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    Clinical Trial Results:
    A randomised, double-blind, placebo-controlled, parallel-group trial investigating the effect of 4 weeks bi-daily dosing of XEN-D0501 on blood glucose reduction as add-on to metformin in patients with diabetes type 2

    Summary
    EudraCT number
    		 2018-001880-22
    Trial protocol
    		 LT  
    Global end of trial date
    19 Dec 2019

    Results information
    Results version number
    		 v2(current)
    This version publication date
    14 Jan 2023
    First version publication date
    21 Jul 2022
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    The data set has been corrected and aligned with the final clinical study report.

    Trial information

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    Trial identification
    Sponsor protocol code
    PP-CT02
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05353686
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PILA PHARMA AB
    Sponsor organisation address
    Västergatan 1 , Malmö, Sweden, 211 21
    Public contact
    Dorte X. Gram, PILA PHARMA AB, +46 73903 6969, info@pilapharma.com
    Scientific contact
    Dorte X. Gram, PILA PHARMA AB, +46 73903 6969, info@pilapharma.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
    23 Jun 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Dec 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Dec 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the effects of four weeks of bi-daily dosing of XEN-D0501 (4 mg BID) as add-on to metformin on fasting blood glucose in patients with diabetes mellitus type 2
    Protection of trial subjects
    None
    Background therapy
    		 All subjects received metformin as background therapy.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Feb 2019
    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
    Lithuania: 60
    Worldwide total number of subjects
    60
    EEA total number of subjects
    60
    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)
    38
    From 65 to 84 years
    22
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 77 subjects gave informed consent and were screened. Of those, 60 subjects fulfilled the eligibility criteria and were randomised to treatment.

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 77 [1]
    Number of subjects completed
    		 60

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Consent withdrawn by subject: 1
    Reason: Number of subjects
    		 Screen failures: 16
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: ���� ���������������� �������� ���������������� �������������� ������ ���� ���� ���������� ������������������ ������ ���������������������� ���������������� ������ �������� �������������������� ���� ������������������77 subject gave informed consent and 60 of those fulfilled the eligibility criteria and were randomized to treatment.
    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 1 tablet twice daily
    Arm type
    		 Placebo

    Investigational medicinal product name
    Reference treatment (placebo)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 oral tablet twice daily

    Arm title
    		 XEN-D0501
    Arm description
    		 1 tablet of 4 mg twice daily
    Arm type
    		 Experimental

    Investigational medicinal product name
    XEN-D0501
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 oral tablet of 4 mg twice daily

    Number of subjects in period 1
    		Placebo XEN-D0501
    Started
    31
    29
    Completed
    31
    29
    Period 2
    Period 2 title
    Treatment
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 1 tablet twice daily
    Arm type
    		 Placebo

    Investigational medicinal product name
    Reference treatment (placebo)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 oral tablet twice daily

    Arm title
    		 XEN-D0501
    Arm description
    		 1 tablet of 4 mg twice daily
    Arm type
    		 Experimental

    Investigational medicinal product name
    XEN-D0501
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 oral tablet of 4 mg twice daily

    Number of subjects in period 2
    		Placebo XEN-D0501
    Started
    31
    29
    Completed
    31
    26
    Not completed
    0
    3
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    -
    1
         Protocol deviation
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 1 tablet twice daily

    Reporting group title
    XEN-D0501
    Reporting group description
    		 1 tablet of 4 mg twice daily

    Reporting group values
    		 Placebo XEN-D0501 Total
    Number of subjects
    		 31 29 60
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 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)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Units: years
        geometric mean (full range (min-max))
    		 60.6 (29 to 84) 62.1 (49 to 77) -
    Gender categorical
    Units: Subjects
        Female
    		 21 16 37
        Male
    		 10 13 23
    Ethnic origin
    Units: Subjects
        White
    		 31 29 60
    Diabetes duration
    Units: Years
        geometric mean (standard deviation)
    		 7.2 ± 5.9 5.8 ± 3.6 -
    Height
    Units: cm
        geometric mean (full range (min-max))
    		 167.3 (149 to 185) 170.2 (146 to 187) -
    Weight
    Units: kg
        geometric mean (full range (min-max))
    		 92.3 (59 to 135) 99.2 (52 to 131) -
    BMI
    Units: mg/kg*2
        geometric mean (full range (min-max))
    		 33 (23 to 46) 34.3 (19 to 46) -
    Waist circumference
    Units: cm
        geometric mean (full range (min-max))
    		 110.1 (87 to 149) 114.2 (79 to 137) -
    Hip circumference
    Units: cm
        geometric mean (full range (min-max))
    		 114.1 (95 to 139) 116.5 (87 to 141) -
    Waist-hip ratio
    Units: cm/cm
        geometric mean (full range (min-max))
    		 1 (0.8 to 1.1) 1 (0.8 to 1.1) -
    HbA1c
    Units: percent
        geometric mean (standard deviation)
    		 7.3 ± 0.92 7.5 ± 0.99 -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 1 tablet twice daily

    Reporting group title
    XEN-D0501
    Reporting group description
    		 1 tablet of 4 mg twice daily
    Reporting group title
    Placebo
    Reporting group description
    		 1 tablet twice daily

    Reporting group title
    XEN-D0501
    Reporting group description
    		 1 tablet of 4 mg twice daily

    Subject analysis set title
    PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants in the study who completed the study and had no major protocol deviations. The PP population also excluded 3 participants with a minor protocol deviation, i.e., those who took the last study medication dose > 1 day (24 hours) before V4

    Subject analysis set title
    ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    All randomized participants

    Primary: Fasting blood glucose after 4 weeks of treatment, PP population

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    End point title
    		 Fasting blood glucose after 4 weeks of treatment, PP population
    End point description
    Measured after four weeks of treatment (visit 4) and at baseline (visit 3) for the PP population
    End point type
    Primary
    End point timeframe
    At 4 weeks after treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: mmol/L
    geometric mean (standard deviation)
        Visit 4
    7.868 ± 1.798
    8.213 ± 2.034
        Change from baseline
    -0.123 ± 1.114
    -0.272 ± 1.250
        Baseline (Visit 3)
    8.0 ± 1.9
    8.5 ± 2.2
    Statistical analysis title
    		 Analysis of primary endpoint - visit 4
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.5123
    Method
    		 t-test, 2-sided
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.345
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.395
         upper limit
    		 0.705
    Statistical analysis title
    		 Analysis of primary endpoint - change from BL
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.6452
    Method
    		 t-test, 2-sided
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.15
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.4986
         upper limit
    		 0.7978

    Primary: Fasting blood glucose after 4 weeks of treatment, ITT population

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    End point title
    		 Fasting blood glucose after 4 weeks of treatment, ITT population
    End point description
    Measured after 4 weeks of treatment (visit 4) and at baseline (visit 3) fro the ITT population
    End point type
    Primary
    End point timeframe
    At 4 weeks after treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    29
    Units: mmol/L
    geometric mean (standard deviation)
        Baseline (visit 3)
    8.0 ± 1.9
    8.6 ± 2.1
        Visit 4
    7.9 ± 1.8
    8.4 ± 2.1
        Change from baseline (visit 4- visit 3)
    -0.1 ± 1.1
    -0.2 ± 1.2
    Statistical analysis title
    		 Analysis of primary endpoint - visit 4, ITT pop
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    60
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.3095
    Method
    		 t-test, 2-sided
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.525
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.5511
         upper limit
    		 0.5007

    Secondary: Self-monitored blood glucose after 2 and 4 weeks of treatment

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    End point title
    		 Self-monitored blood glucose after 2 and 4 weeks of treatment
    End point description
    End point type
    Secondary
    End point timeframe
    At 2 and 4 weeks after treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: mmol/L
    geometric mean (standard deviation)
        Wake-up, 2 wks
    8.4 ± 2.5
    8.7 ± 2.09
        Before breakfast, 2 wks
    8.8 ± 2.3
    9.1 ± 2.0
        2 hrs after breakfast, 2 wks
    9.4 ± 3.1
    9.7 ± 3.2
        Before lunch, 2 wks
    7.3 ± 2.1
    8.4 ± 2.7
        2 hrs after lunch, 2 wks
    9.4 ± 2.4
    9.2 ± 2.3
        2 hrs after dinner, 2 wks
    8.6 ± 2.2
    9.3 ± 2.2
        Bedtime, 2 hrs
    8.8 ± 3.0
    9.0 ± 2.3
        Wake-up, 4 wks
    8.3 ± 1.9
    8.6 ± 1.8
        Before breakfast, 4 wks
    8.4 ± 1.9
    8.9 ± 1.9
        2 hrs after breakfast, 4 wks
    9.0 ± 2.3
    9.6 ± 3.3
        Before lunch, 4 wks
    7.4 ± 1.8
    7.8 ± 2.7
        2 hrs after lunch, 4 wks
    9.1 ± 3.0
    8.9 ± 2.0
        2 hrs after dinner, 4 wks
    9.3 ± 2.6
    9.1 ± 2.2
        Bedtime, 4 wks
    8.8 ± 2.6
    8.4 ± 2.5
        Before dinner, 2 wks
    8.2 ± 2.1
    8.6 ± 2.6
        Before dinner, 4 wks
    8.2 ± 2.5
    8.5 ± 2.2
    No statistical analyses for this end point

    Secondary: Plasma HbA1c after 4 weeks of treatment

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    End point title
    		 Plasma HbA1c after 4 weeks of treatment
    End point description
    After 4 weeks of treatment (visit 4) and as change from baseline (visit 3)
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: percent
    geometric mean (standard deviation)
        Visit 4
    7.2 ± 0.95
    7.2 ± 0.93
        Change from baseline (visit 3)
    -0.1 ± 0.33
    -0.2 ± 0.3
        Visit 3
    7.3 ± 0.92
    7.4 ± 0.98
    No statistical analyses for this end point

    Secondary: Glucose tolerance (OGTT) after 4 weeks of treatment

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    End point title
    		 Glucose tolerance (OGTT) after 4 weeks of treatment
    End point description
    Measured after 4 weeks of treatment (visit 4) and compared to baseline (-60 min) Change of the changes from baseline represents the change between 120 and 0 min at visit 4 minus the change between 120 and 0 min at visit 3
    End point type
    Secondary
    End point timeframe
    At 4 weeks after treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: mmol/L
    geometric mean (standard deviation)
        4 weeks (visit 4), 120 min
    15.18 ± 3.17
    13.42 ± 3.48
        4 weeks (visit 4), change from baseline
    7.3 ± 2.9
    5.2 ± 3.0
        Change of the changes
    0.69 ± 2.68
    -0.15 ± 2.4
    Statistical analysis title
    		 Plasma glucose at 120 min (V4 vs baseline -60 min)
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.013
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: Insulin secretion during an OGTT

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    End point title
    		 Insulin secretion during an OGTT
    End point description
    Measured after 4 weeks of treatment (visit 4) and compared to baseline (-60 min) Change of the changes from baseline represents the change between 120 and 0 min at visit 4 minus the change between 120 and 0 min at visit 3
    End point type
    Secondary
    End point timeframe
    At 4 weeks after treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: μU/mL
    geometric mean (standard deviation)
        Actual 120 min (V4)
    57.2 ± 42.4
    63.4 ± 42.3
        Change from baseline, 30 min
    20.1 ± 14.7
    33.7 ± 22.9
        Change from baseline, 60 min
    33.5 ± 24.1
    50.1 ± 34.9
        Change from baseline, 90 min
    43.3 ± 28.6
    57.3 ± 43.2
        Change from baseline, 120 min
    44.3 ± 37.5
    47.3 ± 35.8
        Change of the changes, 120 min
    2.9 ± 19.8
    2.0 ± 31.1
        Actual 30 min (V4)
    33 ± 19.4
    49.8 ± 30.1
        Actual 60 min (V4)
    46.3 ± 29.7
    66.2 ± 42.4
        Actual 90 min (V4)
    56.5 ± 34.5
    73.4 ± 49.4
    Statistical analysis title
    		 Insulin secretion at visit 4, 30 min (vs -60 min)
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.0178
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Insulin secretion at visit 4, 60 min (vs -60 min)
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.0429
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Insulin secretion at visit 4, 90 min (vs -60 min)
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.1878
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Insulin secretion at visit 4, 120 min (vs -60 min)
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.7688
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: HOMA insulin resistance and beta cell function

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    End point title
    		 HOMA insulin resistance and beta cell function
    End point description
    Measured after 4 weeks of treatment (visit 4) and compared to baseline (visit 3)
    End point type
    Secondary
    End point timeframe
    At 4 weeks after treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: pmol/L
    geometric mean (standard deviation)
        Insulin resistance, visit 4
    4.5 ± 3.1
    5.9 ± 4.5
        Insulin resistance, change from baseline visit 4
    -0.8 ± 2.1
    -1.0 ± 2.0
        Beta cell function, visit 4
    67.1 ± 48.5
    85.2 ± 80.8
        Beta cell function, change from baseline visit 4
    -9.9 ± 28.1
    8.0 ± 78.0
    No statistical analyses for this end point

    Secondary: Fasting insulin after 4 weeks of treatment

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    End point title
    		 Fasting insulin after 4 weeks of treatment
    End point description
    Measured 4 weeks after treatment (visit 4) and compared to baseline (visit 3)
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: μU/mL
    geometric mean (standard deviation)
        Insulin, visit 4
    12.9 ± 8.0
    16.1 ± 11.2
        Insulin, change from baseline
    -2.3 ± 4.8
    -1.6 ± 4.7
    No statistical analyses for this end point

    Secondary: Body weight after 4 weeks of treatment

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    End point title
    		 Body weight after 4 weeks of treatment
    End point description
    Measured at baseline (visit 3) and after 4 weeks of treatment (visit 4)
    End point type
    Secondary
    End point timeframe
    At 4 weeks after treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: Kg
    geometric mean (standard deviation)
        Baseline (visit 3)
    92.3 ± 20.4
    103.4 ± 15.5
        Visit 4
    91.8 ± 20.6
    103.1 ± 15.3
        Change from baseline (visit 4 - visit 3)
    -0.5 ± 1.4
    -0.3 ± 1.2
    No statistical analyses for this end point

    Secondary: Waist circumference

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    End point title
    		 Waist circumference
    End point description
    Measured at baseline (visit 3) and after 4 weeks of treatment (visit 4)
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: cm
    geometric mean (standard deviation)
        Baseline (visit 3)
    109.7 ± 13.6
    116.7 ± 11.8
        Visit 4
    108.7 ± 13.4
    116.5 ± 11.6
        Change from baseline (visit 4 - visit 3)
    -0.9 ± 2.5
    -0.2 ± 1.6
    No statistical analyses for this end point

    Secondary: Waist-hip ratio

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    End point title
    		 Waist-hip ratio
    End point description
    Measured at baseline (visit 3) and after 4 weeks of treatment (visit 4)
    End point type
    Secondary
    End point timeframe
    At 4 weeks after treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: cm/cm
    geometric mean (standard deviation)
        Baseline (visit 3)
    1.0 ± 0.1
    1.0 ± 0.1
        Visit 4
    0.9 ± 0.1
    1.0 ± 0.1
        Change from baseline (visit 4 - visit 3)
    -0.006 ± 0.04
    -0.004 ± 0.02
    No statistical analyses for this end point

    Secondary: Fasting blood lipids after 4 weeks of treatment

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    End point title
    		 Fasting blood lipids after 4 weeks of treatment
    End point description
    Measured after 4 weeks (visit 4) and as change from baseline (visit 4 - visit 3)
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: mmol/L
    geometric mean (standard deviation)
        LDL, visit 4
    3.2 ± 0.9
    3.0 ± 1.17
        LDL, change from baseline
    0.05 ± 0.57
    0.08 ± 0.55
        HDL, visit 4
    1.3 ± 0.27
    1.3 ± 0.35
        HDL, change from baseline
    0.007 ± 0.14
    0.012 ± 0.15
        TAG, visit 4
    2.3 ± 1.21
    2.0 ± 0.7
        TAG, change from baseline
    -0.051 ± 0.76
    -0.021 ± 0.48
    No statistical analyses for this end point

    Secondary: Plasma CRP after 4 weeks of treatment

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    End point title
    		 Plasma CRP after 4 weeks of treatment
    End point description
    Measured after 4 weeks of treatment (visit 4) and as change from baseline (visit 4 - visit 3)
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: mg/L
    geometric mean (standard deviation)
        Visit 4
    4.0 ± 4.7
    5.1 ± 5.7
        Change from baseline
    -1.33 ± 8.29
    0.90 ± 5.61
    No statistical analyses for this end point

    Secondary: Pro-BNP after 4 weeks of treatment

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    End point title
    		 Pro-BNP after 4 weeks of treatment
    End point description
    End point type
    Secondary
    End point timeframe
    At 4 weeks of treatment (visit 4)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    24
    18
    Units: pg/mL
    geometric mean (standard deviation)
        Visit 4
    475.8 ± 754.0
    133.3 ± 85.0
        Change from baseline (visit 4 - visit 3)
    43.3 ± 213.1
    13.1 ± 57.5
    No statistical analyses for this end point

    Secondary: Hyperthermia events

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    End point title
    		 Hyperthermia events
    End point description
    End point type
    Secondary
    End point timeframe
    From start of treatment to follow-up, visit 5
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: Events
    0
    0
    No statistical analyses for this end point

    Secondary: Hypoglycemic events

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    End point title
    		 Hypoglycemic events
    End point description
    End point type
    Secondary
    End point timeframe
    From start of treatment to follow-up (visit 5)
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: Events
    1
    0
    No statistical analyses for this end point

    Secondary: Plasma glucagon after four weeks of bi-daily doses of XEN-D0501

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    End point title
    		 Plasma glucagon after four weeks of bi-daily doses of XEN-D0501
    End point description
    Measured at baseline (visit 3) and after 4 weeks of treatment (visit 4)
    End point type
    Secondary
    End point timeframe
    After 4 weeks
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    28
    22
    Units: pmol/L
    geometric mean (standard deviation)
        Baseline (visit 3)
    6.2 ± 4.5
    5.4 ± 3.6
        Visit 4
    6.4 ± 3.8
    7.5 ± 5.5
        Change from baseline (visit 4 - visit 3)
    0.3 ± 3.7
    3 ± 5.6
    No statistical analyses for this end point

    Secondary: Plasma concentration of XEN-D0501 after four weeks of bi-daily dosing of XEN-D0501

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    End point title
    		 Plasma concentration of XEN-D0501 after four weeks of bi-daily dosing of XEN-D0501
    End point description
    End point type
    Secondary
    End point timeframe
    After 4 weeks
    End point values
    		 XEN-D0501
    Number of subjects analysed
    23
    Units: ng/mL
    arithmetic mean (full range (min-max))
        Cmax
    66.2 (14.1 to 159)
    No statistical analyses for this end point

    Secondary: Safety

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    End point title
    		 Safety
    End point description
    End point type
    Secondary
    End point timeframe
    From signing of informed content to end of trial participation
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    29
    Units: Events
        Adverse events
    13
    57
        SAEs
    0
    0
    No statistical analyses for this end point

    Secondary: ANP after 4 weeks of treatment

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    End point title
    		 ANP after 4 weeks of treatment
    End point description
    Measured 4 weeks after treatment and at baseline (visit 3) and as change from baseline
    End point type
    Secondary
    End point timeframe
    At 4 weeks
    End point values
    		 Placebo XEN-D0501
    Number of subjects analysed
    31
    23
    Units: pmol/L
    geometric mean (standard deviation)
        Visit 4
    83.2 ± 65.3
    58.9 ± 26.2
        Visit 3
    78.2 ± 73.1
    66.4 ± 24.5
        Change from baseline (visit 3)
    3.9 ± 14.1
    -7.5 ± 16.1
    Statistical analysis title
    		 ANP change from baseline
    Comparison groups
    Placebo v XEN-D0501
    Number of subjects included in analysis
    54
    Analysis specification
    Post-hoc
    Analysis type
    		 equivalence
    P-value
    		 = 0.0097
    Method
    		 t-test, 2-sided
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signing of informed consent to end of trial participation
    Adverse event reporting additional description
    Adverse events are collected at each visit. The Investigator asked the subjects about AEs by asking: “Have you experienced any problems since the last contact?”
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group title
    XEN-D0501
    Reporting group description
    		 -

    Serious adverse events
    		 Placebo XEN-D0501
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 29 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Placebo XEN-D0501
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 31 (29.03%)
    17 / 29 (58.62%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Basal cell carcinoma
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Gastrointestinal polyp
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 29 (6.90%)
         occurrences all number
    0
    2
    Hot flush
         subjects affected / exposed
    0 / 31 (0.00%)
    4 / 29 (13.79%)
         occurrences all number
    0
    4
    Hypertension
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Surgical and medical procedures
    Skin neoplasm excision
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Feeling cold
         subjects affected / exposed
    1 / 31 (3.23%)
    3 / 29 (10.34%)
         occurrences all number
    1
    5
    Feeling hot
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 29 (10.34%)
         occurrences all number
    0
    5
    Pyrexia
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 29 (3.45%)
         occurrences all number
    2
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 29 (3.45%)
         occurrences all number
    1
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Investigations
    Body temperature increased
         subjects affected / exposed
    3 / 31 (9.68%)
    2 / 29 (6.90%)
         occurrences all number
    4
    2
    Heart rate increased
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    3
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Headache
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 29 (6.90%)
         occurrences all number
    0
    2
    Hypoaesthesia
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 29 (6.90%)
         occurrences all number
    0
    3
    Hypogeusia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Paraesthesia
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 29 (10.34%)
         occurrences all number
    0
    3
    Taste disorder
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 29 (10.34%)
         occurrences all number
    0
    3
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 29 (3.45%)
         occurrences all number
    1
    1
    Tongue oedema
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Hypoaesthesia oral
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
         subjects affected / exposed
    1 / 31 (3.23%)
    3 / 29 (10.34%)
         occurrences all number
    1
    4
    Dermatitis
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Polyuria
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Urinary incontinence
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Carpal tunnel syndrome
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    2
    Meniscus injury
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Pain in extremity
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 29 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 29 (0.00%)
         occurrences all number
    2
    0
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 29 (3.45%)
         occurrences all number
    0
    1

    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
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