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    Clinical Trial Results:
    A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

    Summary
    EudraCT number
    2019-000969-21
    Trial protocol
    HU   GB   DK   BE   DE   ES   CZ   PL   IT  
    Global end of trial date
    01 Sep 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Sep 2022
    First version publication date
    14 Sep 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MG0004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04124965
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.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
    01 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Sep 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Sep 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate the long-term safety and tolerability of rozanolixizumab in study participants with generalized myasthenia gravis (MG)
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    Not applicable
    Evidence for comparator
    Not applicable
    Actual start date of recruitment
    29 Oct 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Efficacy, Safety
    Long term follow-up duration
    2 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 10
    Country: Number of subjects enrolled
    Czechia: 2
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    France: 12
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Japan: 6
    Country: Number of subjects enrolled
    Poland: 7
    Country: Number of subjects enrolled
    Russian Federation: 6
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Taiwan: 2
    Country: Number of subjects enrolled
    United States: 17
    Worldwide total number of subjects
    71
    EEA total number of subjects
    30
    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)
    55
    From 65 to 84 years
    14
    85 years and over
    2

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll study participants in Oct 2019 and concluded in Sep 2021.

    Pre-assignment
    Screening details
    Participant Flow refers to the Randomized Set which consisted of all study participants who were randomized, using the treatment assigned instead of the actual treatment received.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Rozanolixizumab ~7 mg/kg
    Arm description
    Participants received rozanolixizumab equivalent to approximately 7 milligrams/kilogram (mg/kg), subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received rozanolixizumab equivalent to 7 mg/kg subcutaneously on a weekly basis over a 52-week Treatment Period.

    Arm title
    Rozanolixizumab ~10 mg/kg
    Arm description
    Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received rozanolixizumab equivalent to 10 mg/kg subcutaneously on a weekly basis over a 52-week Treatment Period.

    Number of subjects in period 1
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Started
    35
    36
    Safety Set
    35
    35
    Completed
    5
    3
    Not completed
    30
    33
         Personal surgery
    -
    1
         Physician decision
    -
    1
         Sponsor and participant decision
    -
    1
         Rolled Over To MG0007 Study
    25
    28
         Pregnancy
    1
    -
         Adverse event, non-fatal
    3
    1
         Consent withdrawn by subject
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 7 milligrams/kilogram (mg/kg), subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).

    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).

    Reporting group values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg Total
    Number of subjects
    35 36 71
    Age Categorical
    Units: participants
        <=18 years
    0 0 0
        Between 18 and 65 years
    29 26 55
        >=65 years
    6 10 16
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    50.6 ± 14.2 53.7 ± 17.2 -
    Sex: Female, Male
    Units: participants
        Female
    19 19 38
        Male
    16 17 33

    End points

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    End points reporting groups
    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 7 milligrams/kilogram (mg/kg), subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).

    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60).

    Subject analysis set title
    Rozanolixizumab ~7 mg/kg
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). This set included participants that switched to Rozanolixizumab equivalent to approximately 10 mg/kg at least once during the study.

    Subject analysis set title
    Rozanolixizumab ~10 mg/kg
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). This set included participants that switched to Rozanolixizumab equivalent to approximately 7 mg/kg at least once during the study.

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs)

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    End point title
    Percentage of participants with treatment-emergent adverse events (TEAEs) [1]
    End point description
    A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. The Safety Set (SS) consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab (RLZ) doses.
    End point type
    Primary
    End point timeframe
    From Baseline until End of Study (up to Week 60)
    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 formal statistical hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    50
    42
    Units: percentage of participants
        number (not applicable)
    76.0
    78.6
    No statistical analyses for this end point

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication

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    End point title
    Percentage of participants with treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication [2]
    End point description
    A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab doses.
    End point type
    Primary
    End point timeframe
    From Baseline until End of Study (up to Week 60)
    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 formal statistical hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    50
    42
    Units: percentage of participants
        number (not applicable)
    6.0
    0
    No statistical analyses for this end point

    Secondary: Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at each scheduled assessment during Treatment and Observation Periods

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    End point title
    Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at each scheduled assessment during Treatment and Observation Periods
    End point description
    MG-ADL is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the QMG. The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement. The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed (n) signifies those who were evaluable at specified time points. 99999 signifies that as pre-specified in the Statistical Analysis Plan, Standard Deviation was only calculated if there were a minimum of 4 participants.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    35
    35
    Units: units on a scale
    arithmetic mean (standard deviation)
        Week 5 (n=34, 34)
    -2.7 ± 3.3
    -3.2 ± 3.8
        Week 7 (n=35, 32)
    -2.7 ± 3.8
    -3.7 ± 3.4
        Week 9 (n=29, 31)
    -2.8 ± 3.4
    -3.4 ± 3.7
        Week 13 (n=30, 30)
    -3.1 ± 3.4
    -3.9 ± 4.0
        Week 17 (n=25, 29)
    -2.8 ± 3.3
    -4.0 ± 3.9
        Week 21 (n=20, 24)
    -3.0 ± 3.4
    -4.1 ± 4.3
        Week 25 (n=18, 17)
    -2.7 ± 3.0
    -3.7 ± 4.7
        Week 29 (n=13, 14)
    -2.8 ± 2.1
    -3.6 ± 4.3
        Week 33 (n=10, 12)
    -3.0 ± 2.8
    -3.5 ± 4.5
        Week 37 (n=7, 10)
    -3.9 ± 2.5
    -3.6 ± 3.6
        Week 41 (n=6, 6)
    -2.8 ± 2.1
    -1.8 ± 1.0
        Week 45 (n=4, 7)
    -3.8 ± 2.4
    -2.1 ± 1.1
        Week 49 (n=5, 6)
    -2.4 ± 1.1
    -0.5 ± 3.7
        Week 52 (n=5, 3)
    -2.6 ± 1.3
    -2.0 ± 99999
        Week 60 (n=7, 7)
    -0.3 ± 2.1
    -1.3 ± 3.9
    No statistical analyses for this end point

    Secondary: Change from Baseline in Myasthenia Gravis-Composite (MG-C) total score at each scheduled assessment during Treatment and Observation Periods

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    End point title
    Change from Baseline in Myasthenia Gravis-Composite (MG-C) total score at each scheduled assessment during Treatment and Observation Periods
    End point description
    MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. Items: ptosis/upward gaze (range: 0 [>45 second] -3 [Immediate]), double vision on lateral gaze (0 [>45 second] -4 [Immediate]), eye closure (0 [Normal] -2 [severe weakness]), talking (0 [Normal] -6 [difficult to understand speech]), chewing & swallowing (0 [Normal] -6 [gastric tube]), breathing (0 [Normal] -9 [ventilator dependence]), neck flexion (0 [Normal] -4 [severe weakness]), shoulder abduction & hip felxion (0 [Normal] -5 [severe weakness]), lower scores= lower disease activity. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50,lower scores=lower disease activity.A positive change=worsening and a negative change=improvement.Analysis population was SS.n= participants evaluable at specified time points.99999=mean and S.D. was calculated if there were a minimum of 3 participants and 4 participants respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    35
    35
    Units: units on a scale
    arithmetic mean (standard deviation)
        Week 5 (n=34, 35)
    -4.7 ± 5.5
    -5.5 ± 7.3
        Week 7 (n=35, 33)
    -5.0 ± 5.7
    -7.1 ± 7.4
        Week 9 (n=29, 30)
    -5.0 ± 5.3
    -6.0 ± 7.4
        Week 13 (n=30, 30)
    -4.8 ± 5.5
    -7.0 ± 7.8
        Week 17 (n=25, 29)
    -4.4 ± 5.7
    -5.5 ± 8.6
        Week 21 (n=20, 24)
    -5.3 ± 6.9
    -7.3 ± 8.1
        Week 25 (n=18, 17)
    -6.1 ± 5.8
    -8.8 ± 7.7
        Week 29 (n=13, 14)
    -5.1 ± 5.5
    -9.1 ± 9.2
        Week 33 (n=9, 12)
    -4.6 ± 5.1
    -8.4 ± 8.6
        Week 37 (n=7, 10)
    -6.3 ± 6.8
    -8.6 ± 6.9
        Week 41 (n=6, 6)
    -6.0 ± 7.1
    -6.5 ± 5.8
        Week 45 (n=4, 7)
    -4.0 ± 6.2
    -5.3 ± 4.9
        Week 49 (n=5, 6)
    -1.4 ± 3.8
    -0.8 ± 9.2
        Week 52 (n=5, 2)
    -3.8 ± 3.1
    99999 ± 99999
        Week 60 (n=7, 7)
    1.7 ± 3.7
    -2.3 ± 8.5
    No statistical analyses for this end point

    Secondary: Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at each scheduled assessment during Treatment and Observation Periods

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    End point title
    Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at each scheduled assessment during Treatment and Observation Periods
    End point description
    The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement. The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points. 99999 signifies that as pre-specified in the Statistical Analysis Plan, mean was only calculated if there were a minimum of 3 participants and standard deviation was only calculated if there were a minimum of 4 participants.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    35
    35
    Units: units on a scale
    arithmetic mean (standard deviation)
        Week 5 (n=34, 34)
    -2.9 ± 4.7
    -4.5 ± 4.4
        Week 7 (n=35, 32)
    -3.3 ± 4.4
    -5.2 ± 4.3
        Week 9 (n=28, 30)
    -3.3 ± 3.8
    -4.7 ± 4.3
        Week 13 (n=30, 29)
    -2.6 ± 4.2
    -5.5 ± 4.4
        Week 17 (n=25, 28)
    -3.1 ± 4.9
    -4.2 ± 4.4
        Week 21 (n=20, 23)
    -4.0 ± 4.7
    -5.1 ± 4.9
        Week 25 (n=18, 16)
    -5.1 ± 4.6
    -5.7 ± 5.5
        Week 29 (n=13, 13)
    -5.4 ± 3.6
    -5.5 ± 6.3
        Week 33 (n=10, 11)
    -4.9 ± 4.8
    -6.2 ± 5.7
        Week 37 (n=7, 9)
    -5.3 ± 3.9
    -6.8 ± 5.9
        Week 41 (n=6, 5)
    -5.7 ± 4.3
    -4.0 ± 3.1
        Week 45 (n=4, 6)
    -5.3 ± 2.8
    -4.0 ± 3.5
        Week 49 (n=5, 5)
    -3.8 ± 0.8
    -1.8 ± 1.8
        Week 52 (n=5, 2)
    -4.6 ± 2.9
    99999 ± 99999
        Week 60 (n=7, 6)
    -0.9 ± 2.4
    -1.8 ± 5.1
    No statistical analyses for this end point

    Secondary: Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX))

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    End point title
    Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX))
    End point description
    Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator. The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study.
    End point type
    Secondary
    End point timeframe
    From Baseline until End of Study (up to Week 60)
    End point values
    Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    35
    35
    Units: percentage of participants
        number (not applicable)
    11.4
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline until End of Study (up to Week 60)
    Adverse event reporting additional description
    TEAEs are reported in the safety section. TEAEs were planned to be analyzed using SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both RLZ doses.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). This set included participants that switched to Rozanolixizumab equivalent to approximately 7 mg/kg at least once during the study.

    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 52-week Treatment Period. After 52-week Treatment Period, participants were followed up for 8 weeks (up to Week 60). This set included participants that switched to Rozanolixizumab equivalent to approximately 10 mg/kg at least once during the study.

    Serious adverse events
    Rozanolixizumab ~10 mg/kg Rozanolixizumab ~7 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 42 (4.76%)
    7 / 50 (14.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Biopsy kidney abnormal
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 50 (2.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure congestive
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 50 (2.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pericarditis
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 50 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Myasthenia gravis
         subjects affected / exposed
    1 / 42 (2.38%)
    3 / 50 (6.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Retinal detachment
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 50 (2.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Muscular weakness
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 50 (2.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Rozanolixizumab ~10 mg/kg Rozanolixizumab ~7 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    25 / 42 (59.52%)
    27 / 50 (54.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 42 (2.38%)
    3 / 50 (6.00%)
         occurrences all number
    1
    3
    Investigations
    Blood immunoglobulin G decreased
         subjects affected / exposed
    5 / 42 (11.90%)
    6 / 50 (12.00%)
         occurrences all number
    6
    12
    Immune system disorders
    Hypogammaglobulinaemia
         subjects affected / exposed
    2 / 42 (4.76%)
    2 / 50 (4.00%)
         occurrences all number
    2
    4
    Nervous system disorders
    Headache
         subjects affected / exposed
    12 / 42 (28.57%)
    15 / 50 (30.00%)
         occurrences all number
    40
    55
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 42 (7.14%)
    4 / 50 (8.00%)
         occurrences all number
    4
    5
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    7 / 42 (16.67%)
    6 / 50 (12.00%)
         occurrences all number
    10
    11
    Abdominal pain
         subjects affected / exposed
    2 / 42 (4.76%)
    2 / 50 (4.00%)
         occurrences all number
    2
    2
    Nausea
         subjects affected / exposed
    5 / 42 (11.90%)
    4 / 50 (8.00%)
         occurrences all number
    8
    4
    Vomiting
         subjects affected / exposed
    4 / 42 (9.52%)
    0 / 50 (0.00%)
         occurrences all number
    4
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    4 / 42 (9.52%)
    1 / 50 (2.00%)
         occurrences all number
    7
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 50 (4.00%)
         occurrences all number
    3
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    4 / 42 (9.52%)
    2 / 50 (4.00%)
         occurrences all number
    4
    2
    Urinary tract infection
         subjects affected / exposed
    2 / 42 (4.76%)
    5 / 50 (10.00%)
         occurrences all number
    2
    5

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Nov 2019
    Protocol Amendment 1 (dated 01 Nov 2019) was a substantial amendment implemented to reference to another lead-in study, MGC003, throughout the protocol; however, MGC003 was not conducted. Other changes included additional wording to allow study participants to enroll into a substudy at selected sites (however, no study participants were included in the substudy), addition of 2 exploratory objective and endpoints (1 to assess the effect of rozanolixizumab on tetanus IgG antibodies, and 1 to capture the reduction steroid use in study participants receiving rozanolixizumab) and changes throughout the Schedule of Assessments.
    30 Jul 2020
    Protocol Amendment 2 (dated 30 Jul 2020) was a substantial amendment implemented to introduce the transition of study participants to MG0007 and closure of MG0004, once MG0007 was available as the open-label study to MG0003. Other changes included updates to decrease the complexity of assessments to be performed; to clarify some operational aspects of the study; to incorporate the harmonization of inclusion criteria with studies performed across the rozanolixizumab clinical development program; and to include the management of study participant treatment during the coronavirus disease 2019 (COVID-19) pandemic including contingency measures.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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