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    Clinical Trial Results:
    A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 versus Standard of Care in Pediatric Participants with Wilson Disease

    Summary
    EudraCT number
    		 2021-001015-82
    Trial protocol
    		 DE   FR   ES   PL  
    Global end of trial date
    26 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Jan 2024
    First version publication date
    07 Jan 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    ALXN1840-WD-302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05047523
    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., +33 147100606, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 147100606, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002232-PIP02-19
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Oct 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Jun 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Jun 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of this study was to evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to standard of care (SoC), on copper control in participants with Wilson disease (WD) aged 3 to < 18 years of age at the time of enrollment.
    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
    13 Sep 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
    Spain: 13
    Country: Number of subjects enrolled
    Poland: 6
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Japan: 7
    Country: Number of subjects enrolled
    Korea, Republic of: 5
    Country: Number of subjects enrolled
    Australia: 4
    Worldwide total number of subjects
    40
    EEA total number of subjects
    24
    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)
    18
    Adolescents (12-17 years)
    22
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 The study consisted of 2 periods: Primary Evaluation Period (PEP) and Open-label Extension (OLE) Period. Participants who completed the 48-week PEP were offered the opportunity to continue treatment in an up to 24-week OLE Period.

    Pre-assignment
    Screening details
    		 Participants were randomized, stratified by cohort, in 1:1 ratio to ALXN1840 or continued treatment with SoC in Cohort 1 or as continued or initial therapy in Cohort 2.

    Period 1
    Period 1 title
    Primary Evaluation Period (48 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Assessor [1]

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1: ALXN1840
    Arm description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN1840 was administered per schedule specified in the arm description.

    Arm title
    		 Cohort 1: SoC Therapy
    Arm description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    SoC Therapy
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet, Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    SoC therapy was administered per schedule specified in the arm description.

    Arm title
    		 Cohort 2: ALXN1840
    Arm description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN1840 was administered per schedule specified in the arm description.

    Arm title
    		 Cohort 2: SoC Therapy
    Arm description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    SoC Therapy
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet, Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    SoC therapy was administered per schedule specified in the arm description.

    Notes
    [1] - The roles blinded appear inconsistent with a simple blinded trial.
    Justification: This Warning is appearing here due to the limitations of the database. The blinded roles are consistent with this type of study.”
    Number of subjects in period 1
    		Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Started
    15
    16
    4
    5
    Received at least 1 dose of study drug
    15
    16
    4
    5
    Completed
    12
    11
    0
    1
    Not completed
    3
    5
    4
    4
         Consent withdrawn by subject
    -
    1
    -
    -
         Physician decision
    -
    -
    1
    -
         Adverse event, non-fatal
    1
    -
    -
    -
         Study Terminated by Sponsor
    2
    4
    3
    4
    Period 2
    Period 2 title
    Extension Period (24 Weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 ALXN1840/ALXN1840
    Arm description
    		 Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN1840 was administered per schedule specified in the arm description.

    Arm title
    		 SoC Therapy/ALXN1840
    Arm description
    		 Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN1840 was administered per schedule specified in the arm description.

    Number of subjects in period 2
    		ALXN1840/ALXN1840 SoC Therapy/ALXN1840
    Started
    12
    12
    Received at least 1 dose of study drug
    12
    12
    Completed
    3
    3
    Not completed
    9
    9
         Adverse event, non-fatal
    1
    1
         Study Terminated by Sponsor
    8
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1: ALXN1840
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 1: SoC Therapy
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: ALXN1840
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: SoC Therapy
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.

    Reporting group values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy Total
    Number of subjects
    		 15 16 4 5 40
    Age Categorical
    Units: Subjects
        Children (2-11 years)
    		 6 6 3 3 18
        Adolescents (12-17 years)
    		 9 10 1 2 22
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 11.6 ( 4.10 ) 12.8 ( 2.79 ) 9.0 ( 5.42 ) 10.0 ( 4.36 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 5 4 3 3 15
        Male
    		 10 12 1 2 25
    Race
    Units: Subjects
        Asian
    		 5 6 1 3 15
        White
    		 9 9 0 2 20
        Other
    		 1 0 2 0 3
        Not Reported
    		 0 1 1 0 2
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 5 4 0 0 9
        Not Hispanic or Latino
    		 10 11 4 5 30
        Not Reported
    		 0 1 0 0 1

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1: ALXN1840
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 1: SoC Therapy
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: ALXN1840
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: SoC Therapy
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
    Reporting group title
    ALXN1840/ALXN1840
    Reporting group description
    		 Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.

    Reporting group title
    SoC Therapy/ALXN1840
    Reporting group description
    		 Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.

    Primary: Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper (NCC) in Plasma

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    End point title
    		 Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper (NCC) in Plasma [1]
    End point description
    Due to early termination of study, this endpoint was not analyzed.
    End point type
    Primary
    End point timeframe
    Baseline, Week 48
    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: Statistical analysis was planned for the primary and all secondary endpoints however, the team decided not to perform any efficacy analysis as the study was terminated early.
    End point values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Number of subjects analysed
    0 [2]
    0 [3]
    0 [4]
    0 [5]
    Units: percent change
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    Notes
    [2] - Due to early termination of study, this endpoint was not analyzed.
    [3] - Due to early termination of study, this endpoint was not analyzed.
    [4] - Due to early termination of study, this endpoint was not analyzed.
    [5] - Due to early termination of study, this endpoint was not analyzed.
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period

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    End point title
    		 Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
    End point description
    An adverse event (AE) was any untoward medical occurrence in a participant administered the study drug and which did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs with onset after the first dose of study intervention or existing events that worsened in severity after the first dose of study intervention. 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 included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 48
    End point values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Number of subjects analysed
    15
    16
    4
    5
    Units: participants
    13
    13
    3
    4
    No statistical analyses for this end point

    Secondary: Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC

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    End point title
    		 Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
    End point description
    PD analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint. '99999' signifies 'data could not be calculated due to single participant'.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Number of subjects analysed
    11
    11
    0 [6]
    1
    Units: nanograms (ng)*hours (hr)/milliliter(mL)
    arithmetic mean (standard deviation)
        Total Copper
    6342.909 ( 3038.3317 )
    7528.864 ( 4167.2110 )
    ( )
    16369.000 ( 99999 )
        Direct NCC
    3882.473 ( 1716.4565 )
    3067.864 ( 1524.9183 )
    ( )
    4214.000 ( 99999 )
    Notes
    [6] - No participant was evaluable for this endpoint at specified timepoint.
    No statistical analyses for this end point

    Secondary: Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations

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    End point title
    		 Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
    End point description
    PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint. '99999' signifies 'data could not be calculated due to single participant'.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Number of subjects analysed
    12
    11
    0 [7]
    1
    Units: ng/milliliters (mL)
    arithmetic mean (standard deviation)
        Plasma Total Molybdenum
    183.150 ( 110.4736 )
    209.355 ( 114.3741 )
    ( )
    307.000 ( 99999 )
        Plasma Ultrafiltrate Molybdenum
    13.733 ( 8.6186 )
    17.708 ( 27.9548 )
    ( )
    9.910 ( 99999 )
    Notes
    [7] - No participant was evaluable for this endpoint at specified timepoint.
    No statistical analyses for this end point

    Secondary: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum

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    End point title
    		 Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum
    End point description
    PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint. '99999' signifies 'data could not be calculated due to single participant'.
    End point type
    Secondary
    End point timeframe
    Week 48
    End point values
    		 Cohort 1: ALXN1840 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 2: SoC Therapy
    Number of subjects analysed
    11
    11
    0 [8]
    1
    Units: ng*hr/mL
    arithmetic mean (standard deviation)
        Plasma Total Molybdenum
    4498.082 ( 3054.8300 )
    2931.045 ( 1694.3512 )
    ( )
    4261.100 ( 99999 )
        Plasma Ultrafiltrate Molybdenum
    233.154 ( 145.7960 )
    144.627 ( 88.6710 )
    ( )
    129.370 ( 99999 )
    Notes
    [8] - No participant was evaluable for this endpoint at specified timepoint.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 76
    Adverse event reporting additional description
    Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Cohort 1: SoC Therapy
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: ALXN1840
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 1: ALXN1840
    Reporting group description
    		 Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for >28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.

    Reporting group title
    Cohort 2: SoC Therapy
    Reporting group description
    		 Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.

    Reporting group title
    SoC Therapy/ALXN1840
    Reporting group description
    		 Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.

    Reporting group title
    ALXN1840/ALXN1840
    Reporting group description
    		 Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.

    Serious adverse events
    		 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 1: ALXN1840 Cohort 2: SoC Therapy SoC Therapy/ALXN1840 ALXN1840/ALXN1840
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Gastrointestinal disorders
    Duodenal perforation
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Gastrointestinal infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort 1: SoC Therapy Cohort 2: ALXN1840 Cohort 1: ALXN1840 Cohort 2: SoC Therapy SoC Therapy/ALXN1840 ALXN1840/ALXN1840
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 16 (75.00%)
    2 / 4 (50.00%)
    13 / 15 (86.67%)
    3 / 5 (60.00%)
    8 / 12 (66.67%)
    6 / 12 (50.00%)
    Investigations
    Electrocardiogram abnormal
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Blood triglycerides increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    2
    0
    2
    0
    Blood cholesterol increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    3 / 15 (20.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    3
    0
    0
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 4 (25.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    2
    0
    0
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 4 (25.00%)
    6 / 15 (40.00%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    7
    0
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    2
    0
    3
    0
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    1
    0
    0
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    3
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    3 / 15 (20.00%)
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    5
    0
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Odynophagia
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Vomiting
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    1 / 5 (20.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    2
    1
    1
    2
    Dyspepsia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Aphthous ulcer
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Constipation
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    2
    1
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    3 / 15 (20.00%)
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    3
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    1
    Cough
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Rash
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 4 (25.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    2
    0
    0
    0
    Arthralgia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 4 (25.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    2
    0
    0
    0
    Back pain
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 16 (18.75%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    1 / 5 (20.00%)
    4 / 12 (33.33%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    2
    2
    5
    0
    Gastroenteritis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    2 / 15 (13.33%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    2
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 4 (25.00%)
    1 / 15 (6.67%)
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    2
    2
    2
    0
    0
    COVID-19
         subjects affected / exposed
    4 / 16 (25.00%)
    0 / 4 (0.00%)
    6 / 15 (40.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    6
    0
    0
    0
    Gastrointestinal infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Viral infection
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    1
    0
    0
    Respiratory tract infection
         subjects affected / exposed
    3 / 16 (18.75%)
    0 / 4 (0.00%)
    1 / 15 (6.67%)
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    1
    0
    0
    0
    Influenza
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    2
    1
    Metabolism and nutrition disorders
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 4 (0.00%)
    0 / 15 (0.00%)
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    2
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    08 Mar 2022
    The main reason for this amendment was to reduce the maximum daily dose of ALXN1840 from 60 milligrams (mg) for adolescent participants and 30 mg for pediatric participants to 15 mg for all participants.

    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.
    Due to early termination of study, the efficacy endpoints were not analyzed.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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