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    Clinical Trial Results:
    Efficacy of ALXN1840 on human biliary copper excretion quantified with 64CuCl2 PET/MR-scan

    Summary
    EudraCT number
    		 2021-000102-25
    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-WD-Cu-Excretion
    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,
    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
    20 May 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 whether ALXN1840 increases biliary copper excretion in Wilson's Disease patients.
    Protection of trial subjects
    Blood samples before and after treatment, including liver, kidney and hematological parameters. Only inclusion of stable patients 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
    21 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
    Denmark: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    5
    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)
    4
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participating patients were recruited from the outpatient clinic at the Department of Hepatology and Gastroenterology, Aarhus University Hospital after clinical assessment of disease stability. The diagnosis was confirmed in accordance with the Leipzig criteria.

    Pre-assignment
    Screening details
    		 Interested patients were evaluated for stability, length and choice of current Wilson treatment and after this receive the full study participant information and material. If still interested they would be contacted by telephone and finally be invited for a face-to-face meeting and inclusion into the study.

    Period 1
    Period 1 title
    Pre-treatment
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Pre-treatment
    Arm description
    		 -
    Arm type
    		 Baseline, pre-treatment

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Bis-choline tetrathiomolybdate
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg once daily, oral. Fast one hour before and after each dose.

    Number of subjects in period 1
    		Pre-treatment
    Started
    5
    Completed
    4
    Not completed
    1
         Consent withdrawn by subject
    1
    Period 2
    Period 2 title
    Post-treatment
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Post-treatment
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1840
    Investigational medicinal product code
    Other name
    Bis-choline tetrathiomolybdate
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg once daily, oral. Fast one hour before and after each dose.

    Number of subjects in period 2
    		Post-treatment
    Started
    4
    Completed
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pre-treatment
    Reporting group description
    		 -

    Reporting group values
    		 Pre-treatment Total
    Number of subjects
    		 5 5
    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)
    		 4 4
        From 65-84 years
    		 1 1
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 4 4
    Alanine aminotransferase
    Units: U/L
        median (inter-quartile range (Q1-Q3))
    		 39.50 (25.50 to 59.00) -
    Bilirubine
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 9.50 (7.00 to 16.50) -
    Creatinine
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 63.00 (58.00 to 72.00) -
    P-copper
    Units: umol/L
        median (inter-quartile range (Q1-Q3))
    		 2.45 (1.30 to 5.30) -

    End points

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    End points reporting groups
    Reporting group title
    Pre-treatment
    Reporting group description
    		 -
    Reporting group title
    Post-treatment
    Reporting group description
    		 -

    Primary: Gallbladder

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    End point title
    		 Gallbladder
    End point description
    Multiple scans during the study period both before and after treatment.
    End point type
    Primary
    End point timeframe
    1 - 68H after tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1H
    2.17 (1.08 to 2.97)
    1.31 (0.85 to 1.88)
        2H
    3.44 (2.22 to 4.00)
    2.06 (1.25 to 2.23)
        6H
    3.10 (3.02 to 3.22)
    1.98 (1.21 to 2.48)
        20H
    3.27 (2.58 to 4.33)
    2.44 (1.63 to 4.11)
        48H
    2.80 (2.58 to 3.38)
    2.55 (2.49 to 3.10)
        54H
    3.03 (2.56 to 4.06)
    3.05 (2.44 to 3.70)
        68H
    2.15 (1.18 to 3.76)
    2.78 (1.77 to 3.37)
    Statistical analysis title
    		 Statistical test
    Statistical analysis description
    Each of the four subjects acted as their own control. Pre vs post-treatment.
    Comparison groups
    Pre-treatment v Post-treatment
    Number of subjects included in analysis
    8
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 > 0.05 [1]
    Method
    		 Sign test
    Confidence interval
    Notes
    [1] - At time 1, 2, 20, 48, 54, 68 hours since tracer injection, p>0.05 (Non-significant). At time 6 hours since tracer injection, p=0.0209 (Significant reduction in gallbladder SUV on TTM).

    Secondary: Liver

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    End point title
    		 Liver
    End point description
    End point type
    Secondary
    End point timeframe
    1 - 68H post tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1H
    17.77 (16.67 to 21.19)
    7.44 (5.96 to 8.97)
        2H
    21.77 (19.54 to 24.23)
    8.76 (7.95 to 10.77)
        6H
    27.11 (24.30 to 32.08)
    14.30 (13.32 to 16.83)
        20H
    33.57 (29.80 to 38.42)
    22.59 (19.38 to 26.09)
        48H
    37.71 (34.66 to 40.70)
    28.93 (26.63 to 29.53)
        54H
    36.41 (32.16 to 41.52)
    27.22 (24.65 to 29.06)
        68H
    38.66 (34.67 to 43.54)
    28.48 (25.41 to 31.48)
    No statistical analyses for this end point

    Secondary: Kidney

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    End point title
    		 Kidney
    End point description
    End point type
    Secondary
    End point timeframe
    1 - 68H post tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1H
    7.23 (6.61 to 9.27)
    6.87 (6.28 to 9.27)
        2H
    5.59 (5.17 to 8.89)
    6.78 (6.52 to 9.25)
        6H
    4.81 (3.93 to 7.06)
    9.05 (7.92 to 11.30)
        20H
    2.79 (2.36 to 3.85)
    10.59 (9.24 to 11.34)
        48H
    1.63 (1.38 to 2.10)
    12.72 (11.25 to 14.02)
        54H
    1.73 (1.52 to 1.95)
    12.77 (10.39 to 14.88)
        68H
    1.45 (1.13 to 1.96)
    11.77 (11.19 to 12.54)
    No statistical analyses for this end point

    Secondary: Pancreas

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    End point title
    		 Pancreas
    End point description
    End point type
    Secondary
    End point timeframe
    1 - 68H post tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1H
    7.65 (5.67 to 14.02)
    4.96 (4.25 to 6.94)
        2H
    7.60 (5.85 to 12.49)
    4.36 (3.65 to 5.36)
        6H
    4.37 (2.98 to 9.50)
    4.69 (3.24 to 5.82)
        20H
    4.67 (3.63 to 6.22)
    2.94 (2.76 to 4.10)
        48H
    2.55 (2.22 to 3.14)
    3.37 (2.22 to 3.78)
        54H
    2.76 (2.42 to 3.11)
    2.36 (1.75 to 3.69)
        68H
    1.60 (1.05 to 1.83)
    2.86 (1.59 to 3.35)
    No statistical analyses for this end point

    Secondary: Brain

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    End point title
    		 Brain
    End point description
    The brain was only within the PET-field of view for the first 20H.
    End point type
    Secondary
    End point timeframe
    1 - 68H post tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: SUV
    median (inter-quartile range (Q1-Q3))
        1H
    0.11 (0.05 to 0.20)
    0.20 (0.18 to 0.24)
        2H
    0.06 (0.05 to 0.11)
    0.19 (0.11 to 0.34)
        6H
    0.13 (0.07 to 0.18)
    0.26 (0.18 to 0.31)
        20H
    0.12 (0.08 to 0.20)
    0.09 (0.07 to 0.11)
    No statistical analyses for this end point

    Secondary: Blood

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    End point title
    		 Blood
    End point description
    End point type
    Secondary
    End point timeframe
    1 - 68H post tracer injection.
    End point values
    		 Pre-treatment Post-treatment
    Number of subjects analysed
    4
    4
    Units: kBq/ml
    median (inter-quartile range (Q1-Q3))
        1H
    0.86 (0.71 to 0.94)
    5.53 (4.46 to 5.91)
        2H
    0.69 (0.57 to 0.77)
    5.03 (4.01 to 5.32)
        6H
    0.44 (0.37 to 0.49)
    2.35 (1.98 to 3.16)
        20H
    0.34 (0.31 to 0.39)
    0.73 (0.59 to 0.99)
        48H
    0.35 (0.32 to 0.41)
    0.52 (0.49 to 0.55)
        54H
    0.28 (0.28 to 0.35)
    0.41 (0.38 to 0.44)
        68H
    0.28 (0.25 to 0.33)
    0.39 (0.36 to 0.46)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Adverse events were reported from inclusion into the study (prior to initiation of experimental treatment) and until 2 weeks following final day of treatment.
    Adverse event reporting additional description
    No adverse events were reported during the study period or at follow-up.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 None
    Dictionary version
    0
    Reporting groups
    Reporting group title
    Overall
    Reporting group description
    		 No adverse events were reported by any subject in this study. Participants received a paper form on which they were instructed to report any adverse event, even if they were not sure an event was related to the study. Participants were asked prior to each scan and were contacted by telephone 2 weeks after end-of-treatment.

    Serious adverse events
    		 Overall
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 4 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Overall
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 4 (0.00%)
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: There were no non-serious events reported by participants in this study. Patients were instructed to note any possible adverse events even if not sure. Participants were provided a paper form in which all adverse events could be filled out and were asked prior to each scan. Participants were contacted by phone 14 days after end-of-treatment and were again asked about any adverse events.

    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.
    None reported

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