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    Clinical Trial Results:
    Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan.

    Summary
    EudraCT number
    		 2020-005832-31
    Trial protocol
    		 DK  
    Global end of trial date
    04 Jul 2022

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

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1840-HV-Cu-Absorption
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Aarhus University Hospital
    Sponsor organisation address
    Palle Juul-Jensens Boulevard 99, Aarhus N, Denmark,, Aarhus N, Denmark,
    Public contact
    Public information about the trial., Aarhus University Hospital, thomsand@rm.dk
    Scientific contact
    Public information about the trial., Aarhus University Hospital, thomsand@rm.dk
    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
    20 Nov 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Jul 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Jul 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the effect of ALXN1840 on hepatic copper uptake in healthy participants, compared to the effect of penicillamine, trientine and placebo.
    Protection of trial subjects
    Blood samples before and after treatment, including liver, kidney and hematological parameters. Only inclusion of healthy persons as evaluated by clinical physicians. Medical supervision during tracer injection and scans. Participants given contact information of medical personnel and instructed to note all adverse events.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Feb 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
    Denmark: 32
    Worldwide total number of subjects
    32
    EEA total number of subjects
    32
    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)
    29
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited through posts in local news papers and social media.

    Pre-assignment
    Screening details
    		 Interested persons were contacted by phone and informed about the trial, written participant information was given by mail. An in-person meeting was conducted for inclusion into the study and evaluation of health status.

    Period 1
    Period 1 title
    Pre-treatment
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    The tetrathiomolybdate and placebo groups were double blinded. The Trientine and Penicillamine groups were not blinded.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Trientine
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Cuprior
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    300 mg morning, 225 mg evening.

    Arm title
    		 Penicillamine
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Metalcaptase
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    600 mg morning and 600 mg evening

    Arm title
    		 Tetrathiomolybdate
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Tetrathiomolybdate
    Investigational medicinal product code
    Other name
    ALXN1840, bis-choline tetrathiomolybdate, WTX101
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg once daily

    Arm title
    		 Placebo
    Arm description
    		 Placebo which was visually identical to tetrathiomolybdate.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 tablet pr day. Visually identical to tetrathiomolybdate.

    Number of subjects in period 1
    		Trientine Penicillamine Tetrathiomolybdate Placebo
    Started
    8
    8
    8
    8
    Completed
    8
    8
    8
    8
    Period 2
    Period 2 title
    Post-treatment
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    The tetrathiomolybdate and placebo groups were double blinded. The Trientine and Penicillamine groups were not blinded.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Trientine
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Cuprior
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    300 mg morning, 225 mg evening.

    Arm title
    		 Penicillamine
    Arm description
    		 -
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Metalcaptase
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    600 mg morning and 600 mg evening

    Arm title
    		 Tetrathiomolybdate
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Tetrathiomolybdate
    Investigational medicinal product code
    Other name
    ALXN1840, bis-choline tetrathiomolybdate, WTX101
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg once daily

    Arm title
    		 Placebo
    Arm description
    		 Placebo which was visually identical to tetrathiomolybdate.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 tablet pr day. Visually identical to tetrathiomolybdate.

    Number of subjects in period 2
    		Trientine Penicillamine Tetrathiomolybdate Placebo
    Started
    8
    8
    8
    8
    Completed
    8
    8
    8
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Trientine
    Reporting group description
    		 -

    Reporting group title
    Penicillamine
    Reporting group description
    		 -

    Reporting group title
    Tetrathiomolybdate
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo which was visually identical to tetrathiomolybdate.

    Reporting group values
    		 Trientine Penicillamine Tetrathiomolybdate Placebo Total
    Number of subjects
    		 8 8 8 8 32
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0 0
        Adults (18-64 years)
    		 8 8 7 6 29
        From 65-84 years
    		 0 0 1 2 3
        85 years and over
    		 0 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 4 4 4 6 18
        Male
    		 4 4 4 2 14
    Alanine aminotransferase
    Units: U/L
        median (inter-quartile range (Q1-Q3))
    		 22.50 (18.00 to 28.00) 24.00 (19.50 to 32.00) 17.50 (13.00 to 26.00) 20.00 (17.50 to 29.00) -
    Bilirubin
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 11.00 (9.00 to 20.00) 13.00 (11.00 to 14.50) 8.50 (7.50 to 12.50) 11.00 (8.50 to 13.00) -
    Creatinine
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 64.00 (58.50 to 68.00) 74.50 (61.50 to 78.00) 72.00 (58.50 to 86.50) 70.50 (67.50 to 75.50) -
    Serum copper
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 17.50 (15.80 to 23.60) 16.05 (14.95 to 25.50) 16.20 (13.40 to 19.15) 14.90 (11.90 to 16.40) -

    End points

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    End points reporting groups
    Reporting group title
    Trientine
    Reporting group description
    		 -

    Reporting group title
    Penicillamine
    Reporting group description
    		 -

    Reporting group title
    Tetrathiomolybdate
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo which was visually identical to tetrathiomolybdate.
    Reporting group title
    Trientine
    Reporting group description
    		 -

    Reporting group title
    Penicillamine
    Reporting group description
    		 -

    Reporting group title
    Tetrathiomolybdate
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo which was visually identical to tetrathiomolybdate.

    Primary: Liver SUV

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    End point title
    		 Liver SUV
    End point description
    Two scans (1H and 15H) after tracer ingestion. Subjects acted as their own controls. A change in liver SUV was taken as a reduction in intestinal 64copper uptake.
    End point type
    Primary
    End point timeframe
    1-15H after tracer ingestion
    End point values
    		 Trientine Penicillamine Tetrathiomolybdate Placebo Trientine Penicillamine Tetrathiomolybdate Placebo
    Number of subjects analysed
    8
    8
    8
    8
    8
    8
    8
    8
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1h
    6.17 (3.82 to 8.54)
    8.78 (5.43 to 10.73)
    7.42 (5.56 to 8.19)
    6.31 (5.20 to 8.99)
    1.47 (1.17 to 4.13)
    6.85 (5.81 to 7.71)
    0.48 (0.15 to 0.94)
    6.12 (5.06 to 9.80)
        15h
    14.24 (13.02 to 16.11)
    16.30 (13.55 to 19.18)
    13.22 (11.57 to 16.46)
    13.62 (11.86 to 15.47)
    6.19 (5.24 to 8.67)
    12.17 (11.65 to 13.09)
    3.05 (1.18 to 5.69)
    15.91 (11.70 to 17.94)
    Statistical analysis title
    		 Tetrathiomolybdate
    Comparison groups
    Tetrathiomolybdate v Tetrathiomolybdate
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0117 [1]
    Method
    		 Sign test
    Confidence interval
    Notes
    [1] - p-value < 0.05 at both 1h and 15h timepoint.
    Statistical analysis title
    		 Placebo
    Comparison groups
    Placebo v Placebo
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 1 [2]
    Method
    		 Sign test
    Confidence interval
    Notes
    [2] - p-value > 0.05 at both 1h and 15h timepoint.
    Statistical analysis title
    		 Trientine
    Comparison groups
    Trientine v Trientine
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0117 [3]
    Method
    		 Sign test
    Confidence interval
    Notes
    [3] - p-value < 0.05 at both 1h and 15h timepoint.
    Statistical analysis title
    		 Penicillamine
    Comparison groups
    Penicillamine v Penicillamine
    Number of subjects included in analysis
    16
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1614 [4]
    Method
    		 Sign test
    Confidence interval
    Notes
    [4] - p-value > 0.05 at 1h timepoint. p-value < 0.05 (0.0357) at 15h timepoint.

    Secondary: Blood

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    End point title
    		 Blood
    End point description
    End point type
    Secondary
    End point timeframe
    1-15H after tracer ingestion.
    End point values
    		 Trientine Penicillamine Tetrathiomolybdate Placebo Trientine Penicillamine Tetrathiomolybdate Placebo
    Number of subjects analysed
    8
    8
    8
    8
    8
    8
    8
    8
    Units: kBq/ml
    median (inter-quartile range (Q1-Q3))
        1h
    0.23 (0.06 to 0.38)
    0.35 (0.19 to 0.47)
    0.44 (0.25 to 0.57)
    0.45 (0.19 to 0.56)
    0.05 (0.03 to 0.19)
    0.38 (0.24 to 0.51)
    0.05 (0.05 to 0.09)
    0.26 (0.18 to 0.49)
        15h
    0.70 (0.55 to 0.76)
    0.62 (0.39 to 0.69)
    0.49 (0.44 to 0.57)
    0.55 (0.42 to 0.62)
    0.33 (0.21 to 0.45)
    0.60 (0.45 to 0.75)
    0.29 (0.14 to 0.63)
    0.54 (0.37 to 0.60)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from inclusion into the study (prior to initiation of treatment) and until 2 weeks following final day of treatment.
    Adverse event reporting additional description
    Reporting adverse events possibly related to treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 None
    Dictionary version
    0
    Reporting groups
    Reporting group title
    Trientine
    Reporting group description
    		 -

    Reporting group title
    Penicillamine
    Reporting group description
    		 -

    Reporting group title
    Tetrathiomolybdate
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo which was visually identical to tetrathiomolybdate.

    Serious adverse events
    		 Trientine Penicillamine Tetrathiomolybdate Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Trientine Penicillamine Tetrathiomolybdate Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 8 (62.50%)
    0 / 8 (0.00%)
    4 / 8 (50.00%)
    1 / 8 (12.50%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    4 / 8 (50.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    4
    0
    Sleep difficulties
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Minor change in biochemical values
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Gastrointestinal discomfort
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    3
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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.
    Small sample size and use of healthy individuals.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/38088886
    http://www.ncbi.nlm.nih.gov/pubmed/38081365
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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
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