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    Clinical Trial Results:
    A Phase 2, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study of DS-1211b in Individuals With PseudoXanthoma Elasticum

    Summary
    EudraCT number
    		 2022-000676-19
    Trial protocol
    		 NL  
    Global end of trial date
    21 Nov 2023

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

    Trial information

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    Trial identification
    Sponsor protocol code
    DS1211-A-U201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05569252
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Daiichi Sankyo, Inc.
    Sponsor organisation address
    211 Mt. Airy Road , Basking Ridge, United States, 07920
    Public contact
    Contact for Clinical Trial Information, Daiichi Sankyo, Inc., +1 908-992-6400, CTRinfo_us@daiichisankyo.com
    Scientific contact
    Contact for Clinical Trial Information, Daiichi Sankyo, Inc., +1 908-992-6400, CTRinfo_us@daiichisankyo.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
    21 Nov 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Nov 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objectives of this trial were to assess the safety and tolerability of DS-1211b compared with placebo in patients with PXE and to assess the dose response by assessing the treatment changes in PD endpoints.
    Protection of trial subjects
    The study protocol, patient information and consent form, the Investigator Brochure, any patient written instructions given to the patient, available safety information, patient recruitment procedures (eg, advertisements), information about payments and compensation available to the patients, and documentation evidencing the Investigator’s qualifications were submitted to the EC or IRB for ethical review and approval according to local regulations, prior to the study start. This study was conducted in compliance with the protocol, the ethical principles that have their origin in the Declaration of Helsinki, the International Council for Harmonization (ICH) consolidated Guideline E6 for GCP (CPMP/ICH/135/95), and applicable regulatory requirement(s).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Oct 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 51
    Country: Number of subjects enrolled
    Netherlands: 14
    Worldwide total number of subjects
    65
    EEA total number of subjects
    14
    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)
    57
    From 65 to 84 years
    8
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 65 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study in the United States and in the Netherlands.

    Pre-assignment
    Screening details
    		 Eligible study participants were randomized in a 1:1:1:1 ratio into 4 treatment groups.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 DS-1211b low dose
    Arm description
    		 Participants who were randomized to receive DS-1211 tablets once daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    DS-1211b
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal.

    Arm title
    		 DS-1211b middle dose
    Arm description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    DS-1211b
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal.

    Arm title
    		 DS-1211b high dose
    Arm description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    DS-1211b
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal.

    Arm title
    		 Placebo
    Arm description
    		 Participants who were randomized to receive placebo tablets once daily for 12 weeks.
    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
    Placebo tablet administered once daily in the morning either in the fasted state or with a meal.

    Number of subjects in period 1
    		DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo
    Started
    16
    16
    16
    17
    Completed
    15
    16
    16
    17
    Not completed
    1
    0
    0
    0
         Adverse event (central vision loss)
    1
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    DS-1211b low dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211 tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b middle dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b high dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who were randomized to receive placebo tablets once daily for 12 weeks.

    Reporting group values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo Total
    Number of subjects
    		 16 16 16 17 65
    Age categorical
    Units: Subjects
        ≥18 years and <64 years
    		 14 14 14 12 54
        ≥64 years
    		 2 2 2 5 11
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 55.9 ( 7.65 ) 51.6 ( 10.84 ) 53.8 ( 8.47 ) 53.5 ( 13.92 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 10 10 14 41
        Male
    		 9 6 6 3 24
    Race/Ethnicity, Customized
    Units: Subjects
        Asian
    		 0 0 0 1 1
        Black or African American
    		 0 1 0 0 1
        White
    		 16 15 16 16 63

    End points

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    End points reporting groups
    Reporting group title
    DS-1211b low dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211 tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b middle dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b high dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who were randomized to receive placebo tablets once daily for 12 weeks.

    Primary: Number of Participants with Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b

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    End point title
    		 Number of Participants with Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b [1]
    End point description
    TEAEs are defined as events that start on or after the first dose of study drug or start prior to but then worsen after the first dose of study drug. Adverse events are coded using MedDRA version 26.1.
    End point type
    Primary
    End point timeframe
    From the date of signing informed consent form up to Day 98 (14 days after last dose of study drug) post-dose of 12-week treatment period
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo
    Number of subjects analysed
    16
    16
    16
    17
    Units: participants
        Any TEAE
    8
    7
    6
    6
        Any TEAE leading to death
    0
    0
    0
    0
        Any TEAE by maximum severity, mild
    4
    6
    4
    3
        Any TEAE by maximum severity, moderate
    3
    1
    2
    3
        Any TEAE by maximum severity, severe
    1
    0
    0
    0
        Any serious TEAE
    1
    0
    0
    0
        Any serious TEAE leading to drug interruption
    0
    0
    0
    0
        Any serious TEAE leading to drug discontinuation
    0
    0
    0
    0
        Any serious TEAE related to study drug
    0
    0
    0
    0
        Any TEAE leading to drug interruption
    1
    0
    0
    0
        Any TEAE leading to drug discontinuation
    1
    0
    0
    0
        Any TEAE related to study drug
    2
    2
    1
    1
        Any related TEAE leading to drug interruption
    1
    0
    0
    0
        Any related TEAE leading to drug discontinuation
    1
    0
    0
    0
        Any TEAE related to COVID-19
    0
    0
    1
    1
    No statistical analyses for this end point

    Primary: Percent Change from Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels

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    End point title
    		 Percent Change from Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels [2]
    End point description
    ALP levels were assessed using the IFCC serum assay.
    End point type
    Primary
    End point timeframe
    Pre-dose on Days 15, 43, 84; Day 86-88 and Day 98 of 12-week treatment period
    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: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo
    Number of subjects analysed
    16
    16
    16
    17
    Units: percentage
    arithmetic mean (standard deviation)
        Day 15
    17.93 ( 10.60 )
    24.39 ( 14.45 )
    39.40 ( 11.52 )
    -0.48 ( 6.14 )
        Day 43
    25.02 ( 17.54 )
    42.89 ( 30.26 )
    63.48 ( 35.68 )
    -2.54 ( 10.38 )
        Day 84
    25.54 ( 16.89 )
    39.81 ( 20.19 )
    61.90 ( 37.86 )
    -4.31 ( 9.58 )
        Day 86-88
    16.60 ( 18.39 )
    29.93 ( 14.61 )
    49.67 ( 26.00 )
    -3.20 ( 9.46 )
        Day 98
    7.65 ( 15.34 )
    9.48 ( 14.99 )
    22.95 ( 22.42 )
    -0.05 ( 13.72 )
    No statistical analyses for this end point

    Primary: Percent Change from Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels

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    End point title
    		 Percent Change from Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels [3]
    End point description
    PPi levels were assessed from collected plasma.
    End point type
    Primary
    End point timeframe
    Pre-dose on Days 15, 43, and 84 of 12-week treatment period
    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: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo
    Number of subjects analysed
    16
    16
    16
    17
    Units: percentage
    arithmetic mean (standard deviation)
        Day 15
    118.97 ( 362.42 )
    11.32 ( 50.13 )
    23.95 ( 69.94 )
    13.33 ( 61.00 )
        Day 43
    17.75 ( 50.05 )
    -5.55 ( 36.74 )
    4.59 ( 19.47 )
    12.97 ( 38.33 )
        Day 84
    23.48 ( 78.04 )
    81.77 ( 313.08 )
    24.27 ( 97.87 )
    4.28 ( 41.45 )
    No statistical analyses for this end point

    Primary: Percent Change from Baseline in Pharmacodynamic Parameter Pyridoxal 5’-phosphate (PLP) Levels

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    End point title
    		 Percent Change from Baseline in Pharmacodynamic Parameter Pyridoxal 5’-phosphate (PLP) Levels [4]
    End point description
    PLP levels were assessed from collected plasma.
    End point type
    Primary
    End point timeframe
    Pre-dose on Days 15, 43, 84; Day 86-88 and Day 98 of 12-week treatment period
    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: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose Placebo
    Number of subjects analysed
    16
    16
    16
    17
    Units: percentage
    arithmetic mean (standard deviation)
        Day 15
    11.01 ( 53.93 )
    45.42 ( 92.18 )
    50.97 ( 56.78 )
    3.42 ( 36.86 )
        Day 43
    8.56 ( 42.26 )
    36.73 ( 79.89 )
    25.00 ( 48.51 )
    16.25 ( 35.09 )
        Day 84
    12.03 ( 46.95 )
    53.07 ( 110.74 )
    6.04 ( 38.72 )
    -8.44 ( 41.44 )
        Day 86-88
    -5.96 ( 27.93 )
    -6.52 ( 41.25 )
    -28.76 ( 31.72 )
    -10.77 ( 45.53 )
        Day 98
    6.10 ( 47.54 )
    -10.96 ( 45.58 )
    -8.41 ( 35.10 )
    17.07 ( 98.34 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Parameter Maximum Concentration (Cmax)

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    End point title
    		 Pharmacokinetic Parameter Maximum Concentration (Cmax) [5]
    End point description
    Pharmacokinetic parameter Cmax was estimated using population PK modeling.
    End point type
    Secondary
    End point timeframe
    Day1 and Day 84 post-dose of 12-week treatment period
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose
    Number of subjects analysed
    16
    16
    16
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Day 1
    252 ( 20.5 )
    540 ( 34.8 )
    713 ( 26.9 )
        Day 84
    255 ( 20.6 )
    547 ( 34.9 )
    726 ( 27.0 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Parameter Time to Maximum Concentration (Tmax)

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    End point title
    		 Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) [6]
    End point description
    Pharmacokinetic parameter Tmax was assessed using population PK modeling.
    End point type
    Secondary
    End point timeframe
    Day 1 and Day 84 post-dose of 12-week treatment period
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose
    Number of subjects analysed
    16
    16
    16
    Units: hours
    median (full range (min-max))
        Day 1
    1.25 (0.75 to 2.00)
    1.13 (0.75 to 3.75)
    1.25 (0.75 to 1.75)
        Day 84
    1.25 (0.75 to 2.00)
    1.13 (0.75 to 3.75)
    1.25 (0.75 to 1.75)
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough)

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    End point title
    		 Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough) [7]
    End point description
    Pharmacokinetic parameter Ctrough was assessed using observed concentrations at 10 hours post-dose.
    End point type
    Secondary
    End point timeframe
    Day 1 and Day 84 post-dose of 12-week treatment period
    Notes
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose
    Number of subjects analysed
    16
    16
    16
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 1 (10 hour)
    17.15 ( 11.58 )
    26.30 ( 13.71 )
    48.43 ( 46.50 )
        Day 84 (10 hour)
    15.19 ( 9.00 )
    38.53 ( 30.45 )
    54.48 ( 40.79 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC)

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    End point title
    		 Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC) [8]
    End point description
    Pharmacokinetic parameter AUCtau was assessed using population PK modeling.
    End point type
    Secondary
    End point timeframe
    Day 1 and Day 84 post-dose of 12-week treatment period
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Descriptive statistics were used to assess this outcome.
    End point values
    		 DS-1211b low dose DS-1211b middle dose DS-1211b high dose
    Number of subjects analysed
    16
    16
    16
    Units: ng*h/mL
    geometric mean (geometric coefficient of variation)
        Day 1, AUCtau
    978 ( 28.3 )
    2090 ( 28.0 )
    2780 ( 32.1 )
        Day 84, AUCtau
    1020 ( 29.3 )
    2210 ( 29.0 )
    3000 ( 33.7 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment-emergent adverse events were collected from the time of signing the informed consent form up to 14 (±5) days after the last dose of study medication.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    DS-1211b middle dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b high dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211b tablets once daily for 12 weeks.

    Reporting group title
    DS-1211b low dose
    Reporting group description
    		 Participants who were randomized to receive DS-1211 tablets once daily for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who were randomized to receive placebo tablets once daily for 12 weeks.

    Serious adverse events
    		 DS-1211b middle dose DS-1211b high dose DS-1211b low dose Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Vascular disorders
    Circulatory collapse
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 DS-1211b middle dose DS-1211b high dose DS-1211b low dose Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 16 (43.75%)
    6 / 16 (37.50%)
    8 / 16 (50.00%)
    6 / 17 (35.29%)
    Vascular disorders
    Circulatory collapse
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hot flush
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Reproductive system and breast disorders
    Menstrual disorder
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Postmenopausal haemorrhage
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Psychiatric disorders
    Depression
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Investigations
    Heart rate increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Heart rate irregular
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    SARS CoV-2 test positive
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Injury, poisoning and procedural complications
    Tendon rupture
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Stress fracture
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cardiac disorders
    Atrial flutter
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Palpitations
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nervous system disorders
    Tremor
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sciatica
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ophthalmic migraine
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Headache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Head discomfort
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Ear and labyrinth disorders
    Ear discomfort
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eye disorders
    Blepharospasm
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Cataract
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Central vision loss
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    1
    0
    Paraesthesia oral
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dyspepsia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Constipation
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Flank pain
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Fascitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Arthritis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Arthralgia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Pain in extremity
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal stiffness
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal discomfort
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Muscle spasms
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    0
    2
    Otitis media chronic
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Influenza
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    COVID-19
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    1
    Urinary tract infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    1
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 16 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Oct 2022
    Updated the Schedule of Events for study assessments, clarified the process for safety monitoring, and other minor updates regarding data collection.
    16 Jan 2023
    Updated exploratory analyses, clarified Schedule of Events during treatment period, and revised patient rescreening and AE monitoring procedures.

    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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