40 or >120 beats per minute (bpm),
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 52
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: subjects
        Supine SBP <90 mm Hg
    0
    0
    0
    0
    2
    0
    0
    0
    0
        Supine DBP <50 mm Hg
    0
    0
    0
    0
    0
    0
    1
    0
    0
        Supine Pulse Rate <40 bpm
    0
    0
    0
    0
    0
    0
    0
    0
    0
        Supine Pulse Rate >120 bpm
    0
    0
    0
    0
    0
    3
    0
    1
    0
        Supine SBP >=30 mm Hg Increase From Baseline
    0
    0
    0
    1
    1
    1
    3
    1
    2
        Supine DBP >=20 mm Hg Increase From Baseline
    2
    0
    0
    1
    0
    3
    0
    2
    4
        Supine SBP >=30 mm Hg Decrease From Baseline
    0
    0
    0
    1
    0
    1
    1
    1
    1
        Supine DBP >=20 mm Hg Decrease From Baseline
    1
    1
    0
    0
    0
    6
    1
    3
    0
    No statistical analyses for this end point

    Secondary: Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

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    End point title
    Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16
    End point description
    Fecal calprotectin, a very stable marker, is a 36kDa calcium and zinc binding protein which is neutrophil-derived. It represents 60% of cytosolic proteins in granulocytes and is a measurement of neutrophil migration to the gastrointestinal tract.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8, 12 and 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: milligrams per kilogram (mg/kg)
    geometric mean (geometric coefficient of variation)
        Baseline (n=2,3,1,4,5,21,23,19,20)
    690.7549 ± 17
    1189.8169 ± 22
    739.23 ± 99999
    898.2758 ± 89
    874.1363 ± 1462
    576.5796 ± 237
    476.1504 ± 222
    513.2139 ± 208
    821.1953 ± 220
        Change at Week 4 (n=2,3,1,3,5,20,21,18,19)
    0.6297 ± 50
    1.868 ± 103
    2.0264 ± 99999
    2.0549 ± 112
    1.094 ± 91
    1.2196 ± 158
    0.8179 ± 314
    1.0541 ± 166
    0.6722 ± 193
        Change at Week 8 (n=1,2,1,3,2,18,20,17,20)
    2.4504 ± 99999
    2.4559 ± 51
    2.6199 ± 99999
    0.5744 ± 52
    0.3282 ± 2
    1.0704 ± 220
    0.7462 ± 235
    0.8598 ± 227
    0.3479 ± 242
        Change at Week 12 (n=0,0,0,0,0,20,21,17,18)
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    0.9947 ± 171
    0.8269 ± 310
    0.8788 ± 293
    0.5279 ± 171
        Change at Week 16 (n=2,3,1,4,4,18,18,16,17)
    2.1672 ± 49
    0.7679 ± 32
    1.1956 ± 99999
    0.4459 ± 139
    0.2474 ± 4517
    0.6473 ± 277
    0.8486 ± 149
    0.5473 ± 309
    0.5275 ± 243
    Statistical analysis title
    Fecal Calprotectin (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.53 [7]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.02
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.73
         upper limit
    1.42
    Notes
    [7] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.91 [8]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.48
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.02
         upper limit
    2.14
    Notes
    [8] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 12)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.81 [9]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.38
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.86
         upper limit
    2.23
    Notes
    [9] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 12)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.44 [10]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    0.94
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.59
         upper limit
    1.51
    Notes
    [10] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 12)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.67 [11]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.18
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.72
         upper limit
    1.94
    Notes
    [11] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 16)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.62 [12]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.12
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.68
         upper limit
    1.84
    Notes
    [12] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 16)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.45 [13]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    0.95
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.58
         upper limit
    1.57
    Notes
    [13] - 1-sided p-value
    Statistical analysis title
    Fecal Calprotectin (Week 16)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.3 [14]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    0.81
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.49
         upper limit
    1.36
    Notes
    [14] - 1-sided p-value

    Secondary: Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

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    End point title
    Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16
    End point description
    CRP is an acute-phase protein which provides an objective criterion of inflammatory activity. CRP has a short half-life (19 hours) and therefore rises early after the onset of inflammation and rapidly decreases after resolution of the inflammation. It is induced by interleukin-6, TNF-alpha and other pro-inflammatory cytokines that are produced within the intestinal lamina propria.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8, 12 and 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: milligrams per deciliter (mg/dL)
    geometric mean (geometric coefficient of variation)
        Baseline (n=2,3,2,4,6,22,25,19,20)
    1.272 ± 45
    1.527 ± 118
    0.229 ± 34
    0.578 ± 37
    0.18 ± 74
    0.613 ± 339
    0.542 ± 236
    0.397 ± 157
    0.453 ± 297
        Change at Week 4 (n=2,3,2,3,6,21,25,18,20)
    0.289 ± 9
    0.555 ± 82
    1.937 ± 42
    1.285 ± 60
    1.446 ± 54
    0.891 ± 147
    0.764 ± 140
    0.999 ± 103
    0.588 ± 132
        Change at Week 8 (n=1,3,1,3,5,21,25,19,20)
    1.39 ± 99999
    0.745 ± 49
    2.331 ± 99999
    1.172 ± 20
    1.555 ± 31
    1.189 ± 146
    0.759 ± 207
    1.2 ± 113
    0.623 ± 150
        Change at Week 12 (n=0,0,0,0,0,21,24,17,18)
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    0.937 ± 211
    0.71 ± 365
    0.782 ± 149
    0.373 ± 155
        Change at Week 16 (n=2,3,2,4,5,20,24,17,18)
    0.956 ± 164
    0.481 ± 56
    0.606 ± 248
    0.869 ± 14
    1.184 ± 54
    0.776 ± 156
    0.425 ± 178
    0.612 ± 133
    0.417 ± 197
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.79 [15]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.17
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.91
         upper limit
    1.51
    Notes
    [15] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.8 [16]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.21
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.9
         upper limit
    1.63
    Notes
    [16] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 12)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.99 [17]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    2.75
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.63
         upper limit
    4.64
    Notes
    [17] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 12)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.96 [18]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.96
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.19
         upper limit
    3.25
    Notes
    [18] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 12)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.95 [19]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.98
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.15
         upper limit
    3.41
    Notes
    [19] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 16)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.99 [20]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    2.3
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.52
         upper limit
    3.48
    Notes
    [20] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 16)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.62 [21]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.1
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.74
         upper limit
    1.63
    Notes
    [21] - 1-sided p-value
    Statistical analysis title
    Fold Change From Baseline in CRP (Week 16)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.89 [22]
    Method
    Mixed models analysis
    Parameter type
    Geometric Mean Ratio
    Point estimate
    1.51
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.98
         upper limit
    2.32
    Notes
    [22] - 1-sided p-value

    Secondary: Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

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    End point title
    Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16
    End point description
    Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes).
    End point type
    Secondary
    End point timeframe
    Week 4, 8, 12 and 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
    number (not applicable)
        Week 4
    50
    66.7
    50
    33.3
    40
    52.2
    28
    31.6
    42.9
        Week 8
    50
    66.7
    50
    33.3
    40
    47.8
    36
    31.6
    47.6
        Week 12
    99999
    99999
    99999
    99999
    99999
    63.6
    40
    52.9
    47.4
        Week 16
    50
    100
    50
    50
    40
    52.4
    50
    41.2
    57.9
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.34 [23]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.21
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.66
         upper limit
    2.19
    Notes
    [23] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.33 [24]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.23
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.68
         upper limit
    2.23
    Notes
    [24] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 12)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.1 [25]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    2.37
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.99
         upper limit
    5.67
    Notes
    [25] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 12)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.74 [26]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.66
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.28
         upper limit
    1.55
    Notes
    [26] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 12)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.48 [27]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.04
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.41
         upper limit
    2.64
    Notes
    [27] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 16)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.58 [28]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.88
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.38
         upper limit
    2.03
    Notes
    [28] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 16)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.68 [29]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.74
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.33
         upper limit
    1.66
    Notes
    [29] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore of Zero (Week 16)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.84 [30]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.51
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.21
         upper limit
    1.23
    Notes
    [30] - 1-sided p-value

    Secondary: Percentage of Subjects in Endoscopic Remission at Week 8

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    End point title
    Percentage of Subjects in Endoscopic Remission at Week 8
    End point description
    Endoscopic remission is defined as modified Baron Endoscopic Score equal to 0.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
        number (not applicable)
    0
    0
    0
    0
    16.7
    4.3
    4
    0
    9.5
    Statistical analysis title
    Endoscopic Remission (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.57 [31]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.83
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.22
         upper limit
    3.07
    Notes
    [31] - 1-sided p-value

    Secondary: Percentage of Subjects in Clinical Remission at Week 8

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    End point title
    Percentage of Subjects in Clinical Remission at Week 8
    End point description
    Clinical remission is defined as a total Mayo score of 2 points or lower, with no individual subscores exceeding 1 point.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
        number (not applicable)
    0
    0
    0
    0
    0
    0
    8
    0
    19
    Statistical analysis title
    Clinical Remission (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.85 [32]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.39
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.12
         upper limit
    1.23
    Notes
    [32] - 1-sided p-value

    Secondary: Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

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    End point title
    Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16
    End point description
    Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes). A decrease from baseline score indicates improvement.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8, 12 and 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
    number (not applicable)
        Week 4
    100
    66.7
    0
    0
    40
    43.5
    48
    21.1
    38.1
        Week 8
    100
    33.3
    0
    0
    40
    34.8
    48
    31.6
    47.6
        Week 12
    99999
    99999
    99999
    99999
    99999
    54.5
    60
    41.2
    52.6
        Week 16
    100
    66.7
    0
    25
    60
    47.6
    58.3
    35.3
    47.4
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.05 [33]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    2.27
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    1.21
         upper limit
    4.24
    Notes
    [33] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.43 [34]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.09
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.61
         upper limit
    1.95
    Notes
    [34] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 12)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.44 [35]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.11
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.45
         upper limit
    2.76
    Notes
    [35] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 12)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.38 [36]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.23
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.5
         upper limit
    3.04
    Notes
    [36] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 12)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.89 [37]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.38
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.14
         upper limit
    1.04
    Notes
    [37] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 16)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.47 [38]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.05
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.45
         upper limit
    2.42
    Notes
    [38] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 16)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.24 [39]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    1.58
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.7
         upper limit
    3.57
    Notes
    [39] - 1-sided p-value
    Statistical analysis title
    Rectal Bleeding Mayo Subscore (Week 16)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.81 [40]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.53
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.22
         upper limit
    1.32
    Notes
    [40] - 1-sided p-value

    Secondary: Percentage of Subjects With Endoscopic Response at Week 8

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    End point title
    Percentage of Subjects With Endoscopic Response at Week 8
    End point description
    Endoscopic response is defined as a decrease in modified Baron endoscopic score from baseline of at least 2 points.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
        number (not applicable)
    0
    0
    0
    0
    0
    8.7
    8
    10.5
    19
    Statistical analysis title
    Endoscopic Response (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.84 [41]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.49
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.2
         upper limit
    1.24
    Notes
    [41] - 1-sided p-value

    Secondary: Percentage of Subjects in Clinical Response at Week 8

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    End point title
    Percentage of Subjects in Clinical Response at Week 8
    End point description
    Clinical response is defined as a decrease in total Mayo score from baseline of at least 3 points and at least 30 percent (%), with an accompanying decrease in the subscore for rectal bleeding of at least 1 point or an absolute subscore for rectal bleeding of 0 or 1.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: percentage of subjects
        number (not applicable)
    50
    33.3
    0
    0
    0
    4.3
    24
    15.8
    42.9
    Statistical analysis title
    Clinical Response (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.96 [42]
    Method
    Regression, Logistic
    Parameter type
    Odds ratio (OR)
    Point estimate
    0.3
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.12
         upper limit
    0.73
    Notes
    [42] - 1-sided p-value

    Secondary: Change From Baseline in Total Mayo Scores at Week 8

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    End point title
    Change From Baseline in Total Mayo Scores at Week 8
    End point description
    Mayo Score is an instrument designed to measure disease activity of ulcerative colitis, with total score ranging from 0 to 12. It consists of 4 subscores (stool frequency, rectal bleeding, findings of flexible proctosigmoidoscopy, and physician global assessment [PGA]), each graded from 0 to 3, with higher scores indicating more severe disease.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline (n=2,3,2,4,6,23,25,19,21)
    10 ± 2.828
    7.33 ± 1.155
    8.5 ± 0.707
    7.75 ± 0.957
    7.5 ± 2.258
    8.74 ± 1.839
    8.48 ± 2.143
    8.47 ± 1.806
    8.14 ± 2.2
        Change at Week 8 (n=2,3,2,3,5,23,25,19,21)
    -2 ± 1.414
    0 ± 2.646
    1 ± 1.414
    0.33 ± 0.577
    -0.6 ± 0.894
    -0.78 ± 2.066
    -1.2 ± 2.872
    -0.89 ± 2.132
    -2 ± 2.683
    Statistical analysis title
    Total Mayo Scores (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.87 [43]
    Method
    ANCOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    0.58
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.08
         upper limit
    1.24
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.513
    Notes
    [43] - 1-sided p-value

    Secondary: Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

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    End point title
    Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16
    End point description
    A Partial Mayo Score (Mayo score without endoscopy) ranges from 0 (normal or inactive disease) to 9 (severe disease) and calculated as the sum of 3 subscores (stool frequency, rectal bleeding and PGA) with each ranging from 0 to 3 (0=normal, 1=mild, 2=moderate, 3=severe). Higher scores indicate more severe disease.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 4, 8, 12 and 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline (n=2,3,2,4,6,23,25,19,21)
    7.5 ± 2.121
    5.33 ± 0.577
    6 ± 1.414
    5.75 ± 0.5
    5.5 ± 1.378
    6.22 ± 1.347
    6.08 ± 1.605
    6 ± 1.202
    5.71 ± 1.736
        Change at Week 4 (n=2,3,2,3,5,23,25,19,21)
    -3 ± 0
    -2 ± 1.732
    -0.5 ± 0.707
    0.67 ± 1.528
    -0.4 ± 1.14
    -1 ± 1.414
    -0.96 ± 1.791
    -1.16 ± 1.675
    -1.24 ± 1.895
        Change at Week 8 (n=2,3,2,3,5,23,25,19,21)
    -2 ± 1.414
    -1 ± 1.732
    0.5 ± 0.707
    0.33 ± 0.577
    -0.8 ± 1.095
    -0.91 ± 1.756
    -1.12 ± 2.472
    -0.79 ± 1.653
    -1.62 ± 2.037
        Change at Week 12 (n=0,0,0,0,0,22,25,17,19)
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    99999 ± 99999
    -1.91 ± 1.974
    -1.52 ± 2.312
    -2.06 ± 2.135
    -2.11 ± 2.622
        Change at Week 16 (2,3,2,4,5,21,24,17,19)
    -1.5 ± 0.707
    -3 ± 3
    -1 ± 2.828
    -1.25 ± 3.403
    -2.2 ± 1.924
    -1.86 ± 2.061
    -2.54 ± 2.621
    -2.41 ± 2.181
    -2.63 ± 2.033
    Statistical analysis title
    Partial Mayo Scores (Week 4)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.8 [44]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.29
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.15
         upper limit
    0.74
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.345
    Notes
    [44] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.77 [45]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.3
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.23
         upper limit
    0.84
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.418
    Notes
    [45] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 12)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.74 [46]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.44
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.42
         upper limit
    1.3
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.666
    Notes
    [46] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 12)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.88 [47]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.76
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.08
         upper limit
    1.59
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.645
    Notes
    [47] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 12)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.72 [48]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.42
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.5
         upper limit
    1.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.705
    Notes
    [48] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 16)
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.97 [49]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    1.21
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    0.37
         upper limit
    2.06
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.656
    Notes
    [49] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 16)
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.75 [50]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.43
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.39
         upper limit
    1.25
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.635
    Notes
    [50] - 1-sided p-value
    Statistical analysis title
    Partial Mayo Scores (Week 16)
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.85 [51]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.73
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.16
         upper limit
    1.63
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.692
    Notes
    [51] - 1-sided p-value

    Secondary: Change From Baseline in Biopsy Histology Scores at Week 8

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    End point title
    Change From Baseline in Biopsy Histology Scores at Week 8
    End point description
    A 15 to 25 centimeter (cm) biopsy sample of inflamed mucosal tissue was taken from the worst affected area and scored using the Riley Index. The Riley Index is a histologic scoring system for the assessment of the activity and severity of ulcerative colitis; it consists of 6 histologic features (acute inflammatory cell infiltrate, crypt abscesses, mucin depletion, surface epithelial integrity, chronic inflammatory cell infiltrate, and crypt architectural irregularities), all scored on a 4-point scale (higher scores indicate more severe disease).
    End point type
    Secondary
    End point timeframe
    Baseline and Week 8
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline(n=2,3,2,4,5,22,24,18,21)
    9 ± 1.41
    9.7 ± 3.79
    11 ± 2.83
    7 ± 3.92
    10.8 ± 3.77
    9.4 ± 4.37
    10.4 ± 4.24
    9.9 ± 4.17
    9.7 ± 5.14
        Change at Week 4 (n=2,3,1,3,5,21,24,15,20)
    -3.5 ± 4.95
    1.3 ± 1.15
    -3 ± 99999
    -1 ± 1.73
    -0.4 ± 2.07
    1.4 ± 4.63
    -1 ± 3.76
    0.9 ± 4.09
    -1.5 ± 4.8
    Statistical analysis title
    Biopsy Histology Scores (Week 8)
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.75 [52]
    Method
    ANCOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    0.52
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.46
         upper limit
    1.49
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.753
    Notes
    [52] - 1-sided p-value

    Secondary: Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

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    End point title
    Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16
    End point description
    Patient-reported diary data assessed the number of bowel movements (BM) per day when not having a flare and the presence of blood in the stools (rectal bleeding [RB]), if any.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 16
    End point values
    Cohort 1: Placebo, MultiStem 300 Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300, Placebo Cohort 1: MultiStem 300 (x3), Placebo Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Number of subjects analysed
    2
    3
    2
    4
    6
    23
    25
    19
    21
    Units: scores on a scale
    arithmetic mean (standard deviation)
        BM: Baseline(n=2,3,2,4,6,23,25,19,21)
    12.5 ± 4.95
    8 ± 2.784
    6.75 ± 2.475
    11.75 ± 10.506
    8.42 ± 3.089
    7.5 ± 3.49
    7.78 ± 3.781
    7.5 ± 3.571
    6.88 ± 4.886
        BM: Change at Week 1 (n=2,3,2,4,5,23,25,19,21)
    -1.6 ± 2.263
    -1.89 ± 1.786
    4.15 ± 2.845
    -0.6 ± 2.177
    0.4 ± 1.3
    -0.42 ± 1.509
    -0.59 ± 1.657
    -0.56 ± 1.215
    -0.81 ± 3.421
        BM: Change at Week 4 (n=2,2,2,3,5,23,23,16,19
    -1.43 ± 3.435
    -1.89 ± 5.101
    2.36 ± 0.505
    0.17 ± 2.038
    1.31 ± 1.432
    -0.81 ± 2.088
    0.28 ± 3.956
    -0.63 ± 1.856
    -1.08 ± 2.575
        BM: Change at Week 8 (n=2,3,2,3,6,23,23,16,19)
    -1.67 ± 3.771
    -0.86 ± 3.517
    3.18 ± 0.758
    0.17 ± 2.754
    0.41 ± 2.131
    -0.89 ± 2.864
    -1.15 ± 2.833
    0.18 ± 2.531
    -1.41 ± 3.208
        BM: Change at Week 12 (n=1,1,1,2,2,21,25,16,18)
    0.14 ± 99999
    1.43 ± 99999
    1.57 ± 99999
    -1.68 ± 2.374
    0 ± 0
    -0.67 ± 3.152
    -0.59 ± 4.428
    -1.77 ± 2.301
    -2.02 ± 3.502
        BM: Change at Week 16 (n=1,3,2,3,4,22,24,16,18)
    0 ± 99999
    -2.51 ± 3.716
    1.72 ± 0.596
    -0.12 ± 2.324
    -2.45 ± 2.208
    -1.81 ± 3.278
    -2.17 ± 3.523
    -1.68 ± 2.899
    -1.86 ± 3.616
        RB: Baseline (n=2,3,2,4,6,23,25,19,21)
    1.25 ± 1.061
    1.17 ± 1.258
    1 ± 1.414
    1 ± 0.816
    1.33 ± 0.816
    1.2 ± 0.974
    1.46 ± 0.978
    0.97 ± 0.95
    1.05 ± 0.82
        RB: Change at Week 1 (n=2,3,2,4,5,23,25,19,21)
    -0.32 ± 0.253
    -1 ± 1.167
    -0.33 ± 0.471
    -0.12 ± 0.158
    -0.3 ± 0.415
    -0.36 ± 0.697
    -0.24 ± 0.805
    -0.05 ± 0.574
    -0.1 ± 0.408
        RB: Change at Week 4 (n=2,2,2,3,5,23,23,16,19)
    -0.75 ± 0.354
    -0.29 ± 1.01
    -0.25 ± 0.354
    0.14 ± 0.378
    -0.71 ± 0.99
    -0.34 ± 0.863
    -0.47 ± 0.84
    -0.27 ± 0.738
    -0.26 ± 0.604
        RB: Change at Week 8 (n=2,3,2,3,6,22,23,13,20)
    -0.75 ± 0.354
    -0.5 ± 0.5
    0 ± 0
    0.33 ± 0.577
    -0.47 ± 1.035
    -0.4 ± 0.709
    -0.51 ± 0.805
    -0.19 ± 0.997
    -0.51 ± 0.967
        RB: Change at Week 12 (n=1,1,1,2,2,21,25,16,18)
    -0.57 ± 99999
    0 ± 99999
    0 ± 99999
    0 ± 0
    0 ± 0
    -0.6 ± 0.792
    -0.49 ± 1.084
    -0.48 ± 0.829
    -0.39 ± 0.783
        RB: Change at Week 16 (n=1,3,2,3,4,22,24,16,18)
    -1 ± 99999
    -1.17 ± 1.258
    -0.1 ± 0.141
    0.33 ± 0.577
    -1.14 ± 0.865
    -0.41 ± 0.936
    -0.86 ± 1.106
    -0.41 ± 0.838
    -0.39 ± 0.874
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 4)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.51 [53]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.18
         upper limit
    0.19
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.144
    Notes
    [53] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Pooled MultiStem versus Pooled Placebo (Week 8)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    88
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.62 [54]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.05
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.16
         upper limit
    0.25
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.158
    Notes
    [54] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.29 [55]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.12
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.39
         upper limit
    0.16
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.213
    Notes
    [55] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.74 [56]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.13
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.14
         upper limit
    0.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.209
    Notes
    [56] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 12)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.36 [57]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.08
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.37
         upper limit
    0.21
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.226
    Notes
    [57] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 MM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.56 [58]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    0.04
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.26
         upper limit
    0.34
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.234
    Notes
    [58] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 MP versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.18 [59]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.21
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.51
         upper limit
    0.09
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.231
    Notes
    [59] - 1-sided p-value
    Statistical analysis title
    Patient-Reported Rectal Bleeding up to Week 16
    Statistical analysis description
    Cohort 3 PM versus Cohort 3 PP (Week 16)
    Comparison groups
    Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.31 [60]
    Method
    Mixed models analysis
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.12
    Confidence interval
         level
    80%
         sides
    2-sided
         lower limit
    -0.45
         upper limit
    0.2
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.25
    Notes
    [60] - 1-sided p-value

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to 28 days after last study drug administration.
    Adverse event reporting additional description
    The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 subject and as non-serious in another, or 1 subject may have experienced both an AE and SAE during the study. All treated subjects were included in the analysis.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Cohort 1: Placebo, MultiStem 300
    Reporting group description
    Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 2: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 1: MultiStem 300 (x3), Placebo
    Reporting group description
    Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, MultiStem 750
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 2: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: MultiStem 750, Placebo
    Reporting group description
    Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, MultiStem 750
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

    Reporting group title
    Cohort 3: Placebo, Placebo
    Reporting group description
    Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

    Serious adverse events
    Cohort 1: Placebo, MultiStem 300 Cohort 1: MultiStem 300, Placebo Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300 (x3), Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    3 / 4 (75.00%)
    7 / 23 (30.43%)
    0 / 6 (0.00%)
    4 / 25 (16.00%)
    5 / 19 (26.32%)
    4 / 21 (19.05%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    0
    0
    Vascular disorders
    Diastolic hypertension
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Adenocarcinoma of colon
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Ventricular tachycardia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancytopenia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Epilepsy
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Superior sagittal sinus thrombosis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Colitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Colitis ulcerative
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    5 / 23 (21.74%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    2 / 19 (10.53%)
    3 / 21 (14.29%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
    0 / 7
    0 / 0
    0 / 1
    0 / 2
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Abdominal abscess
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Clostridium difficile colitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Clostridium difficile infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pelvic sepsis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Perirectal abscess
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Cohort 1: Placebo, MultiStem 300 Cohort 1: MultiStem 300, Placebo Cohort 2: Placebo, MultiStem 750 Cohort 1: MultiStem 300 (x3), Placebo Cohort 3: MultiStem 750, MultiStem 750 Cohort 2: MultiStem 750, Placebo Cohort 3: MultiStem 750, Placebo Cohort 3: Placebo, MultiStem 750 Cohort 3: Placebo, Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    0 / 2 (0.00%)
    3 / 3 (100.00%)
    3 / 4 (75.00%)
    18 / 23 (78.26%)
    5 / 6 (83.33%)
    16 / 25 (64.00%)
    15 / 19 (78.95%)
    18 / 21 (85.71%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    2
    1
    Hot flush
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Hypertension
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Surgical and medical procedures
    Ileostomy
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    4
    0
    0
    0
    0
    Chest discomfort
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    2 / 19 (10.53%)
    1 / 21 (4.76%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    0
    2
    1
    Chest pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    1
    0
    2
    Chills
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    2 / 4 (50.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    3
    1
    0
    0
    0
    0
    Fatigue
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    3 / 25 (12.00%)
    1 / 19 (5.26%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    0
    3
    0
    3
    1
    2
    Influenza like illness
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    3
    0
    0
    Oedema peripheral
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    3
    Pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    1
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    3 / 4 (75.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    1 / 19 (5.26%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    7
    2
    0
    2
    1
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    3 / 19 (15.79%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    2
    3
    0
    Confusional state
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Depression
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    0
    2
    Insomnia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    1
    0
    Fall
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    1
    0
    0
    0
    0
    Incision site pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    0
    Laceration
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    0
    Skeletal injury
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    0
    Investigations
    Antibody test
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Bacterial test
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Blood albumin decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Blood bilirubin increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Blood glucose increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Blood urine present
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    0
    C-reactive protein increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    5
    0
    0
    2
    0
    Cold agglutinins
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Heart rate increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Prostatic specific antigen increased
         subjects affected / exposed [1]
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 16 (0.00%)
    0 / 1 (0.00%)
    0 / 13 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    0
    1
    Weight decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    0
    2
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    3 / 25 (12.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    1
    0
    3
    0
    3
    0
    0
    Dyspnoea
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    2
    Nasal congestion
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    1
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    3 / 25 (12.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    4
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    4 / 23 (17.39%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    2 / 19 (10.53%)
    3 / 21 (14.29%)
         occurrences all number
    0
    0
    0
    0
    6
    0
    0
    2
    4
    Lymphadenopathy
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Dizziness
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    1 / 25 (4.00%)
    2 / 19 (10.53%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    1
    2
    0
    Epilepsy
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    2
    0
    Headache
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    3 / 25 (12.00%)
    7 / 19 (36.84%)
    6 / 21 (28.57%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    3
    7
    7
    Lethargy
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    2 / 19 (10.53%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    3
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    1
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    1 / 19 (5.26%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    0
    4
    0
    3
    2
    2
    Abdominal pain upper
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    0
    Abdominal tenderness
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    0
    0
    0
    Colitis ulcerative
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    5 / 23 (21.74%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    3 / 19 (15.79%)
    5 / 21 (23.81%)
         occurrences all number
    0
    0
    0
    0
    8
    0
    3
    4
    5
    Defaecation urgency
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    3
    0
    0
    0
    0
    Dyspepsia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    1
    Gastritis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    0
    0
    Haematochezia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    2 / 6 (33.33%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    2 / 21 (9.52%)
         occurrences all number
    0
    0
    0
    0
    4
    2
    3
    1
    2
    Painful defaecation
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Perianal erythema
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    0
    Rectal haemorrhage
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Tongue disorder
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    2 / 19 (10.53%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    3
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    0
    Erythema
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Night sweats
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Pruritus
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    2
    0
    Rash
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    2
    1
    1
    Rash macular
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    0
    0
    Skin lesion
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    2
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    2
    0
    3
    1
    0
    Back pain
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    0
    0
    4
    0
    0
    0
    1
    Costochondritis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    0
    0
    Muscle spasms
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    1
    0
    0
    Muscle tightness
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    3
    0
    1
    0
    0
    Osteoarthritis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    1
    1
    0
    Pain in jaw
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    1 / 6 (16.67%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    0
    0
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    0
    0
    0
    Hyperglycaemia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Hypokalaemia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    0
    0
    0
    Infections and infestations
    Bacteriuria
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Bronchitis
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    0
    0
    Cellulitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    0 / 23 (0.00%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    1
    0
    Influenza
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    0
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    1 / 25 (4.00%)
    5 / 19 (26.32%)
    4 / 21 (19.05%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    1
    7
    4
    Pharyngitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
    1 / 23 (4.35%)
    0 / 6 (0.00%)
    0 / 25 (0.00%)
    1 / 19 (5.26%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
    3 / 23 (13.04%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    1
    0
    3
    0
    3
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 4 (25.00%)
    2 / 23 (8.70%)
    1 / 6 (16.67%)
    2 / 25 (8.00%)
    1 / 19 (5.26%)
    1 / 21 (4.76%)
         occurrences all number
    0
    0
    2
    1
    3
    1
    3
    1
    1
    Urinary tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
    2 / 23 (8.70%)
    0 / 6 (0.00%)
    2 / 25 (8.00%)
    0 / 19 (0.00%)
    0 / 21 (0.00%)
         occurrences all number
    0
    0
    0
    1
    2
    0
    2
    0
    0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This AE is gender specific event. The percentages of gender specific events are calculated using the corresponding gender count as denominator.

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Oct 2012
    Interim analysis for futility was added.

    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
    The status of studies in GB is no longer updated from 1.1.2021
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