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    Clinical Trial Results:
    A Safety Pilot Study of Px-104 in non alcoholic fatty liver disease (NAFLD) patients

    Summary
    EudraCT number
    		 2013-002984-24
    Trial protocol
    		 AT  
    Global end of trial date
    07 Jan 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Jun 2016
    First version publication date
    23 Jun 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PHS-Px-104-II-01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Phenex Pharmaceuticals AG
    Sponsor organisation address
    Waldhofer Straße 104, Heidelberg, Germany, 69123
    Public contact
    Sponsor, Phenex Pharmaceuticals AG, +49 06221-65282-13, manfred.birkel@phenex-pharma.com
    Scientific contact
    Sponsor, Phenex Pharmaceuticals AG, +49 06221-65282-13, manfred.birkel@phenex-pharma.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
    07 Jan 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    07 Jan 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Jan 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Safety and tolerability assessment will be made by monitoring the subjects for adverse events and by interpreting the results of the ECGs, various laboratory tests (changes in ALT/AST) and the subjects’ diaries.
    Protection of trial subjects
    The following safety assessments were done to protect trial subjects: - Cardiovascular monitoring 12-lead ECG, continuous ECG monitoring 20–24 hours prior to the first dose administration and on Days 7, 14, 21 and 26 (+1) for 23–25 hours, measurement of blood pressure and pulse rate - Laboratory monitoring Hematology, coagulation, clinical chemistry, serology, urinanalysis
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Sep 2013
    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
    Austria: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    12
    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)
    10
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Recruitment of patients was done at the Department for Gastroenterology and Hepatology, General Hospital of Vienna, from 25.10.2013 (first patient in) until 12.11.2014 (last patient in). Adult patients with non-alcoholic fatty liver disease (NAFLD) were screened for eligibility after giving their written informed consent to the clinifal trial.

    Pre-assignment
    Screening details
    		 21 patients were screened for eligibility. 6 patients were considered as screening failures (according to inclusion/exclusion criteria). 7 patients were considered as drop outs during the conduct of the study. 3 of the 7 drop outs occured before receiving study drug. All in all 12 patients received study drug.

    Pre-assignment period milestones
    Number of subjects started
    		 21 [1]
    Number of subjects completed
    		 12

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 In-/Exclusion criteria not fulfilled/fulfilled: 6
    Reason: Number of subjects
    		 Organizational reasons: 3
    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: According to the clinical study protocol, 12 patients in 1 group were planned. Study participants who voluntarily withdraw consent (due to any other reason than an AE) or study drop-outs were replaced. Therefore 21 patients were screened but only 12 patients were enrolled in the study (= received study medication).
    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Treatment arm
    Arm description
    		 All patients enrolled in this study received the study medication; there was no randomization or blinding done.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Px104
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    5 mg Px-104 capsules were taken by the patients once a day for 28 days.

    Number of subjects in period 1
    		Treatment arm
    Started
    12
    Study completion
    8
    Received treatment
    12
    Completed
    8
    Not completed
    4
         Adverse event, non-fatal
    3
         Organizational reasons
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Treatment period
    Reporting group description
    		 -

    Reporting group values
    		 Treatment period Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 10 10
        From 65-84 years
    		 2 2
    Gender categorical
    Units: Subjects
        Female
    		 4 4
        Male
    		 8 8

    End points

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    End points reporting groups
    Reporting group title
    Treatment arm
    Reporting group description
    		 All patients enrolled in this study received the study medication; there was no randomization or blinding done.

    Primary: Number of AEs, SAEs, TEAEs

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    End point title
    		 Number of AEs, SAEs, TEAEs [1]
    End point description
    End point type
    Primary
    End point timeframe
    From Baseline to End of Study Visit.
    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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: number
    27
    No statistical analyses for this end point

    Primary: Changes in blood pressure (systolic) from baseline

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    End point title
    		 Changes in blood pressure (systolic) from baseline [2]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: mmHg
        arithmetic mean (standard deviation)
    1.3 ( 15.4204 )
    No statistical analyses for this end point

    Primary: Changes in blood pressure (diastolic) from baseline

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    End point title
    		 Changes in blood pressure (diastolic) from baseline [3]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: mmHg
        arithmetic mean (standard deviation)
    0.9 ( 10.82641 )
    No statistical analyses for this end point

    Primary: Pulse

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    End point title
    		 Pulse [4]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: bpm
        arithmetic mean (standard deviation)
    3.8 ( 9.46103 )
    No statistical analyses for this end point

    Primary: Body temperature

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    End point title
    		 Body temperature [5]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    Notes
    [5] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: °C
        arithmetic mean (standard deviation)
    -0.02 ( 0.38816 )
    No statistical analyses for this end point

    Primary: Occurence of VES

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    End point title
    		 Occurence of VES [6]
    End point description
    End point type
    Primary
    End point timeframe
    From screening to end of study
    Notes
    [6] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: number
    5
    No statistical analyses for this end point

    Primary: Change in QTc

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    End point title
    		 Change in QTc [7]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    Notes
    [7] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: ms
        arithmetic mean (standard deviation)
    7.81818 ( 23.31016 )
    No statistical analyses for this end point

    Primary: Change of ALT from baseline

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    End point title
    		 Change of ALT from baseline [8]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    Notes
    [8] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: U/l
        arithmetic mean (standard deviation)
    8.90909 ( 16.82531 )
    No statistical analyses for this end point

    Primary: Change of AST from baseline

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    End point title
    		 Change of AST from baseline [9]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    Notes
    [9] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: U/l
        arithmetic mean (standard deviation)
    3.72727 ( 7.44434 )
    No statistical analyses for this end point

    Primary: Change of Bilirubin from baseline

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    End point title
    		 Change of Bilirubin from baseline [10]
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to end of study
    Notes
    [10] - 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: All variables were presented using descriptive statistical techniques.
    End point values
    		 Treatment arm
    Number of subjects analysed
    12
    Units: mg/dl
        arithmetic mean (standard deviation)
    0.03889 ( 0.23677 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From baseline to end of study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18
    Reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Serious adverse events
    		 Overall trial
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Overall trial
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 12 (91.67%)
    Cardiac disorders
    Palpitation
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Asymptomatic ventricular extrasystoles
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Extrasystoles
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    9
    Dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Shivering
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Heartburn
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    RUQ pain
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Diarrhea
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Obstipation
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Abdominal pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Facial swelling
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Exanthema
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infections and infestations
    Common cold
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Jan 2014
    The conduct of another 24h-ECG is optional at the screening visit; On days 1, 7, 14, 21 and 28 an additional urin test is done; Body weight will also be measured on days 1, 7, 14, 21 and 28.
    12 Mar 2014
    Additional exclusion criterion: Monomorphic or polymorphic ventricular ectopic beats ≥ 30 beats/ hours calculated as mean over the continuous ECG recording period; Additional stop criterion: ≥2 subjects experiencing premature ventricular ectopic beats ≥ 30 beats/hour calculated as mean over the continuous ECG recording period.

    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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