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    Clinical Trial Results:
    A Phase 2, Single-arm, Pathologist-blinded Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in Patients with Wilson Disease who are Treated with ALXN1840 for 48 Weeks Followed by an Extension Treatment Period with ALXN1840 for up to an Additional 48 Weeks

    Summary
    EudraCT number
    		 2019-003711-60
    Trial protocol
    		 DE   BE   DK   GB   FR   AT  
    Global end of trial date
    13 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 May 2024
    First version publication date
    26 May 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1840-WD-205
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04422431
    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
    13 Jun 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to evaluate change in liver copper concentration following treatment with ALXN1840 at Week 48 in participants with Wilson disease (WD).
    Protection of trial subjects
    This study was conducted in accordance with the protocol and with the following: • Consensus ethical principles derived from international guidelines including the Declaration of Helsinki and 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 • Applicable laws and regulations
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Dec 2020
    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
    United States: 9
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    New Zealand: 6
    Country: Number of subjects enrolled
    Russian Federation: 5
    Country: Number of subjects enrolled
    Singapore: 2
    Worldwide total number of subjects
    31
    EEA total number of subjects
    6
    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)
    28
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study included a screening period of up to 4 weeks.

    Period 1
    Period 1 title
    Treatment Period (48 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 ALXN1840
    Arm description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period. Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840. Participants who did not enter the Extension Period discontinued dosing at Week 48, and had a final study visit for safety follow-up at Week 52.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    WTX101 (former name)
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Dosage and administration details as described in the arm description.

    Number of subjects in period 1
    		ALXN1840
    Started
    31
    Received at least 1 dose of study drug
    31
    Completed
    26
    Not completed
    5
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    3
         Other than specified
    1
    Period 2
    Period 2 title
    Extension Period (48 Weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 ALXN1840
    Arm description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period. Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840. Participants who did not enter the Extension Period discontinued dosing at Week 48, and had a final study visit for safety follow-up at Week 52.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    WTX101 (former name)
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Dosage and administration details as described in the arm description.

    Number of subjects in period 2 [1]
    		ALXN1840
    Started
    25
    Received at least 1 dose of study drug
    25
    Completed
    21
    Not completed
    4
         Consent withdrawn by subject
    1
         Other than specified
    2
         Lost to follow-up
    1
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: 1 participant completed the Treatment Period, but did not enter the Extension Period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ALXN1840
    Reporting group description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period. Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840. Participants who did not enter the Extension Period discontinued dosing at Week 48, and had a final study visit for safety follow-up at Week 52.

    Reporting group values
    		 ALXN1840 Total
    Number of subjects
    		 31 31
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 28 28
        From 65-84 years
    		 3 3
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.8 ( 14.47 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 11 11
        Male
    		 20 20
    Race
    Units: Subjects
        American Indian/Alaska Native
    		 0 0
        Asian
    		 6 6
        Black or African American
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        White
    		 21 21
        Other
    		 3 3
        Unknown
    		 1 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 2 2
        Not Hispanic or Latino
    		 25 25
        Not Reported
    		 3 3
        Unknown
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    ALXN1840
    Reporting group description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period. Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840. Participants who did not enter the Extension Period discontinued dosing at Week 48, and had a final study visit for safety follow-up at Week 52.
    Reporting group title
    ALXN1840
    Reporting group description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period. Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840. Participants who did not enter the Extension Period discontinued dosing at Week 48, and had a final study visit for safety follow-up at Week 52.

    Primary: Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) [1]
    End point description
    Liver biopsy samples were taken for the assessment of liver Cu concentration. Multiple imputation was used to impute missing data at Week 48 due to any reason based on Baseline values. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable.
    End point type
    Primary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Due to limitations of the EudraCT system, statistical analysis could not be provided for a single reporting group.
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: μg/g
        arithmetic mean (standard error)
    92.8 ( 56.34 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period)

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    End point title
    		 Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period)
    End point description
    Fibrosis from histology was evaluated by NASH CRN Fibrosis Stage, which was scaled from 0 to 4 stages where Score 0: None; Score 1: Perisinusoidal or periportal - 1a - mild, zone 3, perisinusoidal; Score 1: Perisinusoidal or periportal - 1b - moderate, zone 3, perisinusoidal; Score 1: Perisinusoidal or periportal - 1c - portal/periportal; Score 2: Both perisinusoidal and portal/periportal; Score 3: Bridging fibrosis; and Score 4: Cirrhosis. Higher scores indicated greater fibrosis. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: participants
        Baseline Score 0
    7
        Baseline Score 1a
    1
        Baseline Score 1b
    0
        Baseline Score 1c
    7
        Baseline Score 2
    3
        Baseline Score 3
    8
        Baseline Score 4
    2
        Baseline Not Evaluable
    1
        Week 48 Score 0
    5
        Week 48 Score 1a
    0
        Week 48 Score 1b
    0
        Week 48 Score 1c
    6
        Week 48 Score 2
    6
        Week 48 Score 3
    7
        Week 48 Score 4
    0
        Week 48 Not Evaluable
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period)

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    End point title
    		 Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period)
    End point description
    Fibrosis from histology was evaluated by Metavir Fibrosis Score, which was ranged from 0 to 4 where Score 0: No fibrosis; Score 1: Stellate enlargement of portal tract but without septa formation; Score 2: Enlargement of portal tract with rare septa formation; Score 3: Numerous septa without cirrhosis; and Score 4: Cirrhosis. Higher scores indicated greater fibrosis. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: participants
        Baseline Score 0
    8
        Baseline Score 1
    7
        Baseline Score 2
    4
        Baseline Score 3
    7
        Baseline Score 4
    2
        Baseline Not Evaluable
    1
        Week 48 Score 0
    5
        Week 48 Score 1
    4
        Week 48 Score 2
    8
        Week 48 Score 3
    7
        Week 48 Score 4
    0
        Week 48 Not Evaluable
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period)

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    End point title
    		 Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period)
    End point description
    Fibrosis from histology was evaluated by Ishak Fibrosis Score, which was ranged from 0 to 6 where Score 0: No fibrosis; Score 1: Fibrous expansion of some portal areas, with or without short fibrous septa; Score 2: Fibrous expansion of most portal areas, with or without short fibrous septa; Score 3: Fibrous expansion of most portal areas with occasional portal to portal (P-P) bridging; and Score 4: Fibrous expansion of portal areas with marked bridging (P-P) as well as portal central (P-C); Score 5: Marked bridging (P-P and/or P-C) with occasional nodules (incomplete cirrhosis); and Score 6: Cirrhosis, probable or definite. Higher scores indicated greater fibrosis. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: participants
        Baseline Score 0
    8
        Baseline Score 1
    6
        Baseline Score 2
    2
        Baseline Score 3
    9
        Baseline Score 4
    1
        Baseline Score 5
    1
        Baseline Score 6
    1
        Baseline Not Evaluable
    1
        Week 48 Score 0
    5
        Week 48 Score 1
    4
        Week 48 Score 2
    4
        Week 48 Score 3
    8
        Week 48 Score 4
    3
        Week 48 Score 5
    0
        Week 48 Score 6
    0
        Week 48 Not Evaluable
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hepatic Collagen Content at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in Hepatic Collagen Content at Week 48 (Treatment Period)
    End point description
    Fibrosis from histology was evaluated by morphometric quantification of hepatic collagen content. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    24
    Units: percentage of collagen
        arithmetic mean (standard deviation)
    8.5445 ( 15.54224 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in a-SMA Content at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in a-SMA Content at Week 48 (Treatment Period)
    End point description
    Fibrosis from histology was evaluated by morphometric quantification of hepatic a-SMA content. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    24
    Units: percentage of a-SMA
        arithmetic mean (standard deviation)
    1.6002 ( 5.47274 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Change From Baseline in NAS Steatosis Grading Score at Week 48 (Treatment Period)

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    End point title
    		 Number of Participants With Change From Baseline in NAS Steatosis Grading Score at Week 48 (Treatment Period)
    End point description
    Steatosis from histology was evaluated by the steatosis component of the NAS, which was ranged from 0 to 3 where Score 0: < 5% (minimal); Score 1: 5 - 33% (mild); Score 2: 34 - 66% (moderate); and Score 3: > 66% (severe). Higher scores indicated greater steatosis. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: participants
        Baseline Score 0
    19
        Baseline Score 1
    6
        Baseline Score 2
    3
        Baseline Score 3
    1
        Week 48 Score 0
    15
        Week 48 Score 1
    6
        Week 48 Score 2
    2
        Week 48 Score 3
    1
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hepatic Fat Content at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in Hepatic Fat Content at Week 48 (Treatment Period)
    End point description
    Steatosis from histology was evaluated by morphometric quantification of hepatic fat content. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    24
    Units: percentage of fat
        arithmetic mean (standard deviation)
    -0.2504 ( 2.19263 )
    No statistical analyses for this end point

    Secondary: Extension Period: Number of Participants With TEAEs

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    End point title
    		 Extension Period: Number of Participants With TEAEs
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The TEAEs were AEs with onset on or after the first study drug dose. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the ‘Reported Adverse Events’ Section. Safety Analysis Set in the Extension Period included all participants who received at least 1 dose of ALXN1840 in the Extension Period.
    End point type
    Secondary
    End point timeframe
    Day 1 (Extension Period) up Week 52 (Extension Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    25
    Units: participants
    24
    No statistical analyses for this end point

    Secondary: Treatment Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)

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    End point title
    		 Treatment Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The TEAEs were AEs with onset on or after the first study drug dose. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the ‘Reported Adverse Events’ Section. Safety Analysis Set in the Treatment Period included all participants who received at least 1 dose of treatment in the Treatment Period.
    End point type
    Secondary
    End point timeframe
    Day 1 (Treatment Period) up to Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    31
    Units: participants
    30
    No statistical analyses for this end point

    Secondary: Change From Baseline in NAS Total Score at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in NAS Total Score at Week 48 (Treatment Period)
    End point description
    Inflammation was quantified by the NAS total score. The score is defined as the unweighted sum of the scores for steatosis (0 [minimal] to 3 [severe]), lobular inflammation (0 [none] to 3 [>4 foci / 200x field]), and hepatocellular ballooning (0 [none] to 2 [many]), thus ranging from 0 (no inflammation) to 8 (severe inflammation), with higher scores indicating more severe disease. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    24
    Units: units on a scale
        arithmetic mean (standard deviation)
    0.1 ( 1.69 )
    No statistical analyses for this end point

    Secondary: Treatment Period: Predose Trough Plasma Total Mo Concentration

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    End point title
    		 Treatment Period: Predose Trough Plasma Total Mo Concentration
    End point description
    Pharmacokinetic (PK) Analysis Set in the Treatment Period included all participants who received at least 1 dose of treatment in the Treatment Period and had evaluable PK data for total Mo and/or plasma ultrafiltrate (PUF) Mo (as surrogate measure for ALXN1840 PK) in plasma in the Treatment Period. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Predose up to 4 hours postdose at Week 6 (Day 43) and Week 36 (Day 253)
    End point values
    		 ALXN1840
    Number of subjects analysed
    29
    Units: nanograms (ng)/milliliter (mL)
    arithmetic mean (standard deviation)
        Week 6
    174.39 ( 113.824 )
        Week 36
    131.19 ( 103.359 )
    No statistical analyses for this end point

    Secondary: Clinical Global Impression-Improvement (CGI-I) Scale Score at Week 48 (Treatment Period)

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    End point title
    		 Clinical Global Impression-Improvement (CGI-I) Scale Score at Week 48 (Treatment Period)
    End point description
    The CGI-I is a 7-point scale clinician assessment where 1= very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse. Higher scores indicated worsening of disease. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    25
    Units: units on a scale
        arithmetic mean (standard deviation)
    3.1 ( 1.26 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mo in Liver Biopsy Specimen at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in Mo in Liver Biopsy Specimen at Week 48 (Treatment Period)
    End point description
    The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    24
    Units: μg/g
        arithmetic mean (standard deviation)
    69.6976 ( 43.02655 )
    No statistical analyses for this end point

    Secondary: Treatment Period: Predose Trough Plasma Total PUF Mo Concentration

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    End point title
    		 Treatment Period: Predose Trough Plasma Total PUF Mo Concentration
    End point description
    PK Analysis Set in the Treatment Period included all participants who received at least 1 dose of treatment in the Treatment Period and had evaluable PK data for total Mo and/or PUF Mo (as surrogate measure for ALXN1840 PK) in plasma in the Treatment Period. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Predose up to 4 hours postdose at Week 6 (Day 43) and Week 36 (Day 253)
    End point values
    		 ALXN1840
    Number of subjects analysed
    28
    Units: ng/mL
    arithmetic mean (standard deviation)
        Week 6
    5.888 ( 2.0176 )
        Week 36
    7.843 ( 6.0564 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in CGI-S Scale Score at Week 48 (Treatment Period)

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    End point title
    		 Change From Baseline in CGI-S Scale Score at Week 48 (Treatment Period)
    End point description
    The CGI-S is a 7-point scale clinician assessment. Participants were assessed on severity of illness at the time of rating/assessment as follows: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill. Higher scores indicated worsening of disease. The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Treatment Period)
    End point values
    		 ALXN1840
    Number of subjects analysed
    25
    Units: units on a scale
        arithmetic mean (standard deviation)
    -0.4 ( 1.50 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 (Treatment Period) up to Week 52 (Extension Period) (up to a total of approximately 100 weeks)
    Adverse event reporting additional description
    Serious and other adverse events are reported in the Safety Analysis Set in the Treatment Period (all participants who received at least 1 dose of treatment in the Treatment Period), and for the Safety Analysis Set in the Extension Period, (all participants who received at least 1 dose of ALXN1840 in the Extension Period).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Treatment Period: ALXN1840
    Reporting group description
    		 Participants received ALXN1840 orally in the 48-week Treatment Period.

    Reporting group title
    Extension Period: ALXN1840
    Reporting group description
    		 Participants who completed the 48-week Treatment Period and agreed to enter a 48-week Extension Period, continued to receive ALXN1840.

    Serious adverse events
    		 Treatment Period: ALXN1840 Extension Period: ALXN1840
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 25 (8.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Humerus fracture
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Prostatitis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Drug-induced liver injury
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Bence Jones proteinuria
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ureteric obstruction
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Suicidal ideation
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Treatment Period: ALXN1840 Extension Period: ALXN1840
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    30 / 31 (96.77%)
    24 / 25 (96.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Seborrhoeic keratosis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Skin papilloma
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    5 / 31 (16.13%)
    3 / 25 (12.00%)
         occurrences all number
    6
    3
    Fatigue
         subjects affected / exposed
    4 / 31 (12.90%)
    2 / 25 (8.00%)
         occurrences all number
    4
    2
    Asthenia
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Chills
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Generalised oedema
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Feeling abnormal
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Pain
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Temperature intolerance
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Swelling
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    3
    Immunisation reaction
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Drug hypersensitivity
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    4
    Reproductive system and breast disorders
    Breast mass
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Breast tenderness
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Dysmenorrhoea
    Additional description: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.
         subjects affected / exposed [1]
    1 / 11 (9.09%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Haemorrhagic ovarian cyst
    Additional description: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.
         subjects affected / exposed [2]
    1 / 11 (9.09%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Heavy menstrual bleeding
    Additional description: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.
         subjects affected / exposed [3]
    1 / 11 (9.09%)
    2 / 9 (22.22%)
         occurrences all number
    1
    2
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Cough
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Dyspnoea
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Nasal congestion
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Respiratory disorder
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Nasal septum deviation
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Rhinitis allergic
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Sinus congestion
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Respiratory symptom
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Anxiety disorder
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    2
    Depressed mood
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Depression
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Insomnia
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Personality disorder
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Anxiety
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Suicidal ideation
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Mixed anxiety and depressive disorder
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Investigations
    Protein urine present
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    8 / 31 (25.81%)
    8 / 25 (32.00%)
         occurrences all number
    9
    9
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    8 / 31 (25.81%)
    7 / 25 (28.00%)
         occurrences all number
    8
    8
    Blood alkaline phosphatase increased
         subjects affected / exposed
    4 / 31 (12.90%)
    4 / 25 (16.00%)
         occurrences all number
    4
    4
    Hepatic enzyme increased
         subjects affected / exposed
    4 / 31 (12.90%)
    2 / 25 (8.00%)
         occurrences all number
    5
    2
    Aspartate aminotransferase increased
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 25 (8.00%)
         occurrences all number
    3
    2
    Blood creatine phosphokinase increased
         subjects affected / exposed
    3 / 31 (9.68%)
    4 / 25 (16.00%)
         occurrences all number
    3
    5
    Transaminases increased
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 25 (8.00%)
         occurrences all number
    3
    2
    Blood bilirubin increased
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    3
    1
    Liver function test increased
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Alanine aminotransferase abnormal
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Blood albumin decreased
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Blood folate decreased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Creatinine renal clearance decreased
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Creatinine renal clearance increased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Prothrombin time prolonged
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Urobilinogen urine increased
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Weight decreased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Weight increased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    White blood cell count decreased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Urine analysis abnormal
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Head injury
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Ligament sprain
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Post procedural fever
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Post procedural haematuria
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Post procedural hypotension
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Post procedural swelling
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Post vaccination syndrome
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Procedural pain
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Road traffic accident
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Upper limb fracture
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Incision site pain
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Neck injury
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Procedural complication
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Rib fracture
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Limb injury
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    7 / 31 (22.58%)
    6 / 25 (24.00%)
         occurrences all number
    9
    8
    Lethargy
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 25 (8.00%)
         occurrences all number
    3
    2
    Dizziness
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Presyncope
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Tremor
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    3
    1
    Paraesthesia
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Carpal tunnel syndrome
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Balance disorder
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Serotonin syndrome
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Migraine
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Leukopenia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Thrombocytopenia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Ear pain
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Vertigo
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Eye disorders
    Vitreous detachment
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Vision blurred
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Chalazion
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Myopia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    6 / 31 (19.35%)
    6 / 25 (24.00%)
         occurrences all number
    6
    7
    Abdominal pain
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Abdominal discomfort
         subjects affected / exposed
    2 / 31 (6.45%)
    3 / 25 (12.00%)
         occurrences all number
    2
    3
    Haemorrhoids thrombosed
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Mouth ulceration
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    2
    2
    Toothache
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Vomiting
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Haemorrhoids
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Flatulence
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Faeces discoloured
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Dyspepsia
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Dry mouth
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Oral pain
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Diarrhoea
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    2
    2
    Constipation
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    2
    1
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 25 (8.00%)
         occurrences all number
    2
    3
    Skin and subcutaneous tissue disorders
    Diabetic foot
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 25 (8.00%)
         occurrences all number
    3
    3
    Pruritus
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Idiopathic guttate hypomelanosis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Dermatitis contact
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Erythema
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Ingrown hair
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Glycosuria
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Proteinuria
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Haematuria
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 31 (9.68%)
    4 / 25 (16.00%)
         occurrences all number
    4
    5
    Back pain
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Greater trochanteric pain syndrome
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Joint effusion
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Muscle spasms
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Myalgia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Neck pain
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Intervertebral disc protrusion
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Back disorder
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Axillary mass
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Muscular weakness
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    5 / 31 (16.13%)
    8 / 25 (32.00%)
         occurrences all number
    5
    10
    COVID-19
         subjects affected / exposed
    4 / 31 (12.90%)
    10 / 25 (40.00%)
         occurrences all number
    4
    10
    Kidney infection
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Pneumonia
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    2
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 31 (6.45%)
    4 / 25 (16.00%)
         occurrences all number
    2
    7
    Urinary tract infection
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    4
    1
    Bacterial vaginosis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Conjunctivitis
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Dermatophytosis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    2
    0
    Folliculitis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Furuncle
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Gingivitis
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Helicobacter infection
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Paronychia
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Post procedural sepsis
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Sinusitis
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    2
    Respiratory tract infection viral
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Bacteriuria
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Rhinitis
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    3
    Metabolism and nutrition disorders
    Hypercholesterolaemia
         subjects affected / exposed
    3 / 31 (9.68%)
    3 / 25 (12.00%)
         occurrences all number
    3
    3
    Decreased appetite
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 25 (4.00%)
         occurrences all number
    2
    1
    Diabetes mellitus
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Dyslipidaemia
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Hyperglycaemia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Hypertriglyceridaemia
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Hyponatraemia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 25 (0.00%)
         occurrences all number
    1
    0
    Iron deficiency
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Hyperlipidaemia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Gout
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 25 (4.00%)
         occurrences all number
    0
    1
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.
    [3] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: The N at risk for this adverse event has been adjusted to the number of females in the respective study periods as it is a sex-specific event.

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 May 2020
    Addition of the requirement for Alexion approval prior to increasing the dose of ALXN1840. Administrative Change Letters since approval of the previous protocol were also incorporated.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

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