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    Clinical Trial Results:
    A Phase 2, Open-label, Randomized Study to Evaluate Safety and Efficacy of Momelotinib in Subjects with Polycythemia Vera or Essential Thrombocythemia

    Summary
    EudraCT number
    		 2013-004105-11
    Trial protocol
    		 DE  
    Global end of trial date
    07 May 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 May 2016
    First version publication date
    22 May 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    GS-US-354-0101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01998828
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Gilead Sciences
    Sponsor organisation address
    333 Lakeside Drive, Foster City, CA, United States, 94404
    Public contact
    Clinical Trial Mailbox, Gilead Sciences International Ltd, ClinicalTrialDisclosures@gilead.com
    Scientific contact
    Clinical Trial Mailbox, Gilead Sciences International Ltd, ClinicalTrialDisclosures@gilead.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
    07 May 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Mar 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    07 May 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This open-label study was to determine the safety and efficacy of momelotinib in participants with either polycythemia vera (PV) or essential thrombocythemia (ET) who had not yet received treatment with a Janus kinase (JAK) inhibitor.
    Protection of trial subjects
    The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements. This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    19 Feb 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 4
    Country: Number of subjects enrolled
    Germany: 5
    Country: Number of subjects enrolled
    United States: 21
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    Canada: 3
    Worldwide total number of subjects
    39
    EEA total number of subjects
    9
    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)
    27
    From 65 to 84 years
    12
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were enrolled at study sites in Europe, North America, and Australia. The first participant was screened on 19 February 2014. The last study visit occurred on 07 May 2015.

    Pre-assignment
    Screening details
    		 48 participants were screened.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 MMB 100 mg (PV Cohort)
    Arm description
    		 Participants with polycythemia vera received MMB 100 mg for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Momelotinib
    Investigational medicinal product code
    Other name
    GS-0387
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Momelotinib (MMB) 100 mg, 150 mg, or 200 mg once daily

    Arm title
    		 MMB 200 mg (PV Cohort)
    Arm description
    		 Participants with polycythemia vera received MMB 200 mg for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Momelotinib
    Investigational medicinal product code
    Other name
    GS-0387
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Momelotinib (MMB) 100 mg, 150 mg, or 200 mg once daily

    Arm title
    		 MMB 100 mg (ET Cohort)
    Arm description
    		 Participants with essential thrombocythemia received MMB 100 mg for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Momelotinib
    Investigational medicinal product code
    Other name
    GS-0387
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Momelotinib (MMB) 100 mg, 150 mg, or 200 mg once daily

    Arm title
    		 MMB 200 mg (ET Cohort)
    Arm description
    		 Participants with essential thrombocythemia received MMB 200 mg for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Momelotinib
    Investigational medicinal product code
    Other name
    GS-0387
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Momelotinib (MMB) 100 mg, 150 mg, or 200 mg once daily

    Number of subjects in period 1
    		MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Started
    14
    14
    5
    6
    Completed
    5
    5
    3
    2
    Not completed
    9
    9
    2
    4
         Subject Withdrew Consent
    2
    1
    -
    1
         Adverse event, non-fatal
    3
    4
    -
    1
         Study Discontinued by Sponsor
    2
    3
    1
    1
         Investigator's Discretion
    -
    1
    1
    1
         Lost to follow-up
    1
    -
    -
    -
         Disease Progression
    1
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    MMB 100 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 200 mg for 24 weeks.

    Reporting group title
    MMB 100 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 200 mg for 24 weeks.

    Reporting group values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort) Total
    Number of subjects
    		 14 14 5 6 39
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 58.7 ± 16.8 61.3 ± 6.54 60.8 ± 15.78 52 ± 11.93 -
    Gender categorical
    Units: Subjects
        Female
    		 7 4 3 2 16
        Male
    		 7 10 2 4 23

    End points

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    End points reporting groups
    Reporting group title
    MMB 100 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 200 mg for 24 weeks.

    Reporting group title
    MMB 100 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 200 mg for 24 weeks.

    Primary: Overall response rate

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    End point title
    		 Overall response rate [1]
    End point description
    Intent-to-Treat Analysis Set: all participants who are randomized regardless of whether they receive any MMB.
    End point type
    Primary
    End point timeframe
    Up to 24 weeks
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was planned or performed.
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Number of subjects analysed
    14
    14
    5
    6
    Units: percentage of participants
        number (confidence interval 90%)
    0 (0 to 19.3)
    14.3 (2.6 to 38.5)
    0 (0 to 45.1)
    0 (0 to 39.3)
    No statistical analyses for this end point

    Secondary: Confirmed overall response rate

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    End point title
    		 Confirmed overall response rate
    End point description
    Intent-to-Treat Analysis Set
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Number of subjects analysed
    14
    14
    5
    6
    Units: percentage of participants
        number (confidence interval 90%)
    0 (0 to 19.3)
    7.1 (0.4 to 29.7)
    0 (0 to 45.1)
    0 (0 to 39.3)
    No statistical analyses for this end point

    Secondary: Percentage of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks

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    End point title
    		 Percentage of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks [2]
    End point description
    Participants in the Intent-to-Treat Analysis Set from the PV Cohort were analyzed.
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Hematocrit response was only evaluated in the PV Cohort.
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort)
    Number of subjects analysed
    14
    14
    Units: percentage of participants
        number (confidence interval 90%)
    28.6 (10.4 to 54)
    21.4 (6.1 to 46.6)
    No statistical analyses for this end point

    Secondary: Percentage of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks

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    End point title
    		 Percentage of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
    End point description
    Intent-to-Treat Analysis Set
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Number of subjects analysed
    14
    14
    5
    6
    Units: percentage of participants
        number (confidence interval 90%)
    7.1 (0.4 to 29.7)
    35.7 (15.3 to 61)
    100 (54.9 to 100)
    83.3 (41.8 to 99.1)
    No statistical analyses for this end point

    Secondary: Percentage of participants with resolution of palpable splenomegaly that lasts at least 4 weeks

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    End point title
    		 Percentage of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
    End point description
    Participants in the Intent-to-Treat Analysis Set with baseline spleen size ≥ 5 cm were evaluated for spleen response and serve as the denominator for the spleen response rate calculation.
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Number of subjects analysed
    4
    3
    0 [3]
    0 [4]
    Units: percentage of participants
        number (confidence interval 90%)
    25 (1.3 to 75.1)
    33.3 (1.7 to 86.5)
    ( to )
    ( to )
    Notes
    [3] - No participants in this group had a baseline spleen size ≥ 5 cm.
    [4] - No participants in this group had a baseline spleen size ≥ 5 cm.
    No statistical analyses for this end point

    Secondary: Percentage of participants with ≥ 10 point decrease in modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) compared to baseline that lasts at least 12 weeks

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    End point title
    		 Percentage of participants with ≥ 10 point decrease in modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) compared to baseline that lasts at least 12 weeks
    End point description
    Intent-to-Treat Analysis Set
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Number of subjects analysed
    14
    14
    5
    6
    Units: percentage of participants
        number (not applicable)
    0
    7.1
    0
    16.7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline through end of study drug treatment (mean exposure: MMB 100 mg PV = 109.0 days; MMB 200 mg PV = 122.1 days; MMB 100 mg ET = 154.4 days; MMB 200 mg ET = 127.2 days) plus 30 days
    Adverse event reporting additional description
    Safety Analysis Set: all participants in the ITT Analysis Set who received ≥ 1 dose of MMB, with study treatment assignments designated according to the actual treatment received.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    MMB 100 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (PV Cohort)
    Reporting group description
    		 Participants with polycythemia vera received MMB 200 mg for 24 weeks.

    Reporting group title
    MMB 100 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 100 mg for 24 weeks.

    Reporting group title
    MMB 200 mg (ET Cohort)
    Reporting group description
    		 Participants with essential thrombocythemia received MMB 200 mg for 24 weeks.

    Serious adverse events
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         number of deaths (all causes)
    1
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Cardiac disorders
    Cardio-respiratory arrest
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urinary bladder polyp
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 MMB 100 mg (PV Cohort) MMB 200 mg (PV Cohort) MMB 100 mg (ET Cohort) MMB 200 mg (ET Cohort)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 14 (85.71%)
    14 / 14 (100.00%)
    5 / 5 (100.00%)
    5 / 6 (83.33%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Myelofibrosis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin cancer
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 14 (21.43%)
    4 / 14 (28.57%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    3
    4
    0
    2
    Flushing
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    1
    Haematoma
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 14 (14.29%)
    4 / 14 (28.57%)
    0 / 5 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    2
    4
    0
    2
    Asthenia
         subjects affected / exposed
    1 / 14 (7.14%)
    2 / 14 (14.29%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    2
    0
    0
    Pyrexia
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    0
    1
    0
    Chest pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oedema
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Oedema peripheral
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ulcer
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    2
    1
    0
    1
    Cough
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    3
    0
    0
    1
    Asthma
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    1
    Nasal congestion
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Haemoptysis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Productive cough
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Insomnia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Investigations
    Blood phosphorus increased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood uric acid increased
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Creatinine renal clearance decreased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Electrocardiogram QT prolonged
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Platelet count increased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Weight increased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Hand fracture
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Skin abrasion
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 14 (35.71%)
    4 / 14 (28.57%)
    1 / 5 (20.00%)
    3 / 6 (50.00%)
         occurrences all number
    5
    4
    2
    4
    Dizziness
         subjects affected / exposed
    0 / 14 (0.00%)
    6 / 14 (42.86%)
    0 / 5 (0.00%)
    3 / 6 (50.00%)
         occurrences all number
    0
    6
    0
    3
    Somnolence
         subjects affected / exposed
    4 / 14 (28.57%)
    2 / 14 (14.29%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    6
    2
    0
    0
    Peripheral sensory neuropathy
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    1
    1
    0
    Paraesthesia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    1
    Aura
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Burning sensation
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Disturbance in attention
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dysaesthesia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dysgeusia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Psychomotor hyperactivity
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Sciatica
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Syncope
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Tremor
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Thrombocytosis
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    1 / 5 (20.00%)
    3 / 6 (50.00%)
         occurrences all number
    1
    1
    1
    3
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Eye disorders
    Cataract subcapsular
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ocular discomfort
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Vision blurred
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    2 / 14 (14.29%)
    4 / 14 (28.57%)
    0 / 5 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    2
    4
    0
    2
    Diarrhoea
         subjects affected / exposed
    2 / 14 (14.29%)
    2 / 14 (14.29%)
    1 / 5 (20.00%)
    2 / 6 (33.33%)
         occurrences all number
    3
    2
    3
    2
    Vomiting
         subjects affected / exposed
    1 / 14 (7.14%)
    3 / 14 (21.43%)
    0 / 5 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    3
    0
    3
    Abdominal pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    3
    0
    2
    Constipation
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Flatulence
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    1
    Abdominal distension
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Dyspepsia
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gingival recession
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Salivary hypersecretion
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    2 / 14 (14.29%)
    3 / 14 (21.43%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    4
    0
    0
    Alopecia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    1
    Night sweats
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Rash
         subjects affected / exposed
    0 / 14 (0.00%)
    2 / 14 (14.29%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Cold sweat
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Erythrosis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin exfoliation
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 14 (7.14%)
    2 / 14 (14.29%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    2
    1
    0
    Bone pain
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    1
    0
    1
    Arthralgia
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    1
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Back pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Flank pain
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Myalgia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    2
    Urinary tract infection
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Bronchitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Cellulitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Cystitis
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Laryngitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pharyngitis
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Sinusitis
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Tooth abscess
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Tooth infection
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 5 (20.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    2
    Viral infection
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Vulvovaginal mycotic infection
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 5 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    0 / 5 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Jan 2014
    Incorporated newly available data and updates to study procedures.
    14 Jul 2014
    Updates to study procedures and restrictions based on newly available data.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    22 Sep 2014
    The study was discontinued due to limited efficacy observed in planned interim analyses.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    These data represent fewer than the planned number of subjects with limited treatment duration.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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