Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43861   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging, Parallel Group Study to Evaluate Safety, Tolerability, and Pharmacodynamics of PF-05221304 Administered Daily for 16-Weeks to Adult Subjects With Nonalcoholic Fatty Liver Disease

    Summary
    EudraCT number
    		 2017-001156-55
    Trial protocol
    		 PL  
    Global end of trial date
    27 Mar 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Jan 2021
    First version publication date
    29 Jan 2021
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    C1171002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Pfizer, Inc
    Sponsor organisation address
    235 E 42nd Street, New York, United States, NY 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc, +1 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc, +1 8007181021, ClinicalTrials.gov_Inquiries@pfizer.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
    14 Oct 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Feb 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study was designed as a dose-ranging trial with placebo and 4 active doses of PF-05221304 to assess the safety, tolerability and the effect of PF-05221304 on liver fat.
    Protection of trial subjects
    This study was conducted in compliance with the ethical principles originating in or derived from the Declaration of Helsinki and in compliance with all International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guidelines. In addition, all local regulatory requirements were followed, in particular, those affording greater protection to the safety of trial subjects.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Aug 2017
    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
    Australia: 11
    Country: Number of subjects enrolled
    Canada: 56
    Country: Number of subjects enrolled
    Israel: 25
    Country: Number of subjects enrolled
    Poland: 69
    Country: Number of subjects enrolled
    Taiwan: 6
    Country: Number of subjects enrolled
    United States: 138
    Worldwide total number of subjects
    305
    EEA total number of subjects
    69
    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)
    242
    From 65 to 84 years
    63
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This was a randomized, double-blind, placebo-controlled, 5 arm (placebo, plus 4 active doses of PF-05221304), parallel group study. A total of 305 subjects were assigned to study treatment.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo matched to PF-05221304 tablet was administered orally once daily (QD) for up to 16 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo matching PF-05221304 tablet strengths of 1 mg and 5 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    2 tablets orally QD for up to 16 weeks.

    Investigational medicinal product name
    Placebo matching PF-05221304 tablet strengths of 25 mg and 50 mg.
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 tablet orally QD for up to 16 weeks.

    Arm title
    		 PF-05221304 2 mg
    Arm description
    		 PF-05221304 tablet was administered orally at 2 mg QD for up to 16 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    PF-05221304
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg PF-05221304 tablet 2 tables orally QD for up to 16 weeks.

    Arm title
    		 PF-05221304 10 mg
    Arm description
    		 PF-05221304 tablet was administered orally at 10 mg QD for up to 16 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    PF-05221304
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    5 mg PF-05221304 tablet 2 tablets orally QD for up to 16 weeks.

    Arm title
    		 PF-05221304 25 mg
    Arm description
    		 PF-05221304 tablet was administered orally at 25 mg QD for up to 16 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    PF-05221304
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    25 mg PF-05221304 tablet 1 tablet orally QD for up to 16 weeks.

    Arm title
    		 PF-05221304 50 mg
    Arm description
    		 PF-05221304 tablet was administered orally at 50 mg QD for up to 16 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    PF-05221304
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    50 mg PF-05221304 tablet 1 tablet orally QD for up to 16 weeks.

    Number of subjects in period 1
    		Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Started
    61
    63
    62
    58
    61
    Completed
    54
    58
    55
    48
    48
    Not completed
    7
    5
    7
    10
    13
         Consent withdrawn by subject
    2
    2
    3
    4
    2
         Adverse event, non-fatal
    3
    3
    2
    6
    9
         Lost to follow-up
    -
    -
    -
    -
    1
         Protocol deviation
    2
    -
    2
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 -

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 305 305
    Age Categorical
    Units: Subjects
        Adults (18-44 years)
    		 65 65
        Adults (45-64 years)
    		 177 177
        Adults (>=65 years)
    		 63 63
    Age Continuous
    Units: years
        geometric mean (standard deviation)
    		 53.38 ( 11.99 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 171 171
        Male
    		 134 134
    Race/Ethnicity
    Units: Subjects
        White
    		 252 252
        Black or African American
    		 4 4
        Asian
    		 38 38
        American Indian or Alaska Native
    		 1 1
        Native Hawaiian or Other Pacific Islander
    		 1 1
        Not Reported
    		 9 9
    Subject analysis sets

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 2 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 10 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 25 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 50 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis sets values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects
    61
    63
    62
    58
    61
    Age Categorical
    Units: Subjects
        Adults (18-44 years)
    12
    10
    18
    12
    13
        Adults (45-64 years)
    40
    41
    29
    34
    33
        Adults (>=65 years)
    9
    12
    15
    12
    15
    Age Continuous
    Units: years
        geometric mean (standard deviation)
    53.33 ( 10.77 )
    54.10 ( 11.86 )
    52.66 ( 12.75 )
    54.02 ( 11.58 )
    52.82 ( 13.12 )
    Gender Categorical
    Units: Subjects
        Female
    36
    37
    33
    35
    30
        Male
    25
    26
    29
    23
    31
    Race/Ethnicity
    Units: Subjects
        White
    53
    50
    52
    46
    51
        Black or African American
    1
    1
    1
    1
    0
        Asian
    5
    7
    7
    9
    10
        American Indian or Alaska Native
    0
    1
    0
    0
    0
        Native Hawaiian or Other Pacific Islander
    0
    1
    0
    0
    0
        Not Reported
    2
    3
    2
    2
    0

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo matched to PF-05221304 tablet was administered orally once daily (QD) for up to 16 weeks.

    Reporting group title
    PF-05221304 2 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 2 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 10 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 10 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 25 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 25 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 50 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 50 mg QD for up to 16 weeks.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 2 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 10 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 25 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Subject analysis set title
    PF-05221304 50 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects who received PF-05221304 tablet orally at 2/10/25/50 mg QD for up to 16 weeks.

    Primary: Percent Change from Baseline in Liver Fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI- PDFF) at Week 16

    		 Close Top of page
    End point title
    		 Percent Change from Baseline in Liver Fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI- PDFF) at Week 16
    End point description
    MRI-PDFF utilized a gradient echo sequence with low flip angle (FA) to minimize T1 bias, corrected T2* decay (due to iron overload) via modeling of the fat signal as a superposition of multiple frequency components from 5 different lipid types, and was applied in each of the 9 Couinaud segments. This technique improved fat quantification accuracy for the entire liver permitting quantification of small differences/changes following pharmacological intervention. All randomized subjects who received at least 1 dose of randomized study treatment and with non-missing baseline and post-baseline endpoint.
    End point type
    Primary
    End point timeframe
    Baseline (between Day -14 and Day 1), Week 16
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    55
    59
    57
    54
    51
    Units: Percent change
        least squares mean (confidence interval 80%)
    -7.2 (-13.9 to 0.0)
    -17.1 (-22.7 to -11.1)
    -49.9 (-53.3 to -46.2)
    -55.9 (-59.0 to -52.4)
    -64.8 (-67.5 to -62.0)
    Statistical analysis title
    		 Placebo, PF-05221304
    Comparison groups
    Placebo v PF-05221304 2 mg
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -10.7
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -19.4
         upper limit
    		 -1.1
    Statistical analysis title
    		 Placebo, PF-05221304 10 mg
    Comparison groups
    Placebo v PF-05221304 10 mg
    Number of subjects included in analysis
    112
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -46
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -51.3
         upper limit
    		 -40.1
    Statistical analysis title
    		 Placebo, PF-05221304 25 mg
    Comparison groups
    Placebo v PF-05221304 25 mg
    Number of subjects included in analysis
    109
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -52.4
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -57.2
         upper limit
    		 -47.1
    Statistical analysis title
    		 Placebo, PF-05221304 50 mg
    Comparison groups
    Placebo v PF-05221304 50 mg
    Number of subjects included in analysis
    106
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -62.1
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -66
         upper limit
    		 -57.8

    Secondary: Percent Change from Baseline in Alanine Aminotransferase at Week 16

    		 Close Top of page
    End point title
    		 Percent Change from Baseline in Alanine Aminotransferase at Week 16
    End point description
    Potential improvement in liver function was denoted by reduction in alanine transaminase (ALT). All randomized subjects who received at least 1 dose of randomized study treatment and diagnosed/presumed with nonalcoholic steatohepatitis.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1 pre-dose), Week 16
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    40
    42
    42
    39
    40
    Units: Percent change
        least squares mean (confidence interval 80%)
    -8.5 (-15.2 to -1.2)
    -12.5 (-18.7 to -5.8)
    -27.7 (-32.9 to -22.2)
    -31.3 (-36.6 to -25.5)
    -46.8 (-50.8 to -42.4)
    Statistical analysis title
    		 Placebo, PF-05221304 2 mg
    Comparison groups
    Placebo v PF-05221304 2 mg
    Number of subjects included in analysis
    82
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -4.4
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -14
         upper limit
    		 6.3
    Statistical analysis title
    		 Placebo, PF-05221304 10 mg
    Comparison groups
    Placebo v PF-05221304 10 mg
    Number of subjects included in analysis
    82
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -21
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -29
         upper limit
    		 -12.2
    Statistical analysis title
    		 Placebo, PF-05221304 25 mg
    Comparison groups
    Placebo v PF-05221304 25 mg
    Number of subjects included in analysis
    79
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -25
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -32.8
         upper limit
    		 -16.1
    Statistical analysis title
    		 Placebo, PF-05221304 50 mg
    Comparison groups
    Placebo v PF-05221304 50 mg
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (net)
    Point estimate
    -41.8
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -47.9
         upper limit
    		 -35

    Secondary: Number of Subjects With Treatment-Emergent Adverse Events

    		 Close Top of page
    End point title
    		 Number of Subjects With Treatment-Emergent Adverse Events
    End point description
    An adverse event (AE) was any untoward medical occurrence in a study subject administered a product or medical device. A serious AE (SAE) is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; lifethreatening; initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect. Any such events with initial onset or increasing in severity after the first dose of study treatment were counted as treatment-emergent. All randomized subjects who received at least 1 dose of randomized study treatment.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) up to Week 20
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    61
    63
    62
    58
    61
    Units: Subjects
        All-causality AE
    41
    40
    42
    45
    40
        All-causality SAE
    0
    1
    1
    2
    2
        Treatment-related AE
    16
    9
    12
    16
    23
        Treatment-related SAE
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects With Laboratory Abnormalities

    		 Close Top of page
    End point title
    		 Number of Subjects With Laboratory Abnormalities
    End point description
    Following laboratory parameters were assessed against pre-defined abnormality criteria: hematology (hemoglobin, hematocrit, erythrocytes, reticulocytes, platelets, leukocytes, lymphocytes, neutrophils, basophils, eosinophils, monocytes, activated partial thromboplastin time, prothrombin time [PT], PT/international normalized ratio, reticulocytes); chemistry (indirect bilirubin, direct bilirubin, protein, albumin, blood urea nitrogen, creatinine, creatine kinase, urate, calcium, sodium, potassium, chloride, bicarbonate, urine urobilinogen); urinalysis (pH, urine glucose, urine ketones, urine protein, urine hemoglobin, nitrites, leukocyte esterase, urine erythrocytes, urine leukocytes, urine hyaline casts, urine bilirubin, granular casts). All randomized subjects who received at least 1 dose of randomized study treatment and had laboratory data.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) up to Week 20
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    59
    63
    62
    58
    61
    Units: Subjects
        With Laboratory Abnormalities
    39
    44
    36
    33
    40
    No statistical analyses for this end point

    Secondary: Number of Subjects With Vital Signs Data Meeting Predefined Criteria

    		 Close Top of page
    End point title
    		 Number of Subjects With Vital Signs Data Meeting Predefined Criteria
    End point description
    Vital signs categorical summarization criteria: 1) sitting systolic blood pressure (SBP) <90 or >180 millimeters of mercury (mmHg); 2) sitting diastolic blood pressure (DBP) <50 mmHg or >110 mmHg; 3) sitting pulse rate <40 or >120 beats per minute (bpm); 4) change from baseline (increase or decrease) in sitting DBP greater than or equal to (>=) 20 mmHg; 5) change from baseline (increase or decrease) in sitting SBP >=30 mmHg. All randomized subjects who received at least 1 dose of randomized study treatment and had vital signs data.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) up to Week 18
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    60
    63
    62
    58
    61
    Units: Subjects
        Sitting SBP <90 mmHg
    0
    0
    0
    0
    2
        Sitting SBP >180 mmHg
    0
    0
    0
    1
    0
        Sitting SBP increase >=30 mmHg
    5
    6
    2
    2
    0
        Sitting SBP decrease >=30 mmHg
    2
    1
    5
    7
    6
        Sitting DBP >110 mmHg
    0
    0
    0
    0
    1
        Sitting DBP increase >=20 mmHg
    1
    4
    2
    2
    3
        Sitting DBP decrease >=20 mmHg
    0
    4
    3
    4
    4
        Sitting pulse rate <40 bpm
    0
    0
    0
    0
    0
        Sitting pulse rate >120 bpm
    0
    0
    0
    0
    0
        Sitting DBP <50 mmHg
    1
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects With 12-Lead Electrocardiogram (ECG) Data Meeting Predefined Criteria

    		 Close Top of page
    End point title
    		 Number of Subjects With 12-Lead Electrocardiogram (ECG) Data Meeting Predefined Criteria
    End point description
    ECG categorical summarization criteria 1.QRS interval (time from ECG Q wave to end of S wave corresponding to ventricle depolarization) >=140msec 2.QRS interval >=50% change from baseline 3.PR interval (interval between start of P wave and start of QRS complex, corresponding to time between onset of atrial depolarization and onset of ventricular depolarization) >=300msec 4.PR interval >=25% change when baseline is >200msec or >=50% change when baseline is <=200msec 5.QT interval (time from ECG Q wave to end of T wave corresponding to electrical systole): absolute value of >=500msec 6.QTcF interval (QT corrected for heart rate using Fridericia’s formula) absolute value of 450 to <480msec 7.QTcF interval: absolute value of 480 to <500msec 8.QTcF interval: absolute value >=500msec 9.QTcF interval: a change from baseline of 30 to <60msec 10.QTcF interval: a change from baseline >=60 msec. All randomized subjects who received at least 1 dose of randomized study treatment and had ECG data
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) up to Week 18
    End point values
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Number of subjects analysed
    60
    63
    61
    58
    60
    Units: Subjects
        PR interval >=300 msec
    0
    0
    0
    0
    0
        %Change in PR interval >=25/50%
    0
    1
    0
    1
    1
        QRS interval >=140 msec
    0
    0
    1
    0
    0
        %Change in QRS interval >=50%
    0
    0
    0
    0
    0
        QT interval >=500 msec
    0
    0
    0
    1
    0
        QTcF interval >=450 to <480 msec
    6
    10
    7
    9
    3
        QTcF interval >=480 to <500 msec
    0
    1
    0
    1
    1
        QTcF interval >=500 msec
    0
    0
    0
    0
    0
        QTcF interval increase >=30 to 60 msec
    5
    6
    8
    10
    4
        QTcF interval increase >=60 msec
    2
    1
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study treatment up to 20 weeks.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo matched to PF-05221304 tablet was administered orally once daily (QD) for up to 16 weeks.

    Reporting group title
    PF-05221304 2 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 2 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 10 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 10 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 25 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 25 mg QD for up to 16 weeks.

    Reporting group title
    PF-05221304 50 mg
    Reporting group description
    		 PF-05221304 tablet was administered orally at 50 mg QD for up to 16 weeks.

    Serious adverse events
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    1 / 62 (1.61%)
    2 / 58 (3.45%)
    2 / 61 (3.28%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Rib fissure
    Additional description: Rib fracture
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    1 / 62 (1.61%)
    0 / 58 (0.00%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Angina
    Additional description: Angina unstable
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 62 (0.00%)
    1 / 58 (1.72%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocardial infarct
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    0 / 62 (0.00%)
    0 / 58 (0.00%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocardial ischemia
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 62 (0.00%)
    1 / 58 (1.72%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthmatic
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 62 (0.00%)
    0 / 58 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal colic
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 62 (0.00%)
    0 / 58 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    pneumonia
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    0 / 62 (0.00%)
    0 / 58 (0.00%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    upper respiratory tract infection
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    1 / 62 (1.61%)
    0 / 58 (0.00%)
    0 / 61 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo PF-05221304 2 mg PF-05221304 10 mg PF-05221304 25 mg PF-05221304 50 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    27 / 61 (44.26%)
    21 / 63 (33.33%)
    25 / 62 (40.32%)
    31 / 58 (53.45%)
    29 / 61 (47.54%)
    Investigations
    Blood triglycerides increased
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    1 / 62 (1.61%)
    3 / 58 (5.17%)
    2 / 61 (3.28%)
         occurrences all number
    0
    0
    1
    3
    3
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    4 / 61 (6.56%)
    3 / 63 (4.76%)
    1 / 62 (1.61%)
    3 / 58 (5.17%)
    3 / 61 (4.92%)
         occurrences all number
    4
    3
    2
    3
    3
    Headache
         subjects affected / exposed
    8 / 61 (13.11%)
    3 / 63 (4.76%)
    3 / 62 (4.84%)
    7 / 58 (12.07%)
    4 / 61 (6.56%)
         occurrences all number
    10
    3
    4
    7
    5
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    5 / 61 (8.20%)
    3 / 63 (4.76%)
    2 / 62 (3.23%)
    2 / 58 (3.45%)
    2 / 61 (3.28%)
         occurrences all number
    5
    3
    2
    2
    2
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 63 (0.00%)
    1 / 62 (1.61%)
    6 / 58 (10.34%)
    1 / 61 (1.64%)
         occurrences all number
    1
    0
    1
    7
    1
    Diarrhoea
         subjects affected / exposed
    3 / 61 (4.92%)
    3 / 63 (4.76%)
    8 / 62 (12.90%)
    2 / 58 (3.45%)
    4 / 61 (6.56%)
         occurrences all number
    3
    4
    12
    4
    4
    Nausea
         subjects affected / exposed
    3 / 61 (4.92%)
    0 / 63 (0.00%)
    3 / 62 (4.84%)
    5 / 58 (8.62%)
    4 / 61 (6.56%)
         occurrences all number
    3
    0
    3
    6
    4
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 61 (1.64%)
    2 / 63 (3.17%)
    0 / 62 (0.00%)
    3 / 58 (5.17%)
    1 / 61 (1.64%)
         occurrences all number
    1
    2
    0
    3
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 61 (1.64%)
    1 / 63 (1.59%)
    3 / 62 (4.84%)
    4 / 58 (6.90%)
    0 / 61 (0.00%)
         occurrences all number
    1
    1
    3
    4
    0
    Muscle spasms
         subjects affected / exposed
    4 / 61 (6.56%)
    0 / 63 (0.00%)
    2 / 62 (3.23%)
    1 / 58 (1.72%)
    0 / 61 (0.00%)
         occurrences all number
    4
    0
    2
    1
    0
    Pain in extremity
         subjects affected / exposed
    2 / 61 (3.28%)
    3 / 63 (4.76%)
    4 / 62 (6.45%)
    2 / 58 (3.45%)
    1 / 61 (1.64%)
         occurrences all number
    2
    3
    4
    2
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    2 / 61 (3.28%)
    0 / 63 (0.00%)
    3 / 62 (4.84%)
    3 / 58 (5.17%)
    2 / 61 (3.28%)
         occurrences all number
    2
    0
    3
    3
    2
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 61 (3.28%)
    6 / 63 (9.52%)
    3 / 62 (4.84%)
    3 / 58 (5.17%)
    2 / 61 (3.28%)
         occurrences all number
    2
    8
    3
    3
    2
    Urinary tract infection
         subjects affected / exposed
    1 / 61 (1.64%)
    1 / 63 (1.59%)
    2 / 62 (3.23%)
    5 / 58 (8.62%)
    4 / 61 (6.56%)
         occurrences all number
    1
    1
    2
    5
    4
    Metabolism and nutrition disorders
    Hypertriglyceridaemia
         subjects affected / exposed
    2 / 61 (3.28%)
    1 / 63 (1.59%)
    6 / 62 (9.68%)
    4 / 58 (6.90%)
    10 / 61 (16.39%)
         occurrences all number
    2
    1
    6
    4
    12

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jul 2017
    This amendment was making substantial changes as requested by the United States Food and Drug Administration (US FDA) as part of their review of the original protocol submitted on 11May2017.
    03 Oct 2017
    This amendment was making the substantial changes to align with the Pfizer Enterprise-level revision to appropriate measures to prevent pregnancy in the population of childbearing potential enrolled who are sexually active and align with the Clinical Trial Facilitation Group (CTFG) 2014 European Guidance.

    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
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Thu Apr 25 20:10:58 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA