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    Clinical Trial Results:
    A Phase 2, 24-Week, Adaptive, Open Label, Sequential Cohort Trial To Evaluate The Efficacy, Safety, Tolerability and Pharmacokinetics of PF-06730512 Following Multiple Doses in Adult Subjects With Focal Segmental Glomerulosclerosis (FSGS)

    Summary
    EudraCT number
    2019-003607-35
    Trial protocol
    DE   SK   PL   CZ   IT  
    Global end of trial date
    14 Feb 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    17 Feb 2024
    First version publication date
    17 Feb 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    C0221002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03448692
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    ROBO2/PODO: C0221002
    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 Centre, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Centre, Pfizer Inc., 001 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
    25 Sep 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Feb 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of PF-06730512 compared to baseline in the reduction of proteinuria following 12 weeks of treatment in subjects with FSGS.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Council for Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trials subjects were followed.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    15 Oct 2018
    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
    Canada: 4
    Country: Number of subjects enrolled
    Czechia: 2
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Japan: 5
    Country: Number of subjects enrolled
    Mexico: 1
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Slovakia: 5
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 18
    Worldwide total number of subjects
    47
    EEA total number of subjects
    17
    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
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    A total of 47 subjects were enrolled into the study. All subjects received treatment.

    Period 1
    Period 1 title
    Disposition Phase:Screening:up to 43Days
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    PF-06730512 1000 mg IV
    Arm description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 1000 mg IV Q2W.

    Arm title
    PF-06730512 300 mg IV
    Arm description
    Subjects received PF-06730512 300 mg IV Q2W.
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 300 mg IV Q2W.

    Number of subjects in period 1
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Started
    23
    24
    Completed
    23
    24
    Period 2
    Period 2 title
    Lead in Period:8 Weeks (W)
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    PF-06730512 1000 mg IV
    Arm description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 1000 mg IV Q2W.

    Arm title
    PF-06730512 300 mg IV
    Arm description
    Subjects received PF-06730512 300 mg IV Q2W.
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 300 mg IV Q2W.

    Number of subjects in period 2
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Started
    23
    24
    Completed
    23
    24
    Period 3
    Period 3 title
    Investigational Treatment Period:Upto24W
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    PF-06730512 1000 mg IV
    Arm description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 1000 mg IV Q2W.

    Arm title
    PF-06730512 300 mg IV
    Arm description
    Subjects received PF-06730512 300 mg IV Q2W.
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 300 mg IV Q2W.

    Number of subjects in period 3
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Started
    23
    24
    Completed
    22
    15
    Not completed
    1
    9
         Consent withdrawn by subject
    -
    2
         Adverse event, non-fatal
    1
    -
         Study terminated by sponsor
    -
    5
         Lack of efficacy
    -
    1
         Physician's decision
    -
    1
    Period 4
    Period 4 title
    Follow up Period: 9 Weeks
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    PF-06730512 1000 mg IV
    Arm description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 1000 mg IV Q2W.

    Arm title
    PF-06730512 300 mg IV
    Arm description
    Subjects received PF-06730512 300 mg IV Q2W.
    Arm type
    Experimental

    Investigational medicinal product name
    PF-06730512
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received PF-06730512 300 mg IV Q2W.

    Number of subjects in period 4
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Started
    22
    15
    Completed
    23
    23
    Joined
    1
    8
         Continued follow-up
    1
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Disposition Phase:Screening:up to 43Days
    Reporting group description
    -

    Reporting group values
    Disposition Phase:Screening:up to 43Days Total
    Number of subjects
    47 47
    Age categorical
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    0 0
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    45 45
        From 65-84 years
    2 2
        85 years and over
    0 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    41.8 ± 14.01 -
    Sex: Female, Male
    Units: Subjects
        Female
    22 22
        Male
    25 25
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    1 1
        Asian
    8 8
        Native Hawaiian or Other Pacific Islander
    0 0
        Black or African American
    2 2
        White
    36 36
        More than one race
    0 0
        Unknown or Not Reported
    0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    8 8
        Not Hispanic or Latino
    39 39
        Unknown or Not Reported
    0 0

    End points

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    End points reporting groups
    Reporting group title
    PF-06730512 1000 mg IV
    Reporting group description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).

    Reporting group title
    PF-06730512 300 mg IV
    Reporting group description
    Subjects received PF-06730512 300 mg IV Q2W.
    Reporting group title
    PF-06730512 1000 mg IV
    Reporting group description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).

    Reporting group title
    PF-06730512 300 mg IV
    Reporting group description
    Subjects received PF-06730512 300 mg IV Q2W.
    Reporting group title
    PF-06730512 1000 mg IV
    Reporting group description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).

    Reporting group title
    PF-06730512 300 mg IV
    Reporting group description
    Subjects received PF-06730512 300 mg IV Q2W.
    Reporting group title
    PF-06730512 1000 mg IV
    Reporting group description
    Subjects received PF-06730512 1000 milligram (mg) intravenously (IV) every 2 weeks (Q2W).

    Reporting group title
    PF-06730512 300 mg IV
    Reporting group description
    Subjects received PF-06730512 300 mg IV Q2W.

    Primary: Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13

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    End point title
    Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13 [1]
    End point description
    UPCR is a ratio between two measured substances in urine: milligram of protein per millimole (mmol) of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function. The Full Analysis Set (FAS) was defined as all enrolled subjects who had received at least one dose of study treatment and had at least one post-baseline measurement of UPCR based on 24-hour urine collection. Here, "Number of Subjects Analysed" signifies subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline, Week 13
    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: Only descriptive analysis was planned for this endpoint.
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    21
    19
    Units: Percentage change
        least squares mean (confidence interval 90%)
    -12.283 (-26.096 to 4.112)
    -0.045 (-9.528 to 10.432)
    No statistical analyses for this end point

    Secondary: Number of Subjects With Abnormalities in Laboratory Test Parameters

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    End point title
    Number of Subjects With Abnormalities in Laboratory Test Parameters
    End point description
    Hemoglobin(Hg),hematocrit,erythrocytes:<0.8*lower limits of normal(LLN);platelets:<0.5*LLN>1.75*upper LN leukocytes(leu),glucose-fasting:<0.6*LLN>1.5*ULN;lymphocytes(lym),lym/leu, neutrophils(neu),neu/leu,protein,albumin,phosphate,free thyroxine,thyroid stimulating hormone: <0.8*LLN>1.2*ULN;basophils(bas),bas/leu,eosinophils(eos),eos/leu,monocytes(mon),mon/leu:>1.2*ULN;bilirubin (total, direct, indirect):>1.5*ULN;aspartate aminotransferase(AT),alanine AT,lactate dehydrogenase,alkaline phosphatase:>3.0*ULN;blood urea nitrogen,creatinine,cholesterol(total,LDL,HDL),triglycerides,Hg A1C: >1.3*ULN;sodium:<0.95*LLN>1.05*ULN;potassium,chloride,calcium,magnesium,bicarbonate:<0.9*LLN>1.1*ULN;prolactin:>1.1*ULN;creatine kinase:>2.0*ULN;urobilinogen:>=1;Urine-specific gravity:<1.003>1.030,pH:<4.5 >8, glucose,protein,bilirubin,nitrite,leukocyte esterase, ketones:>=1.Categories with at-least 1 non-zero values are reported. SAS population analysed. n= subjects evaluable for specific rows.
    End point type
    Secondary
    End point timeframe
    From Day 1 of treatment up to Week 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Subjects
        Hg<0.8*LLN (n=23,24)
    5
    4
        Hematocrit<0.8*LLN (n=23,24)
    5
    7
        Erythrocytes<0.8*LLN (n=23,24)
    3
    4
        Ery. Mean Corpuscular Volume < 0.9*LLN (n=23,24)
    1
    0
        Ery. Mean Corpuscular Hg<0.9*LLN (n=23,24)
    1
    0
        Platelets>1.75*ULN (n=23,24)
    1
    0
        Lym<0.8*LLN (n=23,24)
    0
    4
        Lym/Leu<0.8*LLN (n=23,24)
    1
    6
        Lym/Leu>1.2*ULN (n=23,24)
    0
    1
        Neu <0.8*LLN (n=23,24)
    1
    2
        Neu >1.2*ULN (n=23,24)
    3
    4
        Neu/Leu<0.8*LLN (n=23,24)
    1
    1
        Neu/Leu>1.2*ULN (n=23,24)
    0
    1
        Eos>1.2*ULN (n=23,24)
    2
    1
        Eos/Leu>1.2*ULN (n=23,24)
    3
    3
        Mon/Leu>1.2*ULN (n=23,24)
    0
    1
        Aspartate AT>3.0*ULN (n=23,23)
    1
    0
        Alanine AT>3.0*ULN (n=23,23)
    1
    0
        Protein<0.8*LLN (n=23,23)
    9
    11
        Albumin<0.8*LLN (n=23,23)
    8
    11
        Blood Urea Nitrogen>1.3*ULN (n=23,23)
    10
    8
        Creatinine>1.3*ULN (n=23,23)
    8
    12
        Urate>1.2*ULN (n=23,23)
    3
    2
        Cholesterol >1.3*ULN (n=23,23)
    6
    7
        HDL Cholesterol<0.8 *LLN (n=23,2)
    1
    0
        LDL Cholesterol>1.2*ULN (n=23,22)
    6
    5
        Triglycerides>1.3*ULN (n=23,23)
    6
    8
        Sodium<0.95*LLN (n=23,23)
    1
    0
        Potassium<0.9*LLN (n=23,23)
    1
    0
        Potassium>1.1*ULN (n=23,23)
    1
    0
        Calcium<0.9*LLN (n=23,23)
    1
    3
        Bicarbonate<0.9*LLN (n=23,23)
    0
    1
        Glucose>1.5*ULN (n=23,23)
    2
    2
        Specific Gravity>1.030 (n=21,24)
    5
    6
        pH>8 (n=23,24)
    1
    0
        Urine Glucose>=1 (n=23,24)
    5
    10
        Urine Protein>=1 (n=23,24)
    22
    24
        Urine Hemoglobin>=1 (n=23,24)
    15
    17
        Nitrite>=1 (n=23,24)
    1
    3
        Leukocyte Esterase>=1 (n=23,24)
    5
    6
        Urine Erythrocytes>=20 (n=22,24)
    3
    3
        Urine Leukocytes>=20 (n=22,24)
    2
    5
        Granular Casts>1 (n=5,2)
    4
    2
        Hyaline Casts>1 (n=18,18)
    17
    15
        Urine Creatinine<40(n=23,24)
    9
    8
        Urine Creatinine>300 (n=23,24)
    1
    0
        Urine (24HR) Creatinine>1.1*ULN (n=23,24)
    2
    6
    No statistical analyses for this end point

    Secondary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs)

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    End point title
    Number of Subjects With Treatment Emergent Adverse Events (TEAEs)
    End point description
    An adverse event was considered treatment emergent relative to a given treatment if the event occurred for the first time during the investigational treatment period and was not seen prior to the start of treatment (during the lead-in period), or the event was seen prior to the start of treatment but increased in severity during treatment. Adverse events occurring during the lead-in period were considered non-treatment emergent. Events that occurred during the follow-up period were counted as treatment emergent and attributed to the previous treatment taken. SAS was defined as all enrolled subjects who had received at least one dose of study treatment.
    End point type
    Secondary
    End point timeframe
    From Day 1 of treatment up to 9 weeks after last dose of study treatment (up to Week 33)
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Subjects
    20
    17
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Weight

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    End point title
    Change From Baseline in Body Weight
    End point description
    Change from baseline in body weight and at baseline values were reported for this endpoint. SAS was defined as all enrolled subjects who had received at least one dose of study treatment. Here, ‘Number Analysed’ (n) signifies number of subjects evaluable for the specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Kilogram
    arithmetic mean (standard deviation)
        Baseline (n=23,24)
    78.3 ± 17.8
    88.9 ± 23.5
        Week 2 (n=23,24)
    1.0 ± 2.2
    -0.3 ± 1.9
        Week 3 (n=23,23)
    0.4 ± 2.5
    -0.3 ± 2.2
        Week 5 (n=22,23)
    0.5 ± 3.0
    -0.5 ± 4.8
        Week 7 (n=23,22)
    0.6 ± 2.6
    -1.0 ± 5.3
        Week 9 (n=22,22)
    0.1 ± 3.1
    -0.4 ± 3.4
        Week 11 (n=22,20)
    0.7 ± 2.8
    -0.1 ± 4.2
        Week 13 (n=23,22)
    -0.2 ± 1.9
    -1.0 ± 3.4
        Week15 (n=4,13)
    -0.3 ± 1.0
    -0.7 ± 4.0
        Week 17 (n=4,9)
    -0.2 ± 1.2
    -1.2 ± 4.8
        Week 19 (n=4,9)
    -0.4 ± 1.0
    -1.3 ± 4.8
        Week 21 (n=4,9)
    0.3 ± 1.7
    -1.3 ± 4.9
        Week 23 (n=4,9)
    0.3 ± 1.2
    -1.5 ± 5.3
        Week 25 (n=4,16)
    -0.1 ± 1.6
    -1.1 ± 4.8
        Week 27 (n=4,16)
    0.7 ± 1.4
    -0.6 ± 5.2
        Week 29 (n=4,16)
    0.5 ± 1.5
    -1.0 ± 4.5
        Week 33 (n=4,16)
    0.5 ± 1.6
    -0.6 ± 5.4
    No statistical analyses for this end point

    Secondary: Change From Baseline in Blood Pressure

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    End point title
    Change From Baseline in Blood Pressure
    End point description
    Change from baseline in blood pressure and at baseline values were reported for this endpoint. SAS was defined as all enrolled subjects who had received at least one dose of study treatment. Here, ‘Number Analysed’ signifies number of subjects evaluable for the specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Millimetre of mercury (mmHg)
    arithmetic mean (standard deviation)
        Baseline (n=23,24)
    131.2 ± 12.8
    125.3 ± 11.8
        Week 2 (n=23,24)
    -1.8 ± 13.1
    -1.3 ± 7.8
        Week 3 (n=23,23)
    -1.3 ± 10.2
    1.7 ± 7.5
        Week 5 (n=22,23)
    -3.0 ± 10.6
    -1.9 ± 9.0
        Week 7 (n=23,22)
    -2.0 ± 11.3
    -1.2 ± 9.0
        Week 9 (n=22,22)
    -5.1 ± 10.7
    -2.6 ± 7.6
        Week 11 (n=22,20)
    -1.5 ± 12.5
    -1.6 ± 6.1
        Week 13 (n=23,22)
    -2.0 ± 11.3
    -1.0 ± 10.0
        Week15 (n=4,13)
    -7.5 ± 5.1
    -2.3 ± 6.2
        Week 17 (n=4,9)
    -7.3 ± 12.7
    -7.7 ± 12.2
        Week 19 (n=4,9)
    -8.0 ± 8.4
    0.4 ± 10.7
        Week 21 (n=4,9)
    -8.0 ± 16.4
    -5.8 ± 10.1
        Week 23 (n=4,9)
    1.0 ± 9.0
    -3.6 ± 8.2
        Week 25 (n=4,16)
    -9.0 ± 13.2
    -2.6 ± 11.2
        Week 27 (n=4,16)
    -3.0 ± 10.4
    3.5 ± 10.5
        Week 29 (n=4,16)
    -8.3 ± 8.4
    0.4 ± 11.2
        Week 33 (n=4,16)
    -12.0 ± 20.7
    4.6 ± 8.9
    No statistical analyses for this end point

    Secondary: Change From Baseline in Pulse Rate

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    End point title
    Change From Baseline in Pulse Rate
    End point description
    Change from baseline in pulse rate and at baseline values were reported for this endpoint. SAS was defined as all enrolled subjects who had received at least one dose of study treatment. Here, ‘Number Analysed’ signifies number of subjects evaluable for the specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Beats per minute
    arithmetic mean (standard deviation)
        Baseline (n=23,24)
    74.1 ± 11.8
    74.2 ± 11.0
        Week 2 (n=23,24)
    2.1 ± 9.6
    0.6 ± 6.9
        Week 3 (n=23,23)
    -0.6 ± 8.0
    -1.1 ± 6.0
        Week 5 (n=22,23)
    0.0 ± 7.3
    -0.3 ± 8.0
        Week 7 (n=23,22)
    -0.3 ± 9.2
    0.0 ± 7.9
        Week 9 (n=22,22)
    -1.0 ± 11.1
    1.5 ± 9.0
        Week 11 (n=22,20)
    -1.7 ± 10.8
    0.6 ± 6.6
        Week 13 (n=23,22)
    3.3 ± 11.9
    0.5 ± 6.9
        Week 15 (n=4,13)
    -5.8 ± 10.4
    5.0 ± 10.1
        Week 17 (n=4,9)
    -6.3 ± 8.8
    -2.2 ± 5.0
        Week 19 (n=4,9)
    -3.5 ± 13.7
    2.9 ± 6.5
        Week 21 (n=4,9)
    -5.5 ± 12.8
    -1.6 ± 5.0
        Week 23 (n=4,9)
    -3.3 ± 10.5
    -3.1 ± 9.0
        Week 25 (n=4,16)
    -6.3 ± 12.5
    1.8 ± 7.6
        Week 27 (n=4,16)
    -0.8 ± 16.0
    2.9 ± 7.2
        Week 29 (n=4,16)
    2.5 ± 14.8
    5.9 ± 8.8
        Week 33 (n=4,16)
    -0.5 ± 15.3
    6.2 ± 10.0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Temperature

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    End point title
    Change From Baseline in Body Temperature
    End point description
    Change from baseline in body temperature and at baseline values were reported for this endpoint. SAS was defined as all enrolled subjects who had received at least one dose of study treatment. Here, ‘Number Analysed’ signifies number of subjects evaluable for the specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Degree Celsius
    arithmetic mean (standard deviation)
        Baseline (n=23,24)
    36.4 ± 0.4
    36.6 ± 0.2
        Week 2 (n=23,24)
    0.1 ± 0.4
    -0.0 ± 0.3
        Week 3 (n=23,23)
    0.0 ± 0.2
    -0.1 ± 0.3
        Week 5 (n=22,23)
    0.0 ± 0.4
    -0.1 ± 0.3
        Week 7 (n=23,22)
    0.1 ± 0.3
    -0.0 ± 0.3
        Week 9 (n=22,22)
    0.0 ± 0.4
    -0.1 ± 0.3
        Week 11 (n=22,20)
    0.1 ± 0.3
    -0.0 ± 0.3
        Week 13 (n=23,22)
    -0.1 ± 0.4
    -0.0 ± 0.2
        Week 15 (n=4,13)
    0.2 ± 0.2
    0.1 ± 0.2
        Week 17 (n=4,9)
    0.1 ± 0.2
    0.1 ± 0.2
        Week 19 (n=4,9)
    0.1 ± 0.4
    0.1 ± 0.3
        Week 21 (n=4,9)
    -0.0 ± 0.1
    -0.1 ± 0.5
        Week 23 (n=4,9)
    0.2 ± 0.2
    0.0 ± 0.3
        Week 25 (n=4,16)
    0.1 ± 0.2
    0.0 ± 0.2
        Week 27 (n=4,16)
    0.1 ± 0.1
    0.1 ± 0.2
        Week 29 (n=4,16)
    0.1 ± 0.3
    -0.1 ± 0.4
        Week 33 (n=4,16)
    0.1 ± 0.1
    -0.1 ± 0.3
    No statistical analyses for this end point

    Secondary: Number of Subjects With Abnormalities in Electrocardiogram (ECG)

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    End point title
    Number of Subjects With Abnormalities in Electrocardiogram (ECG)
    End point description
    ECG abnormalities criteria included: 1) QTc interval adjusted according to Bazett formula (QTcB) in millisecond (msec): greater than (>) 450, >480, >500, increase from baseline >30, increase from baseline >60; 2) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >30, increase from baseline >60; 3) Heart rate (bpm): RR decrease >25% and to a VR (interval between QRS wave and T wave on ECG) >100; RR (interval between 2 successive R waves on ECG) increase >25% and to a VR <50; 4) Pulse rate (msec): increase >25% and to a value >200; 5) QT (msec): >450, >480, >500, increase from baseline >30, increase from baseline >60; 6) QRS (msec): increase >25% and to a value >110. Categories (timepoints) with at least 1 subject having ECG abnormality in any of the reporting arms, were reported for this endpoint. SAS population was analyzed. N= subjects evaluable for this endpoint. n= subjects evaluable for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Weeks 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    23
    Units: Subjects
        Week 3 (Not Clinically Significant) (n=23,23)
    3
    4
        Week 3 (Clinically Significant) (n=23,23)
    1
    0
        Week 7 (Not Clinically Significant) (n=22,22)
    3
    4
        Week 7 (Clinically Significant) (n=22,22)
    0
    0
        Week 11 (Not Clinically Significant) (n=22,20)
    4
    4
        Week 11 (Clinically Significant) (n=22,20)
    1
    0
        Week 13 (Not Clinically Significant) (n=23,22)
    6
    4
        Week 13 (Clinically Significant) (n=23,22)
    1
    0
        Week 17 (Not Clinically Significant) (n=4,9)
    0
    0
        Week 17 (Clinically Significant) (n=4,9)
    0
    0
        Week 21 (Not Clinically Significant) (n=4,9)
    1
    1
        Week 21 (Clinically Significant) (n=4,9)
    0
    0
        Week 25 (Not Clinically Significant) (n=4,16)
    1
    1
        Week 25 (Clinically Significant) (n=4,16)
    0
    0
        Week 33 (Not Clinically Significant) (n=3,16)
    0
    2
        Week 33 (Clinically Significant) (n=3,16)
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) at Weeks 2, 5, 9 and 13

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    End point title
    Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) at Weeks 2, 5, 9 and 13
    End point description
    UPCR is a ratio between two measured substances in urine: mmol of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function. FAS was defined as all enrolled subjects who had received at least one dose of study treatment and had at least one post-baseline measurement of UPCR based on 24-hour urine collected. Here, ‘Number Analysed’ signifies number of subjects evaluable for the specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 2, 5, 9 and 13
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Percent change
    least squares mean (confidence interval 90%)
        Week 2 (n=0,0)
    0 (0 to 0)
    0 (0 to 0)
        Week 5 (n=20,20)
    6.250 (-7.035 to 21.433)
    -3.788 (-11.344 to 4.411)
        Week 9 (n=19,20)
    -11.335 (-20.746 to -0.807)
    -2.354 (-11.069 to 7.215)
        Week 13 (n=21,19)
    -12.283 (-26.096 to 4.112)
    -0.045 (-9.528 to 10.432)
    No statistical analyses for this end point

    Secondary: Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR ) at Weeks 3, 5, 9 and 13

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    End point title
    Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR ) at Weeks 3, 5, 9 and 13
    End point description
    The eGFR was calculated using 4 variable formula developed by the modification of diet in renal disease (MDRD) study group. The 4 variables needed to estimate glomerular filtration rate (GFR) using this formula were serum creatinine concentration, age, sex (for females, eGFR was multiplied by 0.742) and ethnic origin (for African-Caribbean people only, eGFR was multiplied by 1.212). Thus eGFR in milliliter per minute per 1.73 square meter (mL/min/1.73 m^2) = 175*(sCr/88.4)^-1.154*(Age)^-0.203*(0.742 if female)*(1.212 if African-Caribbean). Baseline eGFR was determined predose at Week 0 (Day 1). For Baseline eGFR, the “Low eGFR” group was defined as baseline eGFR < 45 mL/min/1.73m2, and the “High eGFR” group was defined as baseline eGFR > 45 mL/min/1.73 m2. FAS was defined as all enrolled subjects who had received at least one dose of study treatment and had at least one post-baseline measurement of UPCR based on 24-hour urine collected. n= subjects evaluable for specific timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 3, 5, 9 and 13
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Percent change
    least squares mean (confidence interval 90%)
        Week 3 (n=23,22)
    5.657 (-0.722 to 12.446)
    -0.180 (-4.179 to 3.987)
        Week 5 (n=21,23)
    2.174 (-3.012 to 7.637)
    -2.038 (-8.008 to 4.319)
        Week 9 (n=22,22)
    -1.536 (-15.575 to 14.838)
    -5.017 (-11.596 to 2.052)
        Week 13 (n=23,22)
    2.892 (-6.096 to 12.740)
    -12.217 (-18.831 to -5.063)
    No statistical analyses for this end point

    Secondary: Serum PF-06730512 Concentration Versus Time Summary

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    End point title
    Serum PF-06730512 Concentration Versus Time Summary
    End point description
    The PK concentration analysis set was defined as all enrolled subjects treated who received at least one dose of PF-06730512 and had at least 1 measurable concentration. Here, ‘Number Analyzed’ signifies number of subjects evaluable for the specific rows. Here, ‘99999’ indicates data could not be estimated as only one subject was evaluated.
    End point type
    Secondary
    End point timeframe
    30 minutes pre-dose on Day 1, 8, 15, 29, 43, 57, 71, 85, 99, 113, 127, 141, 155, 169, follow-up (Fup) visit on Day 99, 113, 141, 183, 197, 225 and 1 hour post-dose on Day 1, 71, 155
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Microgram per millilitre
    arithmetic mean (standard deviation)
        Day 1 (n=17,22)
    279.4 ± 86.548
    71.94 ± 29.668
        Day 8 (n=1,10)
    39.50 ± 99999
    13.09 ± 6.9363
        Day 15 (n=23,21)
    16.45 ± 11.266
    5.096 ± 3.1184
        Day 29 (n=21,19)
    24.92 ± 20.256
    6.916 ± 5.0154
        Day 43 (n=20,19)
    29.96 ± 22.062
    6.991 ± 5.5862
        Day 57 (n=21,18)
    32.56 ± 23.031
    7.664 ± 6.1325
        Day 71 (n=17,17)
    27.32 ± 16.688
    6.736 ± 5.6237
        Day 85 (n=4,13)
    41.15 ± 23.516
    7.597 ± 6.9184
        Day 99/Fup (n=19,7)
    11.50 ± 12.948
    2.313 ± 3.1827
        Day 113/Fup (n=19,7)
    4.991 ± 6.1121
    0.6356 ± 0.81118
        Day 141/Fup (n=18,7)
    1.385 ± 2.1196
    0.1438 ± 0.21666
        Day 99 (n=4,9)
    35.65 ± 16.065
    7.084 ± 6.9224
        Day 113 (n=4,6)
    42.40 ± 25.049
    9.442 ± 8.6965
        Day 127 (n=3,6)
    30.67 ± 5.3463
    6.732 ± 5.2295
        Day 141 (n=4,7)
    38.00 ± 13.880
    8.957 ± 10.753
        Day 155 (n=3,8)
    32.57 ± 5.8287
    7.585 ± 8.1601
        Day 169 (n=4,16)
    29.32 ± 15.338
    7.989 ± 7.4740
        Day 183/Fup (n=4,16)
    25.79 ± 30.417
    3.046 ± 3.8895
        Day 197/Fup (n=4,16)
    6.108 ± 4.0049
    1.048 ± 1.4978
        Day 225/Fup (n=4,16)
    2.009 ± 1.6808
    0.2659 ± 0.45714
    No statistical analyses for this end point

    Secondary: Number of Subjects With Positive Anti-Drug Antibody (ADA) and/or Neutralizing Antibody(NAb)

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    End point title
    Number of Subjects With Positive Anti-Drug Antibody (ADA) and/or Neutralizing Antibody(NAb)
    End point description
    The immunogenicity analysis population included all treated subjects with at least 1 ADA sample (pre-dose or post-treatment) analysed. Subjects those had only pre-dose baseline data and no post-treatment immunogenicity data, was not evaluated for subject-level ADA. Here, ‘Number Analyzed’ signifies number of subjects evaluable for the specific rows.
    End point type
    Secondary
    End point timeframe
    From Day 1 of treatment up to Week 33
    End point values
    PF-06730512 1000 mg IV PF-06730512 300 mg IV
    Number of subjects analysed
    23
    24
    Units: Subjects
        ADA Positive (n=21,24)
    1
    0
        NAb Positive (n=21,24)
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day 1 of treatment up to 9 weeks after last dose of study treatment (up to Week 33)
    Adverse event reporting additional description
    Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorised as serious in one subject and as non-serious in another subject, or one subject may have experienced both a serious and non-serious event during the study.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    PF-06730512 300 mg IV
    Reporting group description
    Subjects received PF-06730512 300 mg IV Q2W.

    Reporting group title
    PF-06730512 1000 mg IV
    Reporting group description
    Subjects received PF-06730512 1000 mg IV Q2W.

    Serious adverse events
    PF-06730512 300 mg IV PF-06730512 1000 mg IV
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 23 (8.70%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    General disorders and administration site conditions
    Disease progression
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal impairment
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Acute kidney injury
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypervolaemia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    PF-06730512 300 mg IV PF-06730512 1000 mg IV
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 24 (50.00%)
    11 / 23 (47.83%)
    Investigations
    SARS-CoV-2 test positive
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 23 (8.70%)
         occurrences all number
    2
    2
    Injury, poisoning and procedural complications
    Infusion related reaction
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 24 (12.50%)
    2 / 23 (8.70%)
         occurrences all number
    3
    3
    Dizziness
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    3
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Oedema
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    Fatigue
         subjects affected / exposed
    2 / 24 (8.33%)
    3 / 23 (13.04%)
         occurrences all number
    2
    4
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 23 (4.35%)
         occurrences all number
    3
    1
    Vomiting
         subjects affected / exposed
    1 / 24 (4.17%)
    2 / 23 (8.70%)
         occurrences all number
    2
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    Flank pain
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    3
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    COVID-19
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 23 (4.35%)
         occurrences all number
    2
    1
    Viral infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 May 2018
    Specifications for dose levels, dosing frequency and route of administration have been included. Randomisation of subjects to the high versus the low dose has been removed. Instead, a sequential design has been implemented allowing Cohort 1 enrollment to complete prior to enrolment of Cohort 2.
    10 Jun 2019
    Changes in Inclusion and Exclusion Criteria and addition of the optional renal biopsy sub-study.
    03 Jan 2020
    Changes to Inclusion/Exclusion criteria.
    12 Jun 2020
    Revision/Clarification to contraception language for EU member states and Japan.
    18 Aug 2021
    The purpose of this amendment was to extend treatment duration from 12 weeks to 24 weeks, and to add an optional cohort to potentially explore a higher dose.

    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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    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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