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    Clinical Trial Results:
    An explorative study to assess the safety, tolerability, and efficacy of AZD4831 in the treatment of pulmonary arterial hypertension (PAH) (MPO-PAH)

    Summary
    EudraCT number
    		 2020-002788-80
    Trial protocol
    		 DE  
    Global end of trial date
    14 Mar 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Jul 2023
    First version publication date
    15 Jul 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    Uni-Koeln-4243
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Universität zu Köln
    Sponsor organisation address
    Albertus-Magnus-Platz, Köln, Germany, 50923
    Public contact
    KKS Marburg, Koordinierungszentrum für Klinische Studien (KKS), info@kks.uni-marburg.de
    Scientific contact
    KKS Marburg, Koordinierungszentrum für Klinische Studien (KKS), info@kks.uni-marburg.de
    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
    06 Oct 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Dec 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Mar 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the efficacy of 12 weeks of AZD4831 in patients of two groups: 1) Patients with PAH (pre-capillary PH) who are on established therapy (at least 1 targeted PAH drug: ERA, PDE5i/sGC-S, PCA/PRA), and 2) Patients with post-capillary PH by measuring the change from baseline in pulmonary vascular resistance (PVR).
    Protection of trial subjects
    None
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Jul 2020
    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
    Germany: 15
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    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)
    9
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 15 patients have been registered for trail participation.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Treatment with AZD4831
    Arm description
    		 The AZD4831 will be administrated orally over a period of 12 weeks. The dose of study treatment is defined as 5 mg tablet once daily. Single-arm; no placebo.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AZD4831
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    The dose of study treatment is defined as 5 mg tablet once daily over a period of 12 weeks

    Number of subjects in period 1
    		Treatment with AZD4831
    Started
    15
    Completed
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 15 15
    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
        median (full range (min-max))
    		 61 (35 to 83) -
    Gender categorical
    Units: Subjects
        Female
    		 5 5
        Male
    		 10 10

    End points

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    End points reporting groups
    Reporting group title
    Treatment with AZD4831
    Reporting group description
    		 The AZD4831 will be administrated orally over a period of 12 weeks. The dose of study treatment is defined as 5 mg tablet once daily. Single-arm; no placebo.

    Subject analysis set title
    ITT-Analysis
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The intent-to-treat (ITT) population is defined as all patients enrolled, regardless of whether they actually received treatment.

    Primary: Pulmonary vascular resistance (PVR)

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    End point title
    		 Pulmonary vascular resistance (PVR)
    End point description
    Change from baseline to 12 weeks of treatment in pulmonary vascular resistance (PVR) as assessed by right heart catheterization
    End point type
    Primary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 Treatment with AZD4831 ITT-Analysis
    Number of subjects analysed
    15 [1]
    15 [2]
    Units: dyn×sec×cm−5
        median (confidence interval 95%)
    -2 (-3.5 to 0)
    -2 (-3.5 to 0)
    Attachments
    		 Boxplot of PVR (baseline and week 12)
    Notes
    [1] - All patients enrolled
    [2] - Intention-to-treat set equals per-protocol set
    Statistical analysis title
    		 Primary analysis
    Statistical analysis description
    Paired Wilcoxon signed-rank test at two-sided significance level 5%; moreover, a corresponding two-sided 95% confidence interval for the median difference is calculated
    Comparison groups
    Treatment with AZD4831 v ITT-Analysis
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.101
    Method
    		 Paired Wilcoxon signed-rank test
    Parameter type
    		 Median difference (final values)
    Point estimate
    -2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.5
         upper limit
    		 0

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Dec 11 2020-Aug 03 2021
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Group 1
    Reporting group description
    		 Patients with symptomatic PAH (WHO-FC II or III) who are on targeted medical therapy with at least one PAH-specific drug

    Reporting group title
    Group 2
    Reporting group description
    		 Patients with post-capillary PH

    Reporting group title
    All patients / ITT
    Reporting group description
    		 The intent-to-treat (ITT) population is defined as all patients enrolled, regardless of whether they actually received treatment.

    Serious adverse events
    		 Group 1 Group 2 All patients / ITT
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 10 (20.00%)
    2 / 5 (40.00%)
    4 / 15 (26.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Lower limb fracture
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 5 (20.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Syncope
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 5 (20.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0.01%
    Non-serious adverse events
    		 Group 1 Group 2 All patients / ITT
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 10 (50.00%)
    2 / 5 (40.00%)
    7 / 15 (46.67%)
    Investigations
    Blood pressure increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Blood iron decreased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Injury, poisoning and procedural complications
    Vaccination complication
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Atrial fibrillation
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 5 (20.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Palpitations
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Nervous system disorders
    Syncope
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Presyncope
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    2 / 10 (20.00%)
    1 / 5 (20.00%)
    3 / 15 (20.00%)
         occurrences all number
    2
    1
    3
    Infections and infestations
    Fungal infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 5 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 5 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    2
    Hyperkalaemia
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 5 (20.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Mar 2021
    An additional group of 5 patients (with post-capillary pulmonary hypertension) should be included to the study and the effect of AZD4831 on the endothelial dysfunction by these patients will be assessed via flow-mediated dilation. Also zymosan stimulation of plasma MPO should be done prior to the freezing of sample.

    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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