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    Clinical Trial Results:
    A Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Investigate the Efficacy and Safety of Ofatumumab Injection for Subcutaneous Use in Subjects with Pemphigus Vulgaris

    Summary
    EudraCT number
    		 2013-001370-20
    Trial protocol
    		 IT   HR   GR   PL  
    Global end of trial date
    11 Jan 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Jan 2019
    First version publication date
    06 Feb 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    COMB157J2301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01920477
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 OPV116910: GSK1841157
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 011 888-669-6682, Novartis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 011 888-669-6682, Novartis.email@novartis.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
    11 Jan 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Jan 2018
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective was to determine the efficacy, based on disease remission, of ofatumumab SC at a dose of 20 mg administered every 4 weeks (with an additional 20 mg loading dose (ie, 40 mg total) at both Week 0 and Week 4) in subjects with PV.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 Aug 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
    Australia: 3
    Country: Number of subjects enrolled
    Greece: 2
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    Israel: 4
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Poland: 5
    Country: Number of subjects enrolled
    Romania: 2
    Country: Number of subjects enrolled
    United States: 14
    Worldwide total number of subjects
    35
    EEA total number of subjects
    12
    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)
    34
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Sixty-nine subjects were screened.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ofatumumab
    Arm description
    		 Subject will receive subcutaneous administration of ofatumumab 20 mg once every 4 weeks through Week 56, with an additional 20 mg dose (that is 40mg total) at both Week 0 and Week 4.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ofatumumab
    Investigational medicinal product code
    Other name
    OMB157
    Pharmaceutical forms
    Concentrate and solvent for solution for injection/infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Ofatumamab was administered once every 4 weeks for a total of 56 weeks (total of 17 injections across 15 monthly dosing visits). Subjects received two loading doses of 20 mg SC injections (40 mg total) at the Baseline (Week 0) and Week 4 visits.

    Arm title
    		 Placebo
    Arm description
    		 Subject will receive subcutaneous administration of matching placebo of ofatumumab once every 4 weeks through Week 56, with an additional dose at both Week 0 and Week 4.
    Arm type
    		 Placebo

    Investigational medicinal product name
    matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for injection/infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Matching placebo was administered once every 4 weeks for a total of 56 weeks (total of 17 injections across 15 monthly dosing visits).Subjects received two loading doses of 20 mg SC injections (40 mg total) of matching placebo at the Baseline (Week 0) and Week 4 visits.

    Number of subjects in period 1
    		Ofatumumab Placebo
    Started
    17
    18
    Requiried Individualized Follow-up
    1 [1]
    0 [2]
    Completed
    2
    1
    Not completed
    15
    17
         Study closed/terminated
    14
    15
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    -
    1
         Lack of efficacy
    1
    -
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: One patient entered an individualized patient follow up per the protocol
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: One patient entered an individualized patient follow up per the protocol

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ofatumumab
    Reporting group description
    		 Subject will receive subcutaneous administration of ofatumumab 20 mg once every 4 weeks through Week 56, with an additional 20 mg dose (that is 40mg total) at both Week 0 and Week 4.

    Reporting group title
    Placebo
    Reporting group description
    		 Subject will receive subcutaneous administration of matching placebo of ofatumumab once every 4 weeks through Week 56, with an additional dose at both Week 0 and Week 4.

    Reporting group values
    		 Ofatumumab Placebo Total
    Number of subjects
    		 17 18 35
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 17 17 34
        From 65-84 years
    		 0 1 1
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 49.6 ( 8.70 ) 47.1 ( 11.20 ) -
    Sex: Female, Male
    Units: Subjects
        Female
    		 10 8 18
        Male
    		 7 10 17
    Race/Ethnicity, Customized
    Units: Subjects
        African American/African Heritage
    		 1 0 1
        American Indian or Alaskan Native
    		 1 2 3
        Asian - East Asian Heritage
    		 2 0 2
        Asian - Japanese Heritage
    		 1 1 2
        White - Arabic/North African Heritage
    		 2 1 3
        White - White/ Caucasian/ European Heritage
    		 10 14 24

    End points

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    End points reporting groups
    Reporting group title
    Ofatumumab
    Reporting group description
    		 Subject will receive subcutaneous administration of ofatumumab 20 mg once every 4 weeks through Week 56, with an additional 20 mg dose (that is 40mg total) at both Week 0 and Week 4.

    Reporting group title
    Placebo
    Reporting group description
    		 Subject will receive subcutaneous administration of matching placebo of ofatumumab once every 4 weeks through Week 56, with an additional dose at both Week 0 and Week 4.

    Primary: Number of subjects who experienced sustained remission on minimal steroid therapy

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    End point title
    		 Number of subjects who experienced sustained remission on minimal steroid therapy [1]
    End point description
    Time from randomization to the time of the subject’s initial reduction of prednisone/prednisolone dose to <=10 mg/day and maintained a dose <=10 mg/day with no new or nonhealing lesions for >=8 weeks and maintained the status until Week 60 was assessed. No participants met the criteria so no analysis was performed
    End point type
    Primary
    End point timeframe
    Baseline up to approximately 60 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 analysis was completed because no patients experienced sustained remission
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: participants
    0
    0
    No statistical analyses for this end point

    Primary: Duration of remission on minimal steroid therapy

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    End point title
    		 Duration of remission on minimal steroid therapy [2]
    End point description
    Sum of all periods of absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day up to Week 60 was assessed.
    End point type
    Primary
    End point timeframe
    Baseline up to approximately 60 weeks
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No analysis was completed
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: days
        arithmetic mean (standard deviation)
    168.0 ( 73.33 )
    122.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Percentage of subjects achieving remission on minimal steroid therapy at Week 60

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    End point title
    		 Percentage of subjects achieving remission on minimal steroid therapy at Week 60
    End point description
    Percentage of subjects who achieved absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day for > or = 8 weeks at Week 60 was assessed. Percentages were rounded up. Time to remission was not estimable.
    End point type
    Secondary
    End point timeframe
    Week 60
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: percentage of participants
    18
    6
    No statistical analyses for this end point

    Secondary: Time to remission while on minimal steroid therapy by Week 60.

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    End point title
    		 Time to remission while on minimal steroid therapy by Week 60.
    End point description
    Time from randomization to the time of the subject’s initial reduction of prednisone/prednisolone dose to <=10 mg/day and maintained dose at <=10 mg/day with no new or nonhealing lesions for >=8 weeks by Week 60 was assessed
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: days
        median (confidence interval 95%)
    99999 (57.0 to 99999)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Percentage of subjects achieving remission while off steroid therapy by Week 60

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    End point title
    		 Percentage of subjects achieving remission while off steroid therapy by Week 60
    End point description
    Percentage of subjects with initial reduction of all steroids for >=8 weeks with an absence of new or nonhealing (established) lesions by Week 60 were to be assessed. All subjects remained on prednisone/prednisolone so this endpoint could not be analyzed.Time to remission off steroid therapy also could not be analyzed
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: percentage of participants
    99999
    99999
    No statistical analyses for this end point

    Secondary: Number of days a subject maintained minimal steroid therapy by Week 60.

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    End point title
    		 Number of days a subject maintained minimal steroid therapy by Week 60.
    End point description
    Number of days a subject maintained minimal steroid therapy (an oral prednisone/prednisolone dose of ≤10 mg/day in the absence of new or nonhealing lesions) by Week 60.
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: days
        arithmetic mean (standard deviation)
    168 ( 73.33 )
    122.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Time to initial flare/relapse by Week 60

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    End point title
    		 Time to initial flare/relapse by Week 60
    End point description
    Time from randomization to the time of appearance of >=3 new lesions within 1 month that did not heal spontaneously within 1 week, or to the time when there was an extension of lesions that were present at the randomization by Week 60 was assessed. Confidence Intervals for Ofatumumab are not valid values. System limitations do not allow NE for not estimable
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: days
        median (confidence interval 95%)
    448 (99 to 99999)
    169.0 (86.0 to 284.0)
    No statistical analyses for this end point

    Secondary: Percentage of participants with no flare/relapse by Week 60. Values were rounded

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    End point title
    		 Percentage of participants with no flare/relapse by Week 60. Values were rounded
    End point description
    Percentage of participants achieving absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day and did not subsequently have a appearance of >=3 new lesions within 1 month that did not heal spontaneously within 1 week, or to the time when there was an extension of lesions that were present at the randomization by Week 60 was assessed. Percentages were rounded because of system limitation of not allowing a decimal
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: percentage of participants
        Week 2 no flare n=17,17
    100
    94
        Week 2 no new/nonhealing lesions n=17,17
    47
    22
        Week 4 no flare n=15,16
    88
    83
        Week 4 no new/nonhealing lesions n=15,16
    47
    39
        Week 6 no flare n=12,15
    71
    78
        Week 6 no new/nonhealing lesions n=12,15
    41
    33
        Week 8 no flare n=11,14
    65
    72
        Week 8 no new/nonhealing lesions n=11,14
    35
    33
        Week 12 no flare n=10,12
    59
    56
        Week 12 no new/nonhealing lesions n=10,12
    35
    17
        Week 16 no flare n=9,12
    53
    61
        Week 16 no new/nonhealing lesions n=9,12
    26
    17
        Week 20 no flare n=8,12
    47
    50
        Week 20 no new/nonhealing lesions n=8,12
    29
    17
        Week 24 no flare n=8,11
    47
    39
        Week 24 no new/nonhealing lesions n=8,11
    35
    17
        Week 28 no flare n=5,7
    29
    22
        Week 28 no new/nonhealing lesions n=5,7
    18
    11
        Week 32 no flare n=6,6
    35
    28
        Week 32 no new/nonhealing lesions n=6,6
    18
    6
        Week 36 no flare n=4,3
    24
    11
        Week 36 no new/nonhealing lesions n=4,3
    6
    6
        Week 40 no flare n=3,2
    18
    6
        Week 40 no new/nonhealing lesions n=
    12
    0
        Week 44 no flare n=3,2
    18
    6
        Week 44 no new/nonhealing lesions n=3,2
    6
    0
        Week 48 no flare n=3,1
    18
    0
        Week 48 no new/nonhealing lesions n=3,1
    0
    0
        Week 52 no flare n=2,1
    12
    6
        Week 52 no new/nonhealing lesions n=2,1
    12
    0
        Week 56 no flare n=2,1
    12
    0
        Week 56 no new/nonhealing lesions n=2,1
    6
    0
        Week 60 no flare n=17,17
    88
    83
        Week 60 no new/nonhealing lesions n=17,17
    53
    33
    No statistical analyses for this end point

    Secondary: Plasma trough concentrations of population pharmacokinetics (PK) of ofatumumab

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    End point title
    		 Plasma trough concentrations of population pharmacokinetics (PK) of ofatumumab [3]
    End point description
    Plasma (trough) concentrations of ofatumumab, Exposure-response relationship, PK parameters include: Maximum concentration (Cmax); time to maximum concentration (tmax); and area under the time-concentration curve (AUC).
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    Notes
    [3] - 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: No analysis was completed
    End point values
    		 Ofatumumab
    Number of subjects analysed
    17
    Units: ng/mL
    arithmetic mean (standard deviation)
        4 hours post dose SS n=1
    56.60 ( 99999 )
        Day 1 Steady State (SS) n=1
    1917.50 ( 99999 )
        Day 2 Steady State (SS) n=1
    2994.20 ( 99999 )
        Day 3 Steady State (SS) n=1
    3553.80 ( 99999 )
        Day 4 Steady State (SS) n=1
    3619.20 ( 99999 )
        Day 7 Steady State (SS) n=1
    3434.90 ( 99999 )
        Day 14 Steady State (SS) n=1
    2055.30 ( 99999 )
        Week 4 n=12
    311.59 ( 278.011 )
        Week 4 SS n=1
    1132.70 ( 99999 )
        Week 8 n=9
    1253.60 ( 439.574 )
        Week 8 SS n=2
    1456.95 ( 668.994 )
        Week 12 n=7
    727.01 ( 430.834 )
        Week 12 SS n=3
    900.00 ( 455.930 )
        Week 16 n=8
    512.88 ( 377.189 )
        Week 16 SS n=1
    1043.40 ( 99999 )
        Week 20 n=6
    415.88 ( 303.307 )
        Week 20 SS n=2
    720.10 ( 246.356 )
        Week 24 n=5
    461.72 ( 437.717 )
        Week 24 SS n=3
    686.10 ( 329.699 )
        Week 36 n=3
    477.63 ( 378.112 )
        Week 36 SS n=1
    931.20 ( 99999 )
        Week 48 n=2
    352.95 ( 300.591 )
        Week 48 SS n=1
    1230.40 ( 99999 )
        Week 52 SS n=1
    897.30 ( 99999 )
        Week 56 n=2
    436.40 ( 511.662 )
        Week 60 n=2
    76.40 ( 32.244 )
        Early withdrawal n=12
    925.91 ( 971.715 )
    No statistical analyses for this end point

    Secondary: Immunogenicity of ofatumumab - Immunoglobulin A - values below lower limit of normal

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    End point title
    		 Immunogenicity of ofatumumab - Immunoglobulin A - values below lower limit of normal
    End point description
    Immunogenicity will be assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: g/L
        Baseline n=17,18
    1
    0
        Week 12 n=10,11
    1
    0
        Week 16 n=0,1
    0
    0
        Week 24 n=8,11
    1
    0
        Week 36 n=4,3
    1
    0
        Week 48 n=3,1
    1
    0
        Week 52 n=2,1
    1
    0
        Week 56 n=2,1
    1
    0
        Week 60/Early withdrawal n=17,18
    0
    0
        First 12 weeks of therapy n=17,18
    1
    0
        During on therapy period n=17,18
    1
    0
    No statistical analyses for this end point

    Secondary: Immunogenicity of ofatumumab - Immunoglobulin G - values below lower limit of normal

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    End point title
    		 Immunogenicity of ofatumumab - Immunoglobulin G - values below lower limit of normal
    End point description
    Immunogenicity will be assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: g/L
        Baseline n=17,18
    1
    2
        Week 12 n=10,11
    0
    1
        Week 16 n=0,1
    0
    1
        Week 24 n=8,11
    0
    1
        Week 36 n=4,3
    0
    0
        Week 48 n=3,1
    0
    0
        Week 52 n=2,1
    0
    0
        Week 56 n=2,1
    0
    0
        Week 60/Early withdrawal n=17,18
    0
    2
        First 12 weeks of therapy n=17,18
    1
    3
        During on therapy period n=17,18
    1
    4
    No statistical analyses for this end point

    Secondary: Immunogenicity of ofatumumab - Immunoglobulin M - values below lower limit of normal

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    End point title
    		 Immunogenicity of ofatumumab - Immunoglobulin M - values below lower limit of normal
    End point description
    Immunogenicity will be assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: g/L
        Baseline n=17,18
    1
    1
        Week 12 n=10,11
    4
    1
        Week 24 n=8,11
    4
    0
        Week 36 n=4,3
    4
    0
        Week 48 n=3,1
    3
    0
        Week 52 n=2,1
    2
    0
        Week 56 n=2,1
    2
    0
        Week 60/Early withdrawal n=17,18
    6
    2
        First 12 weeks of therapy n=17,18
    4
    1
        During on therapy period n=17,18
    6
    2
    No statistical analyses for this end point

    Secondary: Immunogenicity of ofatumumab - largest decrease from baseline

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    End point title
    		 Immunogenicity of ofatumumab - largest decrease from baseline
    End point description
    Immunogenicity will be assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: g/L
    arithmetic mean (standard deviation)
        Immunoglobulin A n=13,11
    -0.185 ( 0.1181 )
    -0.472 ( 0.7116 )
        Immunoglobulin G n=11,13
    -1.172 ( 0.9390 )
    -0.955 ( 0.6205 )
        Immunoglobulin M n=17,11
    -0.231 ( 0.1152 )
    -0.263 ( 0.3053 )
    No statistical analyses for this end point

    Secondary: Change from Baseline for CD19+ B cell count

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    End point title
    		 Change from Baseline for CD19+ B cell count
    End point description
    CD19+ B cell count will be performed using Flow Cytometry. Week 16 data values were not estimable therefore were not presented.System limitations do not allow NE (not estimable) for standard deviation, substituted values of 99999 are not valid data values
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 60 weeks
    End point values
    		 Ofatumumab Placebo
    Number of subjects analysed
    17
    18
    Units: 10^9/L
    arithmetic mean (standard deviation)
        Week 12 n=10,10
    -0.24940 ( 0.228704 )
    0.03080 ( 0.072358 )
        Week 24 n=7,9
    -0.28607 ( 0.265327 )
    -0.04022 ( 0.197867 )
        Week 36 n=4,3
    -0.20525 ( 0.052703 )
    -0.10033 ( 0.021385 )
        Week 48 n=3,1
    -0.19717 ( 0.061436 )
    -0.00200 ( 99999 )
        Week 52 n=2,1
    -0.16300 ( 0.023335 )
    0.09600 ( 99999 )
        Week 56 n=2,1
    -0.16300 ( 0.023335 )
    0.01000 ( 99999 )
        Week 60/Early withdrawal n=17,18
    -0.21403 ( 0.190282 )
    0.00367 ( 0.190375 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV).  All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit up to approximately 60 weeks
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    Ofatumumab SC
    Reporting group description
    		 Ofatumumab SC

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Serious adverse events
    		 Ofatumumab SC Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Ofatumumab SC Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 17 (82.35%)
    8 / 18 (44.44%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Vascular disorders
    Hot flush
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Hypertension
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    General disorders and administration site conditions
    Chills
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 18 (0.00%)
         occurrences all number
    3
    0
    Fatigue
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    2
    2
    Influenza like illness
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Local reaction
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Malaise
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    2
    Pyrexia
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 18 (0.00%)
         occurrences all number
    5
    0
    Reproductive system and breast disorders
    Semen discolouration
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Epistaxis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Sinus congestion
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Persistent depressive disorder
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Investigations
    B-lymphocyte count decreased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Arthropod sting
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Post procedural complication
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Procedural nausea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Procedural pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    3
    Headache
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    Syncope
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Tremor
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Conjunctival discolouration
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Eye irritation
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Eye swelling
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Ocular hyperaemia
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Vision blurred
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Diarrhoea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nausea
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 18 (5.56%)
         occurrences all number
    3
    1
    Tongue ulceration
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Actinic keratosis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Dermatitis allergic
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Eczema
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    Rash
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 18 (11.11%)
         occurrences all number
    1
    2
    Renal and urinary disorders
    Calculus urinary
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nephrolithiasis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nocturia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    Metatarsalgia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Muscular weakness
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Musculoskeletal discomfort
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Myalgia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Plantar fasciitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Cellulitis
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    2
    Folliculitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Gastroenteritis viral
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Herpes simplex
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Influenza
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Oral candidiasis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Tooth abscess
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    3
    2
    Metabolism and nutrition disorders
    Diabetes mellitus
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Jun 2013
    • The definition of ‘remission’ was clarified such that subjects with existing, healing lesions could be considered in remission if other criteria were met. • It was clarified that all subjects, including early withdrawals from treatment, were to be transitioned to the Individualized Follow-up Period. • Exclusion criterion 4 was modified to include subjects with prior systemic antibiotic pemphigus treatments as treatment effects on pemphigus lesions are minimal but subjects with prior methotrexate treatment within 8 weeks of entry were excluded as were subjects with prior total body irradiation, bone marrow transplantation, or anti-CD4 at any time. • Exclusion criterion 6 was modified to remove the prior treatment with any lymphocyte depleting therapies information which was more appropriately included within the prohibited medications and non-drug therapies section. Exclusion criteria 11 was modified to exclude subjects with a potential risk factor ro adverse cardiac outcomes based on screening ECG.
    26 Sep 2013
    • The pharmacodynamics objective was clarified as a population pharmacodynamics objective. • New PK endpoints, of plasma (trough) concentrations of ofatumumab and exposure response relationship, were added to determine ofatumumab plasma concentrations across the whole study population. Exclusion 9 was amended to include subjects with isolated predominantly indirect hyperbilirubinemia or CD4 count < 500cells/mm3
    13 Mar 2014
    • The dose and dosing interval were changed from 60 mg every 12 weeks to 20 mg every 4 weeks with an additional 20 mg loading dose at both Week 0 and Week 4. Dose rationale details were updated with data and pharmacometric modelling to support the change, details of the supplied study treatment were updated and dilution details were added. • The 48-week Treatment Period was changed to a 56 week Treatment Period and the Follow-up Period was changed from 12 weeks to 4 weeks. This was due to the changed timing of last dose with subsequent change in initial follow-up to align with change in dosing interval.
    02 Apr 2015
    Exclusion criteria4 modified to include treatment with tacrolimus, alesmtuzumab, mitoxantrone and CD20 treatments.
    28 Sep 2015
    Exlusion 8 extended to include subjects requiring treatment with prednisone/prednisolone for conditions other than PV; addition of a minimum 1-year Individualized Follow-up Period for subjects who did not enroll in the extension study
    25 Apr 2016
    Criteria for maintaining subjects in the Individualized Follow-up Period were modified to allow for improved safety monitoring following last dose of study treatment, while facilitating the exit of subjects from the study to progress to alternative treatment if needed. Because the study had been terminated, text added to allow Sponsor unblinding of subjects’ randomized treatment assignments. This was to provide information for the Investigators’ continued monitoring of their subjects.
    05 Sep 2016
    • All mentions of GlaxoSmithKline/GSK were changed to Novartis. Legal registered and contact addresses updated. • Novartis Study IDs were added as follows: Core Study: OPV116910 (also known as COMB157J2301)

    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.
    Novartis terminated the development of the PV program and this study was terminated for non-safety reasons. As described in the End Point descriptions, due to system limitations not accepting NE=not estimable, 99999 values are not valid values
    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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