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    Clinical Trial Results:
    A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis

    Summary
    EudraCT number
    		 2021-001200-15
    Trial protocol
    		 DE   FR   ES   IT  
    Global end of trial date
    08 May 2024

    Results information
    Results version number
    		 v2(current)
    This version publication date
    28 Feb 2025
    First version publication date
    06 Nov 2024
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1210-DM-310
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04999020
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Inc.
    Sponsor organisation address
    121 Seaport Boulevard, Boston, MA, United States, 02210
    Public contact
    European Clinical Trial Information, Alexion Pharmaceuticals Inc., +35 3874162507, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +35 3874162507, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    08 May 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 May 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to determine the effect of ravulizumab compared with placebo in the treatment of dermatomyositis (DM) based on improvement in Total Improvement Score (TIS) International Myositis Assessment and Clinical Studies Total Improvement Score (IMAC-TIS).
    Protection of trial subjects
    This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, applicable International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and other applicable laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    19 Nov 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 5
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    Japan: 1
    Country: Number of subjects enrolled
    Korea, Republic of: 4
    Country: Number of subjects enrolled
    Spain: 2
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 8
    Worldwide total number of subjects
    38
    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)
    33
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was planned to be conducted in 2 parts – Part A and Part B. The study was terminated early and participants were not enrolled into Part B. Therefore, results are presented for Part A of the study only. Part A consisted of a Randomized Controlled Period (RCP) and an Open-Label Extension (OLE) period.

    Period 1
    Period 1 title
    Randomized Controlled Period (RCP)
    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
    		 RCP: Ravulizumab
    Arm description
    		 Participants received a loading dose of ravulizumab on Day 1 followed by a maintenance dose at Week 2 and then ravulizumab once every 8 weeks (Q8W) during the 26-week RCP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Ravulizumab per dosage and administration specified in the arm description.

    Arm title
    		 RCP: Placebo
    Arm description
    		 Participants received placebo on Day 1, and Weeks 2, 10, and 18 during the 26-week RCP.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Placebo per administration specified in the arm description.

    Number of subjects in period 1
    		RCP: Ravulizumab RCP: Placebo
    Started
    26
    12
    Received at Least 1 Dose of Treatment
    26
    12
    Completed
    22
    9
    Not completed
    4
    3
         Consent withdrawn by subject
    1
    2
         Physician decision
    1
    1
         Adverse event, non-fatal
    2
    -
    Period 2
    Period 2 title
    Open-Label Extension (OLE) Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 OLE: Ravulizumab to Ravulizumab
    Arm description
    		 Participants who received ravulizumab during the RCP continued to receive ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Ravulizumab per dosage and administration specified in the arm description.

    Arm title
    		 OLE: Placebo to Ravulizumab
    Arm description
    		 Participants who received placebo during the RCP received ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Ravulizumab per dosage and administration specified in the arm description.

    Number of subjects in period 2
    		OLE: Ravulizumab to Ravulizumab OLE: Placebo to Ravulizumab
    Started
    22
    9
    Received at Least 1 Dose of Treatment
    22
    9
    Completed
    0
    0
    Not completed
    22
    9
         Consent withdrawn by subject
    3
    3
         Physician decision
    1
    -
         Adverse event, non-fatal
    -
    2
         Study Terminated by Sponsor
    17
    4
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    RCP: Ravulizumab
    Reporting group description
    		 Participants received a loading dose of ravulizumab on Day 1 followed by a maintenance dose at Week 2 and then ravulizumab once every 8 weeks (Q8W) during the 26-week RCP.

    Reporting group title
    RCP: Placebo
    Reporting group description
    		 Participants received placebo on Day 1, and Weeks 2, 10, and 18 during the 26-week RCP.

    Reporting group values
    		 RCP: Ravulizumab RCP: Placebo Total
    Number of subjects
    		 26 12 38
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 24 9 33
        From 65-84 years
    		 2 3 5
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 50.7 ( 10.38 ) 59.3 ( 9.31 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 18 9 27
        Male
    		 8 3 11
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 3 2 5
        Not Hispanic or Latino
    		 23 10 33
        Unknown or Not Reported
    		 0 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 4 2 6
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 3 0 3
        White
    		 18 10 28
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 1 0 1

    End points

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    End points reporting groups
    Reporting group title
    RCP: Ravulizumab
    Reporting group description
    		 Participants received a loading dose of ravulizumab on Day 1 followed by a maintenance dose at Week 2 and then ravulizumab once every 8 weeks (Q8W) during the 26-week RCP.

    Reporting group title
    RCP: Placebo
    Reporting group description
    		 Participants received placebo on Day 1, and Weeks 2, 10, and 18 during the 26-week RCP.
    Reporting group title
    OLE: Ravulizumab to Ravulizumab
    Reporting group description
    		 Participants who received ravulizumab during the RCP continued to receive ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.

    Reporting group title
    OLE: Placebo to Ravulizumab
    Reporting group description
    		 Participants who received placebo during the RCP received ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.

    Primary: Number of Participants with International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period

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    End point title
    		 Number of Participants with International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period
    End point description
    Data are presented for the number of participants with a TIS40 response, defined as an IMACS-TIS score ≥ 40 at Week 26. IMACS-TIS is a clinical instrument that encompasses 6 core set measure (CSMs) (physician, patient, extra-muscular global activity, muscle strength, Health Assessment Questionnaire [HAQ], and muscle enzyme levels). A Total Improvement Score (TIS: 0–100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. A higher score indicated greater improvement. TIS40 was considered a moderate improvement score. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Primary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    9
    6
    Statistical analysis title
    		 Ravulizumab vs placebo
    Comparison groups
    RCP: Ravulizumab v RCP: Placebo
    Number of subjects included in analysis
    38
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4192
    Method
    		 Barnard’s unconditional exact test
    Parameter type
    		 Difference in response rates
    Point estimate
    -15.38
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -38.55
         upper limit
    		 8.48

    Secondary: TIS at Week 26

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    End point title
    		 TIS at Week 26
    End point description
    TIS scores ranged from 0–100 with higher scores indicating a greater improvement. Scores were determined by summing scores in each of the 6 CSMs of the IMAC (physician, patient, extra-muscular global activity, muscle strength, HAQ, and muscle enzyme levels). Clinically meaningful thresholds for improvement were defined as ≥ 20 point improvement response on IMACS-TIS (TIS20; mild), ≥ 40 point improvement response on IMACS TIS (TIS40; moderate) and ≥ 60 point improvement response on IMACS-TIS (TIS60; severe). Scores were based on the improvement and relative weight of each CSM. Data are presented for TIS (least squares mean) at Week 26. Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    31.16 ( 4.185 )
    43.28 ( 6.650 )
    No statistical analyses for this end point

    Secondary: Change from Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26

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    End point title
    		 Change from Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26
    End point description
    The CDASI is an instrument that separately measures activity and damage in the skin of dermatomyositis (DM) participants. It is a 1-page instrument that contains 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis). CDASI score is calculated by rating the severity of skin disease in 15 anatomical locations on the body based on activity and damage components. CDASI was completed by the Clinician/Clinician-Investigator. Total CDASI scores ranged from 0-100, higher scores =greater disease severity. Change from baseline in CDASI Total Activity Score at Week 26 was analyzed using a mixed model repeated measures (MMRM). The MMRM model included the observed Total Activity Score values at post baseline visits (Week 26) as the dependent variable. Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    -3.80 ( 1.249 )
    -7.47 ( 2.021 )
    No statistical analyses for this end point

    Secondary: Number of Participants with Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26

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    End point title
    		 Number of Participants with Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26
    End point description
    Laboratory tests were conducted to measure serum activities of muscle associated enzymes including creatine kinase (CK), alanine aminotransferase (ALT), aspartate aminotransferase (AST), lactate dehydrogenase (LDH), and aldolase. Data are presented for the number of participants who had an abnormal muscle enzyme at baseline that had been normalized at Week 26. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    4
    1
    No statistical analyses for this end point

    Secondary: Change from Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26

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    End point title
    		 Change from Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26
    End point description
    The MDAAT assesses disease activity of extra-muscular organ systems and muscles in participants with DM. The validated MDAAT tool measures the degree of disease activity of extra-muscular organ systems and muscle on a 0-10 centimeter (cm) visual analog scale (VAS). Extra-muscular activity ranged between 0 and 10, where, 0 cm = absent and 10 cm = maximum disease activity. Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    -0.92 ( 0.383 )
    -2.13 ( 0.614 )
    No statistical analyses for this end point

    Secondary: Change from Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26

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    End point title
    		 Change from Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26
    End point description
    The physician global activity assessment provides an overall rating of disease activity related to myositis. Disease activity is judged by the physician based on all information available at the time of evaluation, including the participant’s appearance, medical history, physical examination, laboratory testing, and prescribed medical therapy. The global disease activity score is recorded on a 10-cm VAS, where 0 cm= no evidence of disease activity and 10 cm= extremely severe disease activity. Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    -1.18 ( 0.413 )
    -1.97 ( 0.649 )
    No statistical analyses for this end point

    Secondary: Change from Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26

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    End point title
    		 Change from Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26
    End point description
    The purpose of the MMT-8 was to measure muscle strength as part of the physical examination. It included a subset of 8 muscle groups: neck flexors, deltoids, biceps, wrist, extensors, gluteus maximus and medius, quadriceps, and ankle dorsiflexors. Total MMT8 scores ranged from 0 (lowest strength) to 150 (highest strength). Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    9.5 ( 1.85 )
    12.6 ( 2.98 )
    No statistical analyses for this end point

    Secondary: Change from Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26

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    End point title
    		 Change from Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26
    End point description
    The patient global activity assessment provides an overall rating of disease activity related to myositis from the participant’s perspective. Participants were asked to consider all of the active inflammation in their own muscles, skin, joints, intestines, heart, lungs, or other parts of the body that can improve with treatment. The patient global disease activity score was recorded on a 10-cm VAS that contained a smiley face at the 0-cm anchor and a sad face at the 10 cm anchor to help participants understand the scale. Scores ranged from 0 (no evidence of disease activity) to 10 (extremely active or severe disease activity). Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    -1.43 ( 0.432 )
    -1.12 ( 0.699 )
    No statistical analyses for this end point

    Secondary: Change from Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26

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    End point title
    		 Change from Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26
    End point description
    The HAQ is a brief self-report questionnaire that assesses physical function pertaining to activities of daily living in a variety of domains. The HAQ includes 20 questions relating to 8 domains of function: dressing and grooming, arising, eating, walking, hygiene, reach, grip and usual activities. For each of the categories, participants reported the amount of difficulty they had in performing 2 or 3 specific subcategory items. The standard disability score is calculated from the 8 categories by dividing the sum of the individual categories by the number of categories answered, yielding a score from 0 (without any difficulty) to 3 (unable to do), with higher values indicating higher disability. Randomized Set, which included all randomized participants grouped by randomized treatment group. Number of participants analyzed = participants with evaluable data for the outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    25
    12
    Units: scores on a scale
        least squares mean (standard error)
    -0.1289 ( 0.08607 )
    -0.4188 ( 0.13676 )
    No statistical analyses for this end point

    Secondary: Number of Participants with CDASI Response (>=7-point Improvement) at Week 26

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    End point title
    		 Number of Participants with CDASI Response (>=7-point Improvement) at Week 26
    End point description
    The CDASI is an instrument that separately measures activity and damage in the skin of dermatomyositis (DM) participants. It is a 1-page instrument that contains 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis). CDASI score is calculated by rating the severity of skin disease in 15 anatomical locations on the body based on the activity and damage components. CDASI was completed by the Clinician or Clinician-Investigator while examining the participant. Total CDASI scores ranged from 0-100, with higher scores indicating a greater disease severity. Data are presented for the number of participants with a CDASI response. Response was defined as a >=7 point improvement in participants who did not have an intercurrent event at or prior to the relevant timepoint. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    6
    4
    No statistical analyses for this end point

    Secondary: Number of Participants with Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26

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    End point title
    		 Number of Participants with Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26
    End point description
    CDA-IGA is a scale that was created to measure disease severity in participants with skin disease. It is a 5-point scale (0 = clear; 1 = almost clear; 2 = mild; 3 = moderate; 4 = severe) with morphologic descriptors for each score. The CDA-IGA was completed by the Investigator and was used to describe the overall appearance of lesions at a given time point. Data are presented for the number of participants with a CDA-IGA response at Week 26. A response was defined as participants with clear or almost clear skin (score of 0 or 1) who did not have an intercurrent event at or before the relevant timepoint. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    5
    2
    No statistical analyses for this end point

    Secondary: Number of Participants with ≥ 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26

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    End point title
    		 Number of Participants with ≥ 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26
    End point description
    TIS20 was defined as a ≥20-point improvement response on IMACS-TIS. IMACS-TIS is a clinical instrument that encompasses 6 CSMs (physician, patient, extra-muscular global activity, muscle strength, HAQ, and muscle enzyme levels). A Total Improvement Score (TIS: 0–100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. Higher scores indicated greater improvement/response. TIS20 is considered a mild improvement score. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    14
    9
    No statistical analyses for this end point

    Secondary: Number of Participants with ≥ 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26

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    End point title
    		 Number of Participants with ≥ 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26
    End point description
    TIS60 was defined as a ≥60-point improvement response on IMACS-TIS. IMACS-TIS is a clinical instrument that encompasses 6 CSMs (physician, patient, extra-muscular global activity, muscle strength, HAQ, and muscle enzyme levels). A Total Improvement Score (TIS: 0–100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. Higher scores indicated greater improvement/response. TIS60 is considered a severe improvement score. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    3
    2
    No statistical analyses for this end point

    Secondary: Time to First Response of TIS20, TIS40, or TIS60

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    End point title
    		 Time to First Response of TIS20, TIS40, or TIS60
    End point description
    TIS20, 40 and 60 were defined as a ≥20,≥40 and≥60-point improvement response on IMACS-TIS respectively. TIS20, 40 and 60 were considered mild, moderate and severe improvement scores respectively. The median time to TIS20, TIS40, and TIS60 was defined at the time in which 50% of the participants experienced TIS20, TIS40, or TIS60, respectively, based on a Kaplan-Meier analysis. 99999=As fewer than 50% of the participants experienced the event of TIS40 and the median time to TIS40 event was close to the maximum follow-up period, there was not enough information on longer follow-up times to estimate the upper bound of the confidence interval. 9999= <50% of the participants experienced TIS60 response during the RCP (the Week 26 period), the median time could not be estimated along with the associated 80% confidence interval(s) using Kaplan-Meier analysis.
    End point type
    Secondary
    End point timeframe
    Baseline through Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: weeks
    median (confidence interval 80%)
        Time to TIS20 (n=17, n=10)
    10.43 (10.14 to 17.86)
    10.14 (2.14 to 10.14)
        Time to TIS40 (n=10, n=6)
    25.86 (18.14 to 99999)
    26.0 (10.14 to 26.29)
        Time to TIS60 (n=3, n=2)
    9999 (9999 to 9999)
    9999 (26.14 to 9999)
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical Worsening (CW) During the RCP At 2 Consecutive Visits

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    End point title
    		 Number of Participants with Clinical Worsening (CW) During the RCP At 2 Consecutive Visits
    End point description
    CW was defined as one of the following: a. Physician’s global activity VAS worsening ≥ 2 cm and MMT-8 worsening ≥ 20% compared to baseline b. Global extra muscular activity worsening ≥ 2 cm on the MDAAT VAS compared to baseline c. Any 3 of 5 CSMs (excluding muscle enzymes) worsening by ≥ 30% compared to baseline Data are presented for the number of participants with clinical worsening during the RCP at 2 consecutive visits. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline through Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    2
    0
    No statistical analyses for this end point

    Secondary: Number of Participants who Received Acute Rescue Therapy with Standard DM Treatment

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    End point title
    		 Number of Participants who Received Acute Rescue Therapy with Standard DM Treatment
    End point description
    Acute rescue therapy with standard DM treatment included an increased dose of a medication that was being taken for DM or the initiation of a new DM treatment (glucocorticoid and/or immunosuppressive/immunomodulatory therapy [ISTs]). Data are presented for the number of participants who received acute rescue therapy with standard DM treatment. Randomized Set, which included all randomized participants grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline through Week 26
    End point values
    		 RCP: Ravulizumab RCP: Placebo
    Number of subjects analysed
    26
    12
    Units: participants
    2
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to approximately 130 weeks
    Adverse event reporting additional description
    Safety Set for RCP = participants who received ≥1 dose of treatment. Participants were analyzed according to treatment received. One participant who was randomized to ravulizumab during RCP received placebo and was analyzed in placebo group. OLE Set for the OLE period= participants who received at least 1 dose of ravulizumab from Week 26
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    RCP: Ravulizumab
    Reporting group description
    		 Participants received a loading dose of ravulizumab on Day 1 followed by a maintenance dose at Week 2 and then ravulizumab once every 8 weeks (Q8W) during the 26-week RCP.

    Reporting group title
    OLE: Ravulizumab to Ravulizumab
    Reporting group description
    		 Participants who received ravulizumab during the RCP continued to receive ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.

    Reporting group title
    OLE: Placebo to Ravulizumab
    Reporting group description
    		 Participants who received placebo during the RCP received ravulizumab during the 130-week OLE. Participants received a blinded ravulizumab dose at Week 26, a maintenance dose at Week 28, then ravulizumab Q8W for the remainder of the OLE period.

    Reporting group title
    RCP: Placebo
    Reporting group description
    		 Participants received placebo on Day 1, and Weeks 2, 10, and 18 during the 26-week RCP.

    Serious adverse events
    		 RCP: Ravulizumab OLE: Ravulizumab to Ravulizumab OLE: Placebo to Ravulizumab RCP: Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 25 (8.00%)
    4 / 22 (18.18%)
    4 / 9 (44.44%)
    0 / 13 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Hypovolaemic shock
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Coronary artery stenosis
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac failure
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Syncope
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Agranulocytosis
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 22 (4.55%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Anaemia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Impaired healing
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 22 (4.55%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Intestinal haemorrhage
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Endometrial hyperplasia
    Additional description: Number at risk has been adjusted as this is a sex-specific event.
         subjects affected / exposed [1]
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Cutaneous calcification
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 22 (4.55%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 22 (4.55%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Escherichia urinary tract infection
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed to this adverse event. These numbers are expected to be equal.
    Justification: Number at risk has been adjusted as this is a sex-specific event.
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 RCP: Ravulizumab OLE: Ravulizumab to Ravulizumab OLE: Placebo to Ravulizumab RCP: Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 25 (36.00%)
    11 / 22 (50.00%)
    7 / 9 (77.78%)
    10 / 13 (76.92%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Vaccination site swelling
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    3
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary mass
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cough
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vocal cord leukoplakia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Injury, poisoning and procedural complications
    Infusion related reaction
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    2
    0
    1
    Contusion
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Compression fracture
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Nervous system disorders
    Neuralgia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    0
    0
    2
    Taste disorder
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    0
    0
    2
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Eye disorders
    Diabetic retinopathy
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 25 (16.00%)
    0 / 22 (0.00%)
    2 / 9 (22.22%)
    0 / 13 (0.00%)
         occurrences all number
    5
    0
    2
    0
    Constipation
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Anal erosion
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tongue movement disturbance
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Large intestine polyp
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypoaesthesia oral
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Diverticulum intestinal
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Enterocolitis
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Skin and subcutaneous tissue disorders
    Skin lesion
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Pruritus
         subjects affected / exposed
    3 / 25 (12.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    3
    0
    0
    1
    Eczema
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Diffuse alopecia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Dermatomyositis
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Rotator cuff syndrome
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Osteoporosis
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Muscle spasms
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    0 / 9 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Back pain
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Arthralgia
         subjects affected / exposed
    2 / 25 (8.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    1 / 13 (7.69%)
         occurrences all number
    2
    0
    1
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    2
    1
    0
    COVID-19
         subjects affected / exposed
    2 / 25 (8.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    3 / 13 (23.08%)
         occurrences all number
    2
    2
    0
    3
    Pyoderma
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Influenza
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Genital infection fungal
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sepsis
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 25 (0.00%)
    0 / 22 (0.00%)
    1 / 9 (11.11%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    2
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 25 (0.00%)
    2 / 22 (9.09%)
    0 / 9 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    2
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Aug 2021
    • Provided information on the Independent Data Monitoring Committee (IDMC) role • Clarified Part A interim analysis • Clarified individual and study closure criteria
    30 Aug 2022
    • Broadened the target population • Reduced the risk of screen failures • Improved participant experience • Facilitated recruitment of participants
    23 Jun 2023
    • Extended RCP in Part B from 26 weeks to 50 weeks • Extended OLE Period in Part A from 74 weeks to 130 weeks • Implemented requirements for conducting a clinical study under European Union Clinical Trials Regulation (EU CTR)

    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.
    Part A did not meet its primary endpoint and the study was terminated.
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