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    Clinical Trial Results:
    A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

    Summary
    EudraCT number
    		 2020-001104-41
    Trial protocol
    		 GB  
    Global end of trial date
    07 Jun 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Aug 2023
    First version publication date
    23 Aug 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1840-WD-204
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04573309
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    100 College Street, New Haven, CT, United States, 06510
    Public contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 7 87148158, clinicaltrials.eu@alexion.com
    Scientific contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 7 87148158, 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
    07 Jun 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to assess net copper balance with daily repeat-dose ALXN1840 treatment (15 mg and 30 mg) in participants with Wilson disease.
    Protection of trial subjects
    This trial was conducted in compliance with Good Clinical Practice.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Sep 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 Kingdom: 6
    Country: Number of subjects enrolled
    New Zealand: 3
    Worldwide total number of subjects
    9
    EEA total number of subjects
    0
    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)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study started on 07 Sep 2020 and completed on 07 Jun 2022.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1
    Arm description
    		 Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
    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
    Participants received ALXN1840 at prespecified time points.

    Arm title
    		 Cohort 2
    Arm description
    		 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
    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
    Participants received ALXN1840 at prespecified time points.

    Number of subjects in period 1
    		Cohort 1 Cohort 2
    Started
    8
    1
    Received at least 1 dose of study drug
    8
    1
    Completed
    7
    1
    Not completed
    1
    0
         Protocol deviation
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Reporting group values
    		 Cohort 1 Cohort 2 Total
    Number of subjects
    		 8 1 9
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 8 1 9
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    Units: years
        arithmetic mean (standard deviation)
    		 35.1 ± 12.41 26.0 ± 99999 -
    Sex: Female, Male
    Units: participants
        Female
    		 1 1 2
        Male
    		 7 0 7

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Primary: Mean Daily Copper Balance: Day 1 through Day 8

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    End point title
    		 Mean Daily Copper Balance: Day 1 through Day 8 [1]
    End point description
    Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Primary
    End point timeframe
    Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
    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: Only Descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams/day
        arithmetic mean (standard deviation)
    0.8025 ± 0.26860
    0.3062 ± 99999
    No statistical analyses for this end point

    Primary: Mean Daily Copper Balance: Day 31 through Day 35

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    End point title
    		 Mean Daily Copper Balance: Day 31 through Day 35 [2]
    End point description
    Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Primary
    End point timeframe
    Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only Descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    8
    1
    Units: milligrams/day
        arithmetic mean (standard deviation)
    0.9156 ± 0.37982
    0.6613 ± 99999
    No statistical analyses for this end point

    Primary: Mean Daily Copper Balance: Day 36 through Day 39

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    End point title
    		 Mean Daily Copper Balance: Day 36 through Day 39 [3]
    End point description
    Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Primary
    End point timeframe
    Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
    Notes
    [3] - 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: Only Descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    8
    1
    Units: milligrams/day
        arithmetic mean (standard deviation)
    0.9975 ± 0.26543
    0.5662 ± 99999
    No statistical analyses for this end point

    Primary: Mean Daily Copper Balance: Day 25 through Day 28

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    End point title
    		 Mean Daily Copper Balance: Day 25 through Day 28 [4]
    End point description
    Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Primary
    End point timeframe
    Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
    Notes
    [4] - 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: Only Descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    8
    1
    Units: milligrams/day
        arithmetic mean (standard deviation)
    0.7109 ± 0.67915
    0.7072 ± 99999
    No statistical analyses for this end point

    Secondary: Change From Baseline In Mean Daily Copper Balance

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    End point title
    		 Change From Baseline In Mean Daily Copper Balance
    End point description
    Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams/day
    arithmetic mean (standard deviation)
        Day 1 through Day 8
    -0.3780 ± 0.14822
    -0.2060 ± 99999
        Day 25 through Day 28
    -0.4697 ± 0.55770
    0.1950 ± 99999
        Day 31 through Day 35
    -0.2650 ± 0.47135
    0.1492 ± 99999
        Day 36 through Day 39
    -0.1831 ± 0.44589
    0.0540 ± 99999
    No statistical analyses for this end point

    Secondary: Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)

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    End point title
    		 Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
    End point description
    Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams
    arithmetic mean (standard deviation)
        Copper Food Content: Day 1 through Day 8
    1.6423 ± 0.15638
    1.3523 ± 99999
        Copper Food Content: Day 25 through Day 28
    1.8269 ± 0.42485
    1.5637 ± 99999
        Copper Food Content: Day 31 through Day 35
    1.8938 ± 0.21846
    1.6022 ± 99999
        Copper Food Content: Day 36 through Day 39
    1.7850 ± 0.20107
    1.4397 ± 99999
        Copper Drink Content: Day 1 through Day 8
    0.0061 ± 0.00159
    0.0055 ± 99999
        Copper Drink Content: Day 25 through Day 28
    0.0066 ± 0.00180
    0.0080 ± 99999
        Copper Drink Content: Day 31 through Day 35
    0.0071 ± 0.00184
    0.0044 ± 99999
        Copper Drink Content: Day 36 through Day 39
    0.0118 ± 0.00920
    0.0045 ± 99999
        Copper Feces Content: Day 1 through Day 8
    0.7594 ± 0.21993
    0.7718 ± 99999
        Copper Feces Content: Day 25 through Day 28
    0.9373 ± 0.30688
    0.7238 ± 99999
        Copper Feces Content: Day 31 through Day 35
    0.8270 ± 0.33746
    0.8720 ± 99999
        Copper Feces Content: Day 36 through Day 39
    0.6136 ± 0.27213
    0.7787 ± 99999
        Copper Urine Content: Day 1 through Day 8
    0.0865 ± 0.05689
    0.2798 ± 99999
        Copper Urine Content: Day 25 through Day 28
    0.1852 ± 0.09079
    0.1408 ± 99999
        Copper Urine Content: Day 31 through Day 35
    0.1584 ± 0.07823
    0.0732 ± 99999
        Copper Urine Content: Day 36 through Day 39
    0.1857 ± 0.09506
    0.0994 ± 99999
    No statistical analyses for this end point

    Secondary: Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State

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    End point title
    		 Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
    End point description
    Molybdenum balance is defined as the difference between the measured molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine), and was calculated as the average daily molybdenum balance over the collection period. Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams
    arithmetic mean (standard deviation)
        Molybdenum Food Content : Days 25 through 28
    0.3324 ± 0.18039
    0.3905 ± 99999
        Molybdenum Food Content : Days 36 through 39
    0.1923 ± 0.02354
    0.1724 ± 99999
        Molybdenum Drink Content : Days 25 through 28
    0.0006 ± 0.00012
    0.0018 ± 99999
        Molybdenum Drink Content : Days 36 through 39
    0.0007 ± 0.00024
    0.0004 ± 99999
        Molybdenum Feces Content : Days 25 through 28
    1.1004 ± 0.38506
    1.0656 ± 99999
        Molybdenum Feces Content : Days 36 through 39
    1.0541 ± 0.83207
    2.1352 ± 99999
        Molybdenum Urine Content : Days 25 through 28
    0.7250 ± 0.32736
    0.5480 ± 99999
        Molybdenum Urine Content : Days 36 through 39
    0.8984 ± 0.62633
    1.0395 ± 99999
    No statistical analyses for this end point

    Secondary: Change From Baseline In Total Molybdenum Excretion In Urine And Feces

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    End point title
    		 Change From Baseline In Total Molybdenum Excretion In Urine And Feces
    End point description
    Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams
    arithmetic mean (standard deviation)
        Molybdenum Feces Content : Day 1 through 8
    0.7102 ± 0.22072
    0.9510 ± 99999
        Molybdenum Feces Content : Days 25 through 28
    1.0146 ± 0.42658
    0.9736 ± 99999
        Molybdenum Feces Content : Days 31 through 35
    1.0484 ± 0.62291
    1.7188 ± 99999
        Molybdenum Feces Content : Days 36 through 39
    0.9683 ± 0.88027
    2.0432 ± 99999
        Molybdenum Urine Content : Day 1 through 8
    0.3972 ± 0.15972
    0.1725 ± 99999
        Molybdenum Urine Content : Days 25 through 28
    0.6122 ± 0.28154
    0.4010 ± 99999
        Molybdenum Urine Content : Days 31 through 35
    0.7974 ± 0.47607
    0.8085 ± 99999
        Molybdenum Urine Content : Days 36 through 39
    0.7855 ± 0.58820
    0.8926 ± 99999
    No statistical analyses for this end point

    Secondary: Mean Daily Molybdenum Balance At ALXN1840 Steady State

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    End point title
    		 Mean Daily Molybdenum Balance At ALXN1840 Steady State
    End point description
    Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in). Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams
    arithmetic mean (standard deviation)
        Days 25 through 28
    1.3618 ± 0.44254
    2.1087 ± 99999
        Days 36 through 39
    2.7599 ± 1.68008
    3.6581 ± 99999
    No statistical analyses for this end point

    Secondary: Accumulation Of Molybdenum As Determined By Molybdenum Balance

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    End point title
    		 Accumulation Of Molybdenum As Determined By Molybdenum Balance
    End point description
    Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. 99999 signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: milligrams
    arithmetic mean (standard deviation)
        Days 1 through 8
    2.2458 ± 0.23232
    2.0876 ± 99999
        Days 31 through 35
    2.6265 ± 1.96365
    4.0775 ± 99999
    No statistical analyses for this end point

    Secondary: Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum

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    End point title
    		 Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
    End point description
    Pharmacokinetic (PK) analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles. 99999 signifies that Geometric Coefficient of Variation (CV) could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Day 39
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    7
    1
    Units: nanograms/milliliters
    geometric mean (geometric coefficient of variation)
        Total Molydenum
    430.451 ± 46.6
    1271.551 ± 99999
        Plasma ultrafiltrate molybdenum
    13.546 ± 43.5
    17.823 ± 99999
    No statistical analyses for this end point

    Secondary: Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum

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    End point title
    		 Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
    End point description
    Pharmacokinetic analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles. Here, Number of participants analyzed signifies those who were evaluable for this outcome measure and number analyzed signifies those who were evaluable at specified time points. 99999 signifies that signifies that geometric CV could not be calculated as there was only 1 evaluable participant.
    End point type
    Secondary
    End point timeframe
    Day 39
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    6
    1
    Units: hours*nanograms/milliliters
    geometric mean (geometric coefficient of variation)
        Total Molydenum (n=6,1)
    13532.591 ± 49.3
    65694.679 ± 99999
        Plasma ultrafiltrate molybdenum (n=5,1)
    292.737 ± 11.9
    476.960 ± 99999
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Day 56
    Adverse event reporting additional description
    Safety set included all participants who received at least 1 dose of ALXN1840.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 Participants who had received Wilson disease therapy for >28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Reporting group title
    Cohort 2
    Reporting group description
    		 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.

    Serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 8 (75.00%)
    1 / 1 (100.00%)
    Investigations
    Liver function test abnormal
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Transaminases increased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nail injury
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Vascular disorders
    Phlebitis superficial
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Headache
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    2
    Migraine
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 1 (100.00%)
         occurrences all number
    2
    1
    Abdominal distension
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Breast tenderness
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Dry skin
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Hordeolum
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Periodontitis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Pulpitis dental
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Aug 2020
    The main reason for preparation of this amendment was to update procedures outlined in the Schedule of Activities, remove contradictory text on the reporting of serious adverse events, and add details of an interim analysis.
    19 Mar 2021
    The main reason for preparation of this amendment was to revise the exclusion criterion for a urine drug screen. Changes implemented via Administrative Letter 1 and Administrative Letter 2, as well as COVID vaccination guidance, have also been incorporated.
    31 Aug 2021
    The main reason for preparation of this amendment was to update the washout period for zinc.

    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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    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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