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    Clinical Trial Results:
    Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    2023-000321-80
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    09 Aug 2023

    Results information
    Results version number
    v2(current)
    This version publication date
    17 Aug 2024
    First version publication date
    22 Feb 2024
    Other versions
    v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    FGCL-3019-079
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02606136
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    FibroGen, Inc.
    Sponsor organisation address
    409 Illinois Street, San Francisco, United States, CA 94158
    Public contact
    Clinical Trial Information Desk, FibroGen, Inc., FG3019-079DMDStudy@fibrogen.com
    Scientific contact
    Clinical Trial Information Desk, FibroGen, Inc., FG3019-079DMDStudy@fibrogen.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-002979-PIP01-21
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    09 Aug 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Aug 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this trial is to estimate pamrevlumab’s efficacy in non-ambulatory participants with Duchenne Muscular Dystrophy (DMD).
    Protection of trial subjects
    The study was conducted and monitored in accordance with United States (US) Food and Drug Administration (FDA) regulations, the International Council for Harmonisation E6 Guideline for Good Clinical Practice (GCP), the Declaration of Helsinki, any other applicable regulatory requirements, and Institutional Review Board (IRB) requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    04 Jan 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 21
    Worldwide total number of subjects
    21
    EEA total number of subjects
    0
    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)
    18
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    All participants who completed the main portion of the study for a minimum of 104 weeks (2 years) were rolled over to an open-label extension (OLE) for up to an additional 208 weeks (4 years). Some participants remained in the main study for up to 206 weeks before rolling over to the OLE.

    Period 1
    Period 1 title
    Main Study (104 Weeks)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Pamrevlumab
    Arm description
    Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    Other name
    Monoclonal Antibody to Connective tissue growth factor (CTGF), FG-3019
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Pamrevlumab was administered per dose and schedule specified in the arm description.

    Number of subjects in period 1
    Pamrevlumab
    Started
    21
    Received at Least 1 Dose of Study Drug
    21
    Completed
    15
    Not completed
    6
         Consent withdrawn by subject
    1
         Participant/Legal Guardian Decision
    5
    Period 2
    Period 2 title
    OLE (Up to 208 Weeks)
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Pamrevlumab
    Arm description
    Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    Other name
    Monoclonal Antibody to CTGF, FG-3019
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Pamrevlumab was administered per dose and schedule specified in the arm description.

    Number of subjects in period 2
    Pamrevlumab
    Started
    15
    Received at least 1 dose of study drug
    15
    Completed
    0
    Not completed
    15
         Sponsor Decision to Terminate Study
    14
         Participant/Legal Guardian Decision
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.

    Reporting group values
    Pamrevlumab Total
    Number of subjects
    21 21
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    15.99 ( 3.277 ) -
    Sex: Female, Male
    Units: participants
        Female
    0 0
        Male
    21 21
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0
        Asian
    1 1
        Native Hawaiian or Other Pacific Islander
    0 0
        Black or African American
    0 0
        White
    20 20
        More than one race
    0 0
        Unknown or Not Reported
    0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    2 2
        Not Hispanic or Latino
    19 19
        Unknown or Not Reported
    0 0
    Percent Predicted Forced Vital Capacity (ppFVC)
    FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
    Units: percentage of predicted FVC
        arithmetic mean (full range (min-max))
    54.15 (29.1 to 70.7) -
    Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1)
    FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Predicted FEV1 is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FEV1= (observed value)/(predicted value) * 100%.
    Units: percentage of predicted FEV1
        arithmetic mean (full range (min-max))
    53.815 (29.24 to 73.35) -
    Left Ventricular Ejection Fraction Percentage (LVEF%)
    LVEF% is an important measure of cardiac function. LVEF is a fraction of blood (in percent) pumped out of the left ventricle of the heart (the main pumping chamber).
    Units: percentage of LVEF
        arithmetic mean (full range (min-max))
    56.992 (41.03 to 73.81) -
    Performance of Upper Limb (PUL) Total Score
    The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items: maximum score = 12), elbow (middle, 9 items: maximum score = 17) and wrist/hand (distal, 7 items: maximum score = 13). Higher scores of each item indicate higher level of function. Total score was calculated by adding the 3 level scores, with a maximum global score of 42 (total score range = 0-42; with higher score indicating better outcome).
    Units: unit on a scale
        arithmetic mean (full range (min-max))
    24.4 (13 to 41) -
    Pinch Strength (Dominant Pinch Best Result), as Measured by Hand Held Myometry (HHM)
    The HHM was used to measure distal upper arm strength (pinch strength).
    Units: newton
        arithmetic mean (full range (min-max))
    17.003 (0 to 45.1) -
    Percent Predicted Peak Expiratory Flow (PEF)
    Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort‐dependent measure of lung function.
    Units: percentage of predicted PEF
        arithmetic mean (full range (min-max))
    54.66 (37.9 to 82.7) -
    Pinch Strength, as Measured by Hand Held Myometry (HHM)
    The HHM was used to measure distal upper arm strength (pinch strength). Data has been presented by dominant and non-dominant hand.
    Units: newton
        arithmetic mean (full range (min-max))
    16.607 (0 to 47.1) -
    Cardiac Fibrosis Score (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI)
    'Number of participants analyzed' = 20
    Units: grams
        arithmetic mean (full range (min-max))
    24.100 (0.44 to 76.09) -
    Grip Strength by Hand (Dominant Grip Best Result), as Measured by HHM
    The HHM was used to measure distal upper arm strength (grip strength).
    Units: newton
        arithmetic mean (full range (min-max))
    45.861 (3 to 142.2) -
    Grip Strength by Hand, as Measured by HHM
    The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand.
    Units: newton
        arithmetic mean (full range (min-max))
    45.861 (3 to 142.2) -
    Pinch Strength (Non-Dominant Pinch Best Result), as Measured by Hand Held Myometry (HHM)
    The HHM was used to measure distal upper arm strength (pinch strength).
    Units: newton
        arithmetic mean (full range (min-max))
    16.607 (0 to 47.1) -
    Fat Fraction Percentage (%F), as Measured by MRI
    'Number of participants analyzed' = 9
    Units: percentage of fat
        arithmetic mean (full range (min-max))
    22.07 (4 to 32.6) -
    Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score
    T2-mapping with MRI was conducted to measure upper arm muscle fibrosis and fat in the biceps brachii muscle. 'Number of participants analyzed' = 12
    Units: unit on a scale
        arithmetic mean (full range (min-max))
    8.01 (3.9 to 17.2) -
    Grip Strength by Hand (Non-Dominant Grip Best Result), as Measured by HHM
    The HHM was used to measure distal upper arm strength (grip strength).
    Units: newton
        arithmetic mean (full range (min-max))
    41.977 (2 to 104.9) -

    End points

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    End points reporting groups
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks in the main study. Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.

    Primary: Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104

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    End point title
    Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 [1]
    End point description
    FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%. Analysis was done using a random coefficient model (RCM), which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Primary
    End point timeframe
    Baseline, Week 104
    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: Statistical analysis was not planned for this endpoint.
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: percentage of predicted FVC
        least squares mean (standard error)
    -4.17 ( 0.655 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104

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    End point title
    Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104
    End point description
    FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Predicted FEV1 is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FEV1= (observed value)/(predicted value) * 100%. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: percentage of predicted FEV1
        least squares mean (standard error)
    -8.32 ( 1.217 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104

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    End point title
    Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104
    End point description
    LVEF% is an important measure of cardiac function. LVEF is a fraction of blood (in percent) pumped out of the left ventricle of the heart (the main pumping chamber). Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: percentage of LVEF
        least squares mean (standard error)
    -2.73 ( 1.651 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104

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    End point title
    Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104
    End point description
    The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items: maximum score = 12), elbow (middle, 9 items: maximum score = 17) and wrist/hand (distal, 7 items: maximum score = 13). Higher scores of each item indicate higher level of function. Total score was calculated by adding the 3 level scores, with a maximum global score of 42 (total score range = 0-42; with higher score indicating better outcome). Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: unit on a scale
        least squares mean (standard error)
    -4.14 ( 0.651 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104

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    End point title
    Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104
    End point description
    The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: newton
    least squares mean (standard error)
        Dominant Grip Best Result
    -2.52 ( 3.610 )
        Non-Dominant Grip Best Result
    -1.31 ( 3.591 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104

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    End point title
    Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104
    End point description
    Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort‐dependent measure of lung function. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: percentage of predicted PEF
        least squares mean (standard error)
    -7.13 ( 2.313 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104

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    End point title
    Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104
    End point description
    Cardiac MRI was used to assess the cardiac fibrosis by detecting the presence of late gadolinium enhancement (LGE), a marker for myocardial fibrosis. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    20
    Units: grams
        least squares mean (standard error)
    3.74 ( 4.475 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Pinch Strength, as Measured by HHM at Week 104

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    End point title
    Change From Baseline in Pinch Strength, as Measured by HHM at Week 104
    End point description
    The HHM was used to measure distal upper arm strength (pinch strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    21
    Units: newton
    least squares mean (standard error)
        Dominant Pinch Best Result
    -4.24 ( 1.547 )
        Non-Dominant Pinch Best Result
    -3.46 ( 1.378 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104

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    End point title
    Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104
    End point description
    T2-mapping with MRI was conducted to measure upper arm muscle fibrosis and fat in the biceps brachii muscle. Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. The visual score for muscle fat and fibrosis will be assessed using a modified 5-point Mercuri score in which 0 = normal muscle appearance and 5 = complete replacement of muscle by connective tissue and fat, where a lower score indicated visually healthier muscle. Change from baseline was calculated as the score at Week 104 – the score at baseline. ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    12
    Units: unit on scale
        least squares mean (standard error)
    -2.22 ( 1.088 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104

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    End point title
    Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104
    End point description
    Analysis was done using an RCM, which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect. ITT population included all participants who enrolled in the study. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 104
    End point values
    Pamrevlumab
    Number of subjects analysed
    9
    Units: percentage of fat
        least squares mean (standard error)
    4.49 ( 2.030 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 210
    Adverse event reporting additional description
    Safety population included participants who received at least one dose of study drug.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    OLE: Pamrevlumab
    Reporting group description
    Participants who completed the main study, continued to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.

    Reporting group title
    Main Study: Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of 104 weeks.

    Serious adverse events
    OLE: Pamrevlumab Main Study: Pamrevlumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 15 (33.33%)
    6 / 21 (28.57%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Concussion
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skull fracture
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Adverse drug reaction
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Retinal exudates
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Food poisoning
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Hydronephrosis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pilonidal disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Catheter site abscess
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (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
    Metabolic acidosis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 21 (4.76%)
         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
    OLE: Pamrevlumab Main Study: Pamrevlumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 15 (100.00%)
    21 / 21 (100.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 15 (6.67%)
    8 / 21 (38.10%)
         occurrences all number
    6
    17
    Chills
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Fatigue
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 21 (4.76%)
         occurrences all number
    3
    1
    Oedema peripheral
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Pain
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 21 (4.76%)
         occurrences all number
    3
    1
    Vaccination site pain
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    5
    0
    Medical device site rash
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    3
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 15 (26.67%)
    9 / 21 (42.86%)
         occurrences all number
    8
    20
    Sinus congestion
         subjects affected / exposed
    1 / 15 (6.67%)
    6 / 21 (28.57%)
         occurrences all number
    2
    12
    Oropharyngeal pain
         subjects affected / exposed
    4 / 15 (26.67%)
    4 / 21 (19.05%)
         occurrences all number
    7
    9
    Rhinorrhoea
         subjects affected / exposed
    2 / 15 (13.33%)
    4 / 21 (19.05%)
         occurrences all number
    3
    8
    Nasal congestion
         subjects affected / exposed
    1 / 15 (6.67%)
    3 / 21 (14.29%)
         occurrences all number
    1
    5
    Productive cough
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Epistaxis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Pulmonary congestion
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    3
    0
    Sneezing
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Restrictive pulmonary disease
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 21 (0.00%)
         occurrences all number
    3
    0
    Respiratory disorder
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract congestion
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 15 (6.67%)
    3 / 21 (14.29%)
         occurrences all number
    1
    3
    Depression
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Insomnia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Bone density decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Weight decreased
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Cystatin C increased
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 21 (9.52%)
         occurrences all number
    1
    3
    Ejection fraction decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    SARS-CoV-2 test positive
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Sunburn
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Fall
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 21 (4.76%)
         occurrences all number
    2
    1
    Contusion
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 21 (4.76%)
         occurrences all number
    4
    1
    Bone contusion
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Muscle strain
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 21 (9.52%)
         occurrences all number
    2
    3
    Thermal burn
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Procedural pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Accident
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 21 (14.29%)
         occurrences all number
    0
    3
    Cardiac dysfunction
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Cardiomyopathy
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 15 (26.67%)
    14 / 21 (66.67%)
         occurrences all number
    14
    66
    Dizziness
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 21 (14.29%)
         occurrences all number
    0
    4
    Migraine
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 21 (9.52%)
         occurrences all number
    2
    3
    Sinus headache
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Fine motor skill dysfunction
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    2 / 15 (13.33%)
    3 / 21 (14.29%)
         occurrences all number
    2
    3
    Vertigo positional
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Chalazion
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    2 / 15 (13.33%)
    10 / 21 (47.62%)
         occurrences all number
    4
    13
    Nausea
         subjects affected / exposed
    2 / 15 (13.33%)
    7 / 21 (33.33%)
         occurrences all number
    2
    11
    Abdominal pain upper
         subjects affected / exposed
    1 / 15 (6.67%)
    5 / 21 (23.81%)
         occurrences all number
    1
    8
    Diarrhoea
         subjects affected / exposed
    2 / 15 (13.33%)
    5 / 21 (23.81%)
         occurrences all number
    2
    6
    Abdominal distension
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Constipation
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 21 (4.76%)
         occurrences all number
    2
    1
    Dyspepsia
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 21 (9.52%)
         occurrences all number
    2
    4
    Oral pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Irritable bowel syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Hyperaesthesia teeth
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Haemorrhoids
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Rectal haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Toothache
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Melaena
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 21 (9.52%)
         occurrences all number
    1
    2
    Rash
         subjects affected / exposed
    3 / 15 (20.00%)
    2 / 21 (9.52%)
         occurrences all number
    4
    2
    Skin discolouration
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Alopecia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Eczema
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Dermal cyst
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Decubitus ulcer
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Ingrowing nail
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 21 (9.52%)
         occurrences all number
    1
    2
    Haematuria
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    3
    0
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    1 / 15 (6.67%)
    5 / 21 (23.81%)
         occurrences all number
    1
    6
    Arthralgia
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 21 (9.52%)
         occurrences all number
    2
    3
    Back pain
         subjects affected / exposed
    3 / 15 (20.00%)
    8 / 21 (38.10%)
         occurrences all number
    3
    13
    Temporomandibular joint syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Scoliosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Pain in extremity
         subjects affected / exposed
    4 / 15 (26.67%)
    0 / 21 (0.00%)
         occurrences all number
    4
    0
    Neuromuscular scoliosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Bone pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Dactylitis
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Joint contracture
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Muscular weakness
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 21 (4.76%)
         occurrences all number
    1
    1
    Influenza
         subjects affected / exposed
    0 / 15 (0.00%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    Sinusitis
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 21 (14.29%)
         occurrences all number
    0
    6
    Nasopharyngitis
         subjects affected / exposed
    3 / 15 (20.00%)
    11 / 21 (52.38%)
         occurrences all number
    4
    26
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 15 (20.00%)
    5 / 21 (23.81%)
         occurrences all number
    4
    6
    Pilonidal disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Anal abscess
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    COVID-19
         subjects affected / exposed
    4 / 15 (26.67%)
    0 / 21 (0.00%)
         occurrences all number
    4
    0
    Ear infection
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    3
    0
    Fungal infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Fungal skin infection
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0
    Infected cyst
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Skin candida
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Rhinitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 21 (0.00%)
         occurrences all number
    1
    0
    Hyperphosphataemia
         subjects affected / exposed
    4 / 15 (26.67%)
    0 / 21 (0.00%)
         occurrences all number
    5
    0
    Hyponatraemia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 21 (0.00%)
         occurrences all number
    2
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Aug 2015
    It included following changes: - Deleted exploratory endpoint: Effect of concomitant corticosteroid treatment on LVEF and cardiac fibrosis. - Inclusion criteria was updated to specify wheelchair dependent for <5 years. - Inclusion criteria was updated to increased length of stable regimen of heart failure cardiac medications prior to screening from 6 weeks to 3 months. - Exclusion criteria was updated to mention the need for intravenous diuretics or inotropic support increased from within 6 weeks to at least 3 months prior to screening; and hospitalization for a heart failure exacerbation or arrhythmia increased from last 6 weeks to last 3 months. - Deleted inclusion criteria for the option of no corticosteroid use for at least 6 months prior to screening and throughout the study participation. - Mandated stable treatment with corticosteroids at baseline. - Changed duration of follow-up period from 6 weeks to 10 weeks. - Allowed use of deflazacort, if regarded by the principal investigator as standard of care.
    06 May 2016
    It included following changes: - Replaced forearm muscle MRI with upper arm (bicep) muscle MRI. - Revised Inclusion criterion 8 to allow only enrollment of participants with a ppFVC ≥50. - Dosing based on body weight measured at screening and every 3 months thereafter. - Safety follow-up period reduced from 10 weeks to 4 weeks after the last dose of study drug. - Infusion rate of FG-3019 not to exceed 150 cubic centimeters (cc)/hour. Adjustments to lower infusion rate allowed per investigator’s clinical judgment. - Clarified requirement for local safety labs (including hematocrit) to be drawn prior to conduct of MRIs. - Added stipulation that home health care may be considered for administration of future pamrevlumab infusions during the conduct of the study.
    09 Dec 2016
    It included following changes: - Replacement of FG-3019 with pamrevlumab. - Decreased study duration from 104 weeks to 52 weeks with option to continue an additional 26 weeks (78 weeks total) for participants who achieve a ≤5% decline from baseline in ppFVC by Week 52. - Updated time on study to reflect weeks vs years (ie, change from 1 year to 52 weeks). - Removed Interim Analysis at Week 52. - Deleted Inclusion criterion: Wheelchair dependency <5 years. - Revised Inclusion criterion to only allow enrollment of participants with a ppFVC between 40 and 90, inclusive. Deleted “estimated annual decline of FVC (% predicted) of ≥5% based upon at least 2 PFTs done in the previous 18 months, in addition to the screening FVC” from Inclusion criterion. Changed criteria to “At least one historical FVC % predicted value within 18 months of baseline”. - Modified Inclusion criterion to “Participants currently receiving heart failure cardiac medications (for example, angiotensin converting enzyme inhibitors, angiotensin-receptor blockers, and beta-blockers) must achieve a stable regimen for at least 3 months prior to screening”. - Modified Exclusion criterion to update time frame of anticipated spine surgery from 2 years to 78 weeks. - Modified Exclusion criterion to clarify that the use of another investigational drug or another approved product for DMD (for example, eteplirsen) within 28 days or 5 half-lives of the product, whichever was longer, prior to first Screening Visit, with the exception of deflazacort, was exclusionary. Allowed use of deflazacort if regarded by the principal investigator as standard of care. - Clarified that any approved product for DMD (for example, eteplirsen) during study treatment was prohibited.
    10 Jul 2017
    It included following changes: -Sample size changed to reflect that study may enroll up to 32 participants dependent on outcome of interim analysis. - Extended treatment duration from 52 weeks plus extension to 104 weeks; removed extension and added interim analysis. - Changed primary endpoint to annual change in ppFVC during treatment. - Changed Exclusion criteria to reflect increased treatment duration. - Amended language to reflect that only the first infusion was based on the screening weight. - Updated to reflect that infusion reactions are considered an identified risk of pamrevlumab administration and deleted that they did not recur with readministration.
    14 Nov 2017
    It included following changes: - Primary efficacy endpoint changed from “annual change in ppFVC during treatment with pamrevlumab” to “annual change from baseline to Week 104 in ppFVC during treatment with pamrevlumab”. - Removed sentence about analysis method (“t-test”) for primary efficacy endpoint. - Modified number of participants required for interim analysis from “at least 12” to “at least 10 to 12”. - Clarified PK sample time-point collection. - Extended treatment duration from 104 weeks to 156 weeks with exploratory analyses at Week 156.
    27 Sep 2019
    It included following changes: - Addition of Appendix: OLE for all participants who completed 104 weeks of treatment on the main study and completed end of treatment (EOT). Modifications made in main study to address addition of Appendix. - Added Respiratory Muscles and Diaphragm MRI in OLE.
    18 Nov 2020
    It included following changes: - Safety follow-up extended to 60 days (+3 days) after the last infusion. - Study drug administration window time updated from 24 to 48 hours. - Provided further clarity on the overall duration of the Open Label Extension. - Provided allowance for administration of approved DMD therapies during the OLE treatment period >3 hours after pamrevlumab administration.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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