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    Clinical Trial Results:
    A 12-WEEK, MULTICENTER, RANDOMIZED, PARALLEL-GROUP STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, BIOMARKER EFFECTS, EFFICACY, AND EFFECT ON MICROGLIA ACTIVATION, AS MEASURED BY POSITRON EMISSION TOMOGRAPHY, OF AZD3241 IN SUBJECTS WITH MULTIPLE SYSTEM ATROPHY

    Summary
    EudraCT number
    2014-004902-13
    Trial protocol
    GB   FI   SE  
    Global end of trial date
    29 Sep 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    20 Aug 2017
    First version publication date
    20 Aug 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    D0490C00023
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    AstraZeneca AB
    Sponsor organisation address
    Pepparedsleden 1, Molndal, Sweden, SE - 431 83
    Public contact
    AstraZeneca AB, AstraZeneca AB, Clinicaltrialtransparency@astrazeneca.net
    Scientific contact
    AstraZeneca AB, AstraZeneca AB, Clinicaltrialtransparency@astrazeneca.net
    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
    26 Jan 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    29 Sep 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Sep 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objectives of the study were: • To assess the safety and tolerability of AZD3241 in subjects with MSA. • To determine the effect of AZD3241 on microglia activation, as measured by [11C]PBR28 binding, in subjects with MSA.
    Protection of trial subjects
    Periodic review of patient safety (quarterly review) Interim analysis when 40 subjects had reached 4 weeks of treatment with potential to stop a treatment arm for patient safety. Conducted in April 2016, with recommendation to continue per protocol.
    Background therapy
    Allowed if stable for minimum of 30 days dosing and not on list of prohibited medications.
    Evidence for comparator
    Placebo used since currently no specific treatments for Multiple System Atropy.
    Actual start date of recruitment
    22 Apr 2015
    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
    Austria: 1
    Country: Number of subjects enrolled
    Finland: 1
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Italy: 12
    Country: Number of subjects enrolled
    Sweden: 1
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    United States: 24
    Worldwide total number of subjects
    59
    EEA total number of subjects
    35
    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)
    45
    From 65 to 84 years
    14
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    124 subjects were screened,signed consent. A total of 59 subjects were dispensed treatment through randomization. 1 subject was notified as ineligible prior to first dose, provided no safety data and is excluded from analysis data sets.

    Pre-assignment
    Screening details
    Rescreening was permitted if eligibility could not be confirmed within the 49 day screening period. A total of 59 were actually considered to have completed screening and were randomized, 58 were included in analyses as 1 subject not dosed provided no information.

    Period 1
    Period 1 title
    Dose escalation Week 1
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Carer, Data analyst, Assessor
    Blinding implementation details
    subjects assigned treatment through central randomisation schedule

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Placebo to match AZD3241 dosed twice daily
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo (to match AZD3241)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    0 mg, Twice daily, Week 1

    Arm title
    AZD3241 300 mg
    Arm description
    AZD3241 300 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 100 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    100 mg, Twice daily, Week 1

    Arm title
    AZD3241 600 mg
    Arm description
    AZD3241 600 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 100 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    100 mg, Twice daily, Week 1

    Number of subjects in period 1 [1]
    Placebo AZD3241 300 mg AZD3241 600 mg
    Started
    19
    19
    20
    Completed
    17
    16
    18
    Not completed
    2
    3
    2
         Determined not eligible
    1
    1
    1
         Adverse event, non-fatal
    1
    2
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 61 subjects were issued randomization assignments, 2 were determined to be screen failures and were never dispensed study medication, thus 59 were dispensed study medication. 1 additional subject (randomized to placebo group) was determined to be a screen failure after the subject left the study site, and was notified by the site prior to the first dose. The subject refused to return to the site, and provided no safety information. Thus only 58 subjects were included in the Safety analysis se
    Period 2
    Period 2 title
    Dose escalation Week 2
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Carer, Data analyst, Assessor
    Blinding implementation details
    continuation of study

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Placebo to match AZD3241 dosed twice daily
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo (to match AZD3241)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    0 mg, Twice daily, Week 2

    Arm title
    AZD3241 300 mg
    Arm description
    AZD3241 300 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 300 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    300 mg, Twice daily, Week 2

    Arm title
    AZD3241 600 mg
    Arm description
    AZD3241 600 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 300 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    300 mg, Twice daily, Week 2

    Number of subjects in period 2
    Placebo AZD3241 300 mg AZD3241 600 mg
    Started
    17
    16
    18
    Completed
    16
    14
    17
    Not completed
    1
    2
    1
         Protocol deviation
    -
    1
    -
         Determined not eligible
    1
    1
    1
    Period 3
    Period 3 title
    Treatment Weeks 3 to 12
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Carer, Data analyst, Assessor
    Blinding implementation details
    Continuation of study

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Placebo to match AZD3241 dosed twice daily
    Arm type
    Placebo

    Investigational medicinal product name
    Plcebo to match AZD3241
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    0 mg, Twice a day, Week 3 through 12

    Arm title
    AZD3241 300 mg
    Arm description
    AZD3241 300 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 300 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    300 mg, Twice daily, Week 3 through 12

    Arm title
    AZD3241 600 mg
    Arm description
    AZD3241 600 mg dosed twice daily
    Arm type
    Experimental

    Investigational medicinal product name
    AZD3241 300 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Two 300 mg, Twice daily, Week 3 through 12

    Number of subjects in period 3
    Placebo AZD3241 300 mg AZD3241 600 mg
    Started
    16
    14
    17
    Completed
    15
    13
    17
    Not completed
    1
    1
    0
         Adverse event, non-fatal
    1
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Placebo to match AZD3241 dosed twice daily

    Reporting group title
    AZD3241 300 mg
    Reporting group description
    AZD3241 300 mg dosed twice daily

    Reporting group title
    AZD3241 600 mg
    Reporting group description
    AZD3241 600 mg dosed twice daily

    Reporting group values
    Placebo AZD3241 300 mg AZD3241 600 mg Total
    Number of subjects
    19 19 20 58
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    14 15 15 44
        From 65-84 years
    5 4 5 14
        85 years and over
    0 0 0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    59.3 ± 7.9 59.9 ± 6.1 58 ± 8.5 -
    Gender, Male/Female
    Units: Subjects
        Female
    7 5 5 17
        Male
    12 14 15 41
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0 0
        Asian
    0 1 2 3
        Native Hawaiian or Other Pacific Islander
    0 0 0 0
        Black or African American
    1 0 0 1
        White
    16 15 16 47
        More than one race
    0 0 0 0
        Unknown or Not Reported
    2 3 2 7
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    1 0 0 1
        Not Hispanic or Latino
    16 16 18 50
        Unknown or Not Reported
    2 3 2 7
    Multiple System Atropy Subtype
    Multiple System Atropy (MSA) Subtype, Parkinsonian (P) or Cerebellar (C)
    Units: Subjects
        MSA - P
    7 10 7 24
        MSA - C
    12 9 13 34
    Diagnostic Category
    Diagnostic Category for Multiple System Atropy (MSA); Possible or Probable were the eligible categories
    Units: Subjects
        Possible MSA
    1 4 8 13
        Probable MSA
    18 15 12 45
    Genotype
    Translocator Protein binding (TSPO) : genotype (affinity for binding); Low Affinity Binding was exclusionary
    Units: Subjects
        TSPO - High Affinity Binding
    12 10 16 38
        TSPO - Mixed Affinity Binding
    7 9 4 20
        TSPO- Low Affinity Binding
    0 0 0 0
    Subject analysis sets

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Subjects randomised who took at least one dose of study medication (1 subject in the Placebo, not included, withdrawn before first dose)

    Subject analysis set title
    PET Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Subjects with PET scan at baseline and at week 12

    Subject analysis sets values
    Safety Analysis Set PET Analysis Set
    Number of subjects
    58
    43
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    44
    33
        From 65-84 years
    14
    10
        85 years and over
    0
    0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    59 ± 7.5
    60.5 ± 7.8
    Gender, Male/Female
    Units: Subjects
        Female
    17
    13
        Male
    41
    30
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0
    0
        Asian
    3
    2
        Native Hawaiian or Other Pacific Islander
    0
    0
        Black or African American
    1
    0
        White
    47
    36
        More than one race
    0
    0
        Unknown or Not Reported
    7
    5
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    1
    1
        Not Hispanic or Latino
    50
    37
        Unknown or Not Reported
    7
    5
    Multiple System Atropy Subtype
    Multiple System Atropy (MSA) Subtype, Parkinsonian (P) or Cerebellar (C)
    Units: Subjects
        MSA - P
    34
    26
        MSA - C
    24
    17
    Diagnostic Category
    Diagnostic Category for Multiple System Atropy (MSA); Possible or Probable were the eligible categories
    Units: Subjects
        Possible MSA
    13
    8
        Probable MSA
    45
    35
    Genotype
    Translocator Protein binding (TSPO) : genotype (affinity for binding); Low Affinity Binding was exclusionary
    Units: Subjects
        TSPO - High Affinity Binding
    38
    29
        TSPO - Mixed Affinity Binding
    20
    14
        TSPO- Low Affinity Binding
    0
    0

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Placebo to match AZD3241 dosed twice daily

    Reporting group title
    AZD3241 300 mg
    Reporting group description
    AZD3241 300 mg dosed twice daily

    Reporting group title
    AZD3241 600 mg
    Reporting group description
    AZD3241 600 mg dosed twice daily
    Reporting group title
    Placebo
    Reporting group description
    Placebo to match AZD3241 dosed twice daily

    Reporting group title
    AZD3241 300 mg
    Reporting group description
    AZD3241 300 mg dosed twice daily

    Reporting group title
    AZD3241 600 mg
    Reporting group description
    AZD3241 600 mg dosed twice daily
    Reporting group title
    Placebo
    Reporting group description
    Placebo to match AZD3241 dosed twice daily

    Reporting group title
    AZD3241 300 mg
    Reporting group description
    AZD3241 300 mg dosed twice daily

    Reporting group title
    AZD3241 600 mg
    Reporting group description
    AZD3241 600 mg dosed twice daily

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Subjects randomised who took at least one dose of study medication (1 subject in the Placebo, not included, withdrawn before first dose)

    Subject analysis set title
    PET Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Subjects with PET scan at baseline and at week 12

    Primary: Striatum Brain region: Change from baseline in microglia activation via Positron Emission Tomography(PET)

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    End point title
    Striatum Brain region: Change from baseline in microglia activation via Positron Emission Tomography(PET)
    End point description
    Striatum Brain region: Change from baseline in microglia activation via PET By [11C]PBR28 binding to translocator protein (looking for decrease in total distribution volume)
    End point type
    Primary
    End point timeframe
    Baseline (pre randomization) and Week 12
    End point values
    Placebo AZD3241 300 mg AZD3241 600 mg PET Analysis Set
    Number of subjects analysed
    16
    14
    17
    43
    Units: Total distribution Volume (VT)
        arithmetic mean (standard deviation)
    -0.26 ± 0.75
    -0.08 ± 0.84
    -0.31 ± 1.37
    -0.25 ± 1.09
    Statistical analysis title
    Placebo change from baseline
    Statistical analysis description
    Change from baseline within treatment arm compared to 0
    Comparison groups
    Placebo v PET Analysis Set
    Number of subjects included in analysis
    59
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.13 [1]
    Method
    ANOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.38
    Confidence interval
         level
    90%
         sides
    1-sided
         lower limit
    -
         upper limit
    0.03
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    Notes
    [1] - Not adjusted for multiple comparisons, alpha=0.10
    Statistical analysis title
    AZD3241 300 mg change from baseline
    Statistical analysis description
    Change from baseline within treatment compared to 0
    Comparison groups
    AZD3241 300 mg v PET Analysis Set
    Number of subjects included in analysis
    57
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.91 [2]
    Method
    ANOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.03
    Confidence interval
         level
    90%
         sides
    1-sided
         lower limit
    -
         upper limit
    0.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.25
    Notes
    [2] - not adjusted
    Statistical analysis title
    AZD3241 600 mg change from baseline
    Statistical analysis description
    Change from baseline within treatment compared to 0
    Comparison groups
    AZD3241 600 mg v PET Analysis Set
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.68
    Method
    ANOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    -0.1
    Confidence interval
         level
    90%
         sides
    1-sided
         lower limit
    -
         upper limit
    0.31
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.25
    Statistical analysis title
    AZD3241 300 mg compared to Placebo
    Statistical analysis description
    Secondary objective - comparison to placebo
    Comparison groups
    Placebo v AZD3241 300 mg v PET Analysis Set
    Number of subjects included in analysis
    73
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.32 [3]
    Method
    ANOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    0.35
    Confidence interval
         level
    90%
         sides
    2-sided
         lower limit
    -0.24
         upper limit
    1.02
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.34
    Notes
    [3] - no adjustment
    Statistical analysis title
    AZD3241 600 mg comparison to Placebo
    Statistical analysis description
    Secondary objective - comparison to placebo
    Comparison groups
    Placebo v AZD3241 600 mg v PET Analysis Set
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.45 [4]
    Method
    ANOVA
    Parameter type
    Mean difference (final values)
    Point estimate
    0.28
    Confidence interval
         level
    90%
         sides
    2-sided
         lower limit
    -0.39
         upper limit
    0.92
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.36
    Notes
    [4] - no adjustment

    Secondary: Myeloperoxidase (MPO) inhibition in plasma (change from baseline), specific activity

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    End point title
    Myeloperoxidase (MPO) inhibition in plasma (change from baseline), specific activity
    End point description
    Myeloperoxidase (MPO) inhibition in plasma (change from baseline), on samples collected and analyzed, specific activity (activity/protein)
    End point type
    Secondary
    End point timeframe
    Baseline (Day -1) and week 12
    End point values
    Placebo AZD3241 300 mg AZD3241 600 mg
    Number of subjects analysed
    12
    9
    15
    Units: ratio
    arithmetic mean (standard deviation)
        Pre dose (week 12)
    -0.05 ± 0.15
    -0.12 ± 0.11
    -0.1 ± 0.2
        1 hour post dose (week 12)
    0.08 ± 0.28
    -0.18 ± 0.24
    0.19 ± 1.3
        2 to 6 hours post dose (week 12)
    0.03 ± 0.23
    -0.19 ± 0.24
    -0.15 ± 0.14
    No statistical analyses for this end point

    Other pre-specified: Exploratory efficacy: Unified Multiple System Atropy Rating Scale, change from baseline (Total Score, Part 1 + Part 2)

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    End point title
    Exploratory efficacy: Unified Multiple System Atropy Rating Scale, change from baseline (Total Score, Part 1 + Part 2)
    End point description
    Exploratory efficacy: Unified Multiple System Atropy Rating Scale, change from baseline (total Score, Part 1 + Part 2) : Score range 0 to 104, positive value indicates worsening symptoms
    End point type
    Other pre-specified
    End point timeframe
    Baseline to final treatment visit (early termination visit for those not completing week 12)
    End point values
    Placebo AZD3241 300 mg AZD3241 600 mg
    Number of subjects analysed
    17
    17
    18
    Units: Score
        arithmetic mean (standard deviation)
    4.59 ± 4.46
    3.71 ± 4.98
    2.56 ± 6.15
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    12 weeks of randomized treation plus a follow up period up to 14 days after last dose
    Adverse event reporting additional description
    Adverse events starting during screening period Before randomization or starting greater that 14 days after the last dose of study medication are not included in these tabulations.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Placebo to match AZD3241 dosed twice daily

    Reporting group title
    AZD3241 300 mg
    Reporting group description
    AZD3241 300 mg dosed twice daily

    Reporting group title
    AZD3241 600 mg
    Reporting group description
    AZD3241 600 mg dosed twice daily

    Serious adverse events
    Placebo AZD3241 300 mg AZD3241 600 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         number of deaths (all causes)
    1
    0
    1
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Hip fracture
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Choking
    Additional description: Subject found dead with cheese in throat
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Nervous system disorders
    Multipla System Atrophy
    Additional description: Subject found face down on bedding - considered disease related death
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    Placebo AZD3241 300 mg AZD3241 600 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 19 (73.68%)
    15 / 19 (78.95%)
    15 / 20 (75.00%)
    Social circumstances
    Alcohol use
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    2
    General disorders and administration site conditions
    Adverse drug reaction
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Discomfort
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 19 (5.26%)
    3 / 19 (15.79%)
    3 / 20 (15.00%)
         occurrences all number
    1
    3
    4
    Feeling jittery
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Influenza like illness
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Gait disturbance
         subjects affected / exposed
    2 / 19 (10.53%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    4
    0
    0
    Pain
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Depressed mood
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Depression
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Depressive symptom
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Abnormal dreams
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Confusional state
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Disorientation
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Panic attack
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Suicidal ideation
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Injury, poisoning and procedural complications
    Administration related reaction
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Fall
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 19 (5.26%)
    4 / 20 (20.00%)
         occurrences all number
    12
    1
    6
    Thermal burn
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Procedural pain
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Investigations
    Lymphocyte count decreased
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Weight decreased
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    1
    1
    0
    Weight increased
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Cardiac disorders
    atrial fibrilation
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 19 (10.53%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    2
    0
    1
    Dyspnoea
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Sleep apnoe syndrome
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    1
    1
    0
    Clumsiness
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Dizziness
         subjects affected / exposed
    2 / 19 (10.53%)
    2 / 19 (10.53%)
    2 / 20 (10.00%)
         occurrences all number
    2
    2
    4
    Dysarthria
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Headache
         subjects affected / exposed
    4 / 19 (21.05%)
    3 / 19 (15.79%)
    2 / 20 (10.00%)
         occurrences all number
    5
    6
    4
    Hypokinesia
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    Migraine with aura
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Lethargy
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Presyncope
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    2 / 20 (10.00%)
         occurrences all number
    0
    2
    3
    Radicular pain
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Restless leg syndrome
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Somnolence
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    0
    1
    Syncope
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    4
    Eye disorders
    Diplopia
         subjects affected / exposed
    0 / 19 (0.00%)
    2 / 19 (10.53%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Vertigo
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    1
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Constipation
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    0
    1
    Diarrhoea
         subjects affected / exposed
    2 / 19 (10.53%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         occurrences all number
    2
    1
    2
    Dysphagia
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 19 (5.26%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    1
    Salivary hypersecretion
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Toothache
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Nausea
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Skin discolouration
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Skin lesion
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    2
    Skin necrosis
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Joint swelling
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    3
    0
    1
    Back pain
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    0
    1
    Muscle fatigue
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    2 / 20 (10.00%)
         occurrences all number
    0
    1
    2
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    2
    Myalgia
         subjects affected / exposed
    0 / 19 (0.00%)
    0 / 19 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    0
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    4
    0
    Rhinitis
         subjects affected / exposed
    0 / 19 (0.00%)
    1 / 19 (5.26%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 19 (5.26%)
    0 / 19 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    Urinary tract infection
         subjects affected / exposed
    3 / 19 (15.79%)
    5 / 19 (26.32%)
    3 / 20 (15.00%)
         occurrences all number
    3
    5
    3

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    29 Jan 2015
    Changed designation of Investigational product manufacturer; Updated Reason for discontinuing study drug on study withdrawal (implimented before first patient in)
    28 May 2015
    Amended options for Data Safety Monitoring Board recommendations based on unblinded interim analysis; Updated prohibitied medication information

    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.
    Although 59 subjects entered, 1 notified immediately as not eligible, thus only 58 are included in safety analysis set. Biomarker samples included if testing done within 6 months of collection per protocol although during study expiry changed.
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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