Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   38958   clinical trials with a EudraCT protocol, of which   6398   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects with Moderate to Severe Myasthenia Gravis

    Summary
    EudraCT number
    2016-002698-36
    Trial protocol
    DE   BE   ES   CZ   DK  
    Global end of trial date
    06 Aug 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    05 Sep 2019
    First version publication date
    05 Sep 2019
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    MG0002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03052751
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SPRL
    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
    04 Sep 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Aug 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe.
    Protection of trial subjects
    During the conduct of the study all subjects were closely monitored. If required, subjects received rescue therapy and were withdrawn from treatment. Subjects were then monitored during the 8 weeks observation period until lab levels returned to normal.
    Background therapy
    Background therapy as permitted in the protocol.
    Evidence for comparator
    Not Applicable
    Actual start date of recruitment
    15 May 2017
    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
    Canada: 11
    Country: Number of subjects enrolled
    Czech Republic: 2
    Country: Number of subjects enrolled
    Denmark: 7
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    Belgium: 10
    Country: Number of subjects enrolled
    United States: 9
    Worldwide total number of subjects
    43
    EEA total number of subjects
    23
    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)
    32
    From 65 to 84 years
    11
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    The study started to enroll patients in May 2017 and concluded in August 2018.

    Pre-assignment
    Screening details
    The Participant Flow refers to the Randomized Set (RS) which consisted of all participants randomized into the study at the first randomization visit.

    Period 1
    Period 1 title
    Dosing Period 1
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Assessor, Carer, Investigator, Subject
    Blinding implementation details
    For reporting of this study, due to limitations of the drop-down list for blinding, the wording Double Blind was utilized instead of Investigator- and Subject-Blind.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    PBO
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo was administered in Dosing Period 1.

    Arm title
    UCB7665 Dose 1
    Arm description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Number of subjects in period 1
    Placebo UCB7665 Dose 1
    Started
    22
    21
    Completed Period 1
    22
    21
    Completed Period 1 and started Period 2
    22
    20
    Completed
    22
    20
    Not completed
    0
    1
         Adverse event, non-fatal
    -
    1
    Period 2
    Period 2 title
    Dosing Period 2
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Assessor, Carer, Investigator, Subject
    Blinding implementation details
    For reporting of this study, due to limitations of the drop-down list for blinding, the wording Double Blind was utilized instead of Investigator- and Subject-Blind.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo - UCB7665 Dose 1
    Arm description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    Placebo - UCB7665 Dose 2
    Arm description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    UCB7665 Dose 1 - UCB7665 Dose 1
    Arm description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    UCB7665 Dose 1 - UCB7665 Dose 2
    Arm description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Number of subjects in period 2
    Placebo - UCB7665 Dose 1 Placebo - UCB7665 Dose 2 UCB7665 Dose 1 - UCB7665 Dose 1 UCB7665 Dose 1 - UCB7665 Dose 2
    Started
    11
    11
    10
    10
    Completed
    8
    11
    10
    10
    Not completed
    3
    0
    0
    0
         Adverse event, non-fatal
    3
    -
    -
    -
    Period 3
    Period 3 title
    Observation Period
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Assessor, Carer, Investigator, Subject
    Blinding implementation details
    For reporting of this study, due to limitations of the drop-down list for blinding, the wording Double Blind was utilized instead of Investigator- and Subject-Blind.

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Placebo
    Arm description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    PBO
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo was administered in Dosing Period 1.

    Arm title
    UCB7665 Dose 1
    Arm description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    Placebo - UCB7665 Dose 1
    Arm description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    Placebo - UCB7665 Dose 2
    Arm description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    UCB7665 Dose 1 - UCB7665 Dose 1
    Arm description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Arm title
    UCB7665 Dose 1 - UCB7665 Dose 2
    Arm description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 was administered in 2 different dosages (dose 1 and dose 2) in Dosing Period 1 and 2.

    Number of subjects in period 3
    Placebo UCB7665 Dose 1 Placebo - UCB7665 Dose 1 Placebo - UCB7665 Dose 2 UCB7665 Dose 1 - UCB7665 Dose 1 UCB7665 Dose 1 - UCB7665 Dose 2
    Started
    22
    21
    11
    11
    10
    10
    Completed
    22
    21
    11
    11
    9
    10
    Not completed
    0
    0
    0
    0
    1
    0
         Adverse event, non-fatal
    -
    -
    -
    -
    1
    -

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

    Reporting group title
    UCB7665 Dose 1
    Reporting group description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

    Reporting group values
    Placebo UCB7665 Dose 1 Total
    Number of subjects
    22 21 43
    Age categorical
    Units: Subjects
        <=18 years
    0 0 0
        Between 18 and 65 years
    14 18 32
        >=65 years
    8 3 11
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    53.3 ± 15.7 50.5 ± 14.7 -
    Gender categorical
    Units: Subjects
        Male
    8 8 16
        Female
    14 13 27
    Subject analysis sets

    Subject analysis set title
    Placebo (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Participants received 3 doses of placebo in dosing period 1. Participants formed the Full Analysis Set (FAS) which consisted of all participants in the Safety Set (SS) who had a baseline and at least 1 post-Baseline QMG measurement during Dosing Period 1 (up to and including Visit 9, ie, Day 29).

    Subject analysis set title
    UCB7665 Dose 1 (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1. Participants formed the Full Analysis Set (FAS) which consisted of all participants in the Safety Set (SS) who had a baseline and at least 1 post-Baseline QMG measurement during Dosing Period 1 (up to and including Visit 9, ie, Day 29).

    Subject analysis set title
    Placebo (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Subject analysis set title
    UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Subject analysis set title
    Placebo - UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    Placebo - UCB7665 Dose 2 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    UCB7665 Dose 1 - UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis sets values
    Placebo (FAS) UCB7665 Dose 1 (FAS) Placebo (SS) UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 2 (SS) UCB7665 Dose 1 - UCB7665 Dose 1 (SS) UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Number of subjects
    22
    21
    22
    21
    11
    11
    10
    10
    Age categorical
    Units: Subjects
        <=18 years
    0
    0
    0
    0
    0
    0
    0
    0
        Between 18 and 65 years
    14
    18
    14
    18
    8
    6
    9
    8
        >=65 years
    8
    3
    8
    3
    3
    5
    1
    2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    53.3 ± 15.7
    50.5 ± 14.7
    53.3 ± 15.7
    50.5 ± 14.7
    52.1 ± 14.4
    54.5 ± 17.5
    47.9 ± 16.0
    52.0 ± 14.2
    Gender categorical
    Units: Subjects
        Male
    8
    8
    8
    8
    5
    3
    4
    4
        Female
    14
    13
    14
    13
    6
    8
    6
    6

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

    Reporting group title
    UCB7665 Dose 1
    Reporting group description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).
    Reporting group title
    Placebo - UCB7665 Dose 1
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.

    Reporting group title
    Placebo - UCB7665 Dose 2
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 1
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 2
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.
    Reporting group title
    Placebo
    Reporting group description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

    Reporting group title
    UCB7665 Dose 1
    Reporting group description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2).

    Reporting group title
    Placebo - UCB7665 Dose 1
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.

    Reporting group title
    Placebo - UCB7665 Dose 2
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 1
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2.

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 2
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2.

    Subject analysis set title
    Placebo (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Participants received 3 doses of placebo in dosing period 1. Participants formed the Full Analysis Set (FAS) which consisted of all participants in the Safety Set (SS) who had a baseline and at least 1 post-Baseline QMG measurement during Dosing Period 1 (up to and including Visit 9, ie, Day 29).

    Subject analysis set title
    UCB7665 Dose 1 (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1. Participants formed the Full Analysis Set (FAS) which consisted of all participants in the Safety Set (SS) who had a baseline and at least 1 post-Baseline QMG measurement during Dosing Period 1 (up to and including Visit 9, ie, Day 29).

    Subject analysis set title
    Placebo (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Subject analysis set title
    UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Subject analysis set title
    Placebo - UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    Placebo - UCB7665 Dose 2 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    UCB7665 Dose 1 - UCB7665 Dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Subject analysis set title
    UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Primary: Change from Baseline in Quantitative Myasthenia Gravis (QMG) score to Visit 9

    Close Top of page
    End point title
    Change from Baseline in Quantitative Myasthenia Gravis (QMG) score to Visit 9
    End point description
    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). The score ranges from 0 to 39, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.
    End point type
    Primary
    End point timeframe
    From Baseline to Visit 9 (up to Day 29)
    End point values
    Placebo (FAS) UCB7665 Dose 1 (FAS)
    Number of subjects analysed
    22
    21
    Units: scores on a scale
        least squares mean (standard error)
    -1.2 ± 0.6
    -1.8 ± 0.6
    Statistical analysis title
    Statistical analysis 1
    Statistical analysis description
    Mixed Model Repeated Measures (MMRM) Analysis of Covariance (ANCOVA) model included fixed terms for treatment group, visit, interaction between treatment group and visit, covariate of Baseline QMG score, and random effect for participant. The differences presented was 'UCB7665 Dose 1 minus Placebo'.
    Comparison groups
    Placebo (FAS) v UCB7665 Dose 1 (FAS)
    Number of subjects included in analysis
    43
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.221 [1]
    Method
    MMRM
    Parameter type
    LS Mean Difference vs Placebo
    Point estimate
    -0.7
    Confidence interval
         level
    95%
         sides
    1-sided
         lower limit
    -
         upper limit
    0.8
    Notes
    [1] - One-sided p-value was presented for difference.

    Secondary: Change from Baseline in Myasthenia Gravis-Composite score to Visit 9

    Close Top of page
    End point title
    Change from Baseline in Myasthenia Gravis-Composite score to Visit 9
    End point description
    The total Myasthenia Gravis (MG)-composite score was obtained by summing the responses to each individual item (10 items; Grade: 0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.
    End point type
    Secondary
    End point timeframe
    From Baseline to Visit 9 (up to Day 29)
    End point values
    Placebo (FAS) UCB7665 Dose 1 (FAS)
    Number of subjects analysed
    22
    21
    Units: scores on a scale
        least squares mean (standard error)
    -1.2 ± 0.9
    -3.1 ± 0.9
    Statistical analysis title
    Statistical analysis 1
    Statistical analysis description
    MMRM ANCOVA model included fixed terms for treatment group, visit, interaction between treatment group and visit, covariate of Baseline MG-composite score, and random effect for participant. The differences presented was 'UCB7665 Dose 1 minus Placebo'.
    Comparison groups
    Placebo (FAS) v UCB7665 Dose 1 (FAS)
    Number of subjects included in analysis
    43
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.089 [2]
    Method
    MMRM
    Parameter type
    LS Mean Difference vs Placebo
    Point estimate
    -1.8
    Confidence interval
         level
    95%
         sides
    1-sided
         lower limit
    -
         upper limit
    0.4
    Notes
    [2] - One-sided p-value was presented for difference.

    Secondary: Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) score to Visit 9

    Close Top of page
    End point title
    Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) score to Visit 9
    End point description
    The total MGDAL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with lower scores indicating lower disease activity. The change from Baseline is calculated, a negative value indicating improvement and a positive value worsening.
    End point type
    Secondary
    End point timeframe
    From Baseline to Visit 9 (up to Day 29)
    End point values
    Placebo (FAS) UCB7665 Dose 1 (FAS)
    Number of subjects analysed
    22
    21
    Units: scores on a scale
        least squares mean (standard error)
    -0.4 ± 0.5
    -1.8 ± 0.5
    Statistical analysis title
    Statistical analysis 1
    Statistical analysis description
    ANCOVA model included fixed terms for treatment group, covariate of Baseline MGADL score.
    Comparison groups
    Placebo (FAS) v UCB7665 Dose 1 (FAS)
    Number of subjects included in analysis
    43
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.036 [3]
    Method
    ANCOVA
    Parameter type
    LS Mean Difference vs Placebo
    Point estimate
    -1.4
    Confidence interval
         level
    95%
         sides
    1-sided
         lower limit
    -
         upper limit
    -0.1
    Notes
    [3] - One-sided p-value was presented for difference.

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From the start of Treatment Period at Baseline and up to Observation Period at 8 weeks after the final dose of IMP.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    Placebo (SS)
    Reporting group description
    Participants received 3 doses of placebo in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Reporting group title
    UCB7665 Dose 1 (SS)
    Reporting group description
    Participants received 3 doses of UCB7665 (dose 1) in dosing period 1 and then were re-randomized into dosing period 2 to receive 3 doses of UCB7665 (dose 1 or dose 2). Participants formed the Safety Set (SS) which consisted of all participants in the Randomized Set (RS) who had received at least 1 dose of investigational product (IMP).

    Reporting group title
    Placebo - UCB7665 Dose 1 (SS)
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Reporting group title
    Placebo - UCB7665 Dose 2 (SS)
    Reporting group description
    Participants randomized to receive 3 doses of placebo at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 1 (SS)
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Reporting group title
    UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Reporting group description
    Participants randomized to receive 3 doses of UCB7665 dose 1 at weekly intervals in dosing period 1 were then re-randomized to receive 3 doses of UCB7665 dose 2 at weekly intervals in dosing period 2. Participants formed the Safety Set (SS).

    Serious adverse events
    Placebo (SS) UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 2 (SS) UCB7665 Dose 1 - UCB7665 Dose 1 (SS) UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 22 (9.09%)
    0 / 21 (0.00%)
    3 / 11 (27.27%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Ulna fracture
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Presyncope
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myasthenia gravis
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myasthenia gravis crisis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo (SS) UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 1 (SS) Placebo - UCB7665 Dose 2 (SS) UCB7665 Dose 1 - UCB7665 Dose 1 (SS) UCB7665 Dose 1 - UCB7665 Dose 2 (SS)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 22 (31.82%)
    13 / 21 (61.90%)
    7 / 11 (63.64%)
    9 / 11 (81.82%)
    8 / 10 (80.00%)
    9 / 10 (90.00%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    3 / 22 (13.64%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    4
    0
    1
    3
    0
    0
    Gait disturbance
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    3
    0
    0
    0
    Infusion site pruritus
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    3
    0
    Infusion site swelling
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Pyrexia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Asthenia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Inflammatory pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Infusion site reaction
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Stress
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Injury corneal
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Investigations
    Lymphocyte count decreased
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Cardiac disorders
    Bundle branch block left
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    2 / 11 (18.18%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    3
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    2
    1
    0
    Dysphonia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Laryngeal inflammation
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Orthopnoea
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Paranasal sinus discomfort
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 22 (13.64%)
    12 / 21 (57.14%)
    3 / 11 (27.27%)
    2 / 11 (18.18%)
    4 / 10 (40.00%)
    4 / 10 (40.00%)
         occurrences all number
    5
    22
    4
    2
    5
    6
    Dizziness
         subjects affected / exposed
    3 / 22 (13.64%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    1 / 10 (10.00%)
         occurrences all number
    3
    0
    0
    0
    2
    1
    Myasthenic syndrome
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    2
    1
    Dysarthria
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Hypoaesthesia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Eye disorders
    Diplopia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Eyelid ptosis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Keratitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vision blurred
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 22 (9.09%)
    3 / 21 (14.29%)
    1 / 11 (9.09%)
    4 / 11 (36.36%)
    1 / 10 (10.00%)
    2 / 10 (20.00%)
         occurrences all number
    2
    3
    1
    4
    1
    2
    Nausea
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 21 (9.52%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    3
    0
    0
    2
    2
    Vomiting
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Abdominal pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Cheilitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Muscular weakness
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    2 / 11 (18.18%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    2
    0
    0
    1
    Back pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Limb discomfort
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Musculoskeletal pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Arthralgia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Groin pain
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Muscle spasms
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Fluid retention
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Hypophosphataemia
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 21 (9.52%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    2
    1
    1
    0
    0
    Cystitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Bronchitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    3 / 22 (13.64%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    0
    0
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Rhinitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 10 (10.00%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Urinary tract infection
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 21 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 10 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Feb 2017
    Protocol Amendment 1 (dated 07 Feb 2017) was implemented to incorporate the feedback from the US Food and Drug administration (FDA) received on 09 December 2016 and the new company standard in tuberculosis (TB) management. Secondary efficacy variables and other efficacy variables were updated. In addition, minor administrative changes were made.
    15 Sep 2017
    Protocol Amendment 2 (dated 15 Sep 2017) was implemented to include an additional patient-reported outcome (PRO) assessment, intended as a measure of disease severity. This patient-centered index was developed for an outpatient setting and is sensitive to detect clinical change after interventions, and most importantly detect patient-meaningful change. Two additional other efficacy variables were added to capture changes for mean consecutive difference (MCD) and normal fiber pairs in jitter (single-fiber electromyogram [SFEMG]) studies to show the clinical trend from predose to postdose treatment. Furthermore, permitted concomitant medications were revised to include: modified and unmodified dose formulations for cyclosporin and increased dose strength for tacrolimus. With no specific dosing for tacrolimus, dosing is variable in patients with myasthenia gravis (MG), but for transplant related immunosuppression, higher doses are cited in literature; therefore, a slightly higher fixed dose was allowed. In addition, minor administrative and stylistic changes were made.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA