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    Clinical Trial Results:
    A Phase 3, Randomized, Double-Blind Trial of Pamrevlumab (FG 3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-Ambulatory Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    2020-000698-26
    Trial protocol
    FR   AT   CZ   NL   BE   GB   IT   ES  
    Global end of trial date
    17 Aug 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    06 Mar 2024
    First version publication date
    06 Mar 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    FGCL-3019-093
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04371666
    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., FG3019-093DMDStudy@fibrogen.com
    Scientific contact
    Clinical Trial Information Desk, FibroGen, Inc., FG3019-093DMDStudy@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
    17 Aug 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Aug 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with DMD (non-ambulatory, aged ≥12 years).
    Protection of trial subjects
    This study was conducted in accordance with the Declaration of Helsinki, Good Clinical Practice (GCP), the International Council for Harmonisation (ICH) E6 Guidance for GCP, any other applicable local health and regulatory requirements, and Ethics Committee (EC) requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    30 Jul 2020
    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: 51
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Italy: 10
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    Switzerland: 1
    Country: Number of subjects enrolled
    Austria: 1
    Country: Number of subjects enrolled
    New Zealand: 1
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Czechia: 5
    Country: Number of subjects enrolled
    China: 12
    Worldwide total number of subjects
    98
    EEA total number of subjects
    31
    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)
    77
    Adults (18-64 years)
    21
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    The study included 2 periods: Double-blind (DB) Treatment Period and Open-label Extension (OLE) Period.

    Period 1
    Period 1 title
    DB Treatment Period (52 Weeks)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Pamrevlumab
    Arm description
    Participants received pamrevlumab intravenously (IV) every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    Other name
    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.

    Arm title
    Placebo
    Arm description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Placebo matched to pamrevlumab was administered per schedule specified in the arm description.

    Number of subjects in period 1
    Pamrevlumab Placebo
    Started
    49
    49
    Received at least 1 dose of study drug
    48
    49
    Completed
    44
    46
    Not completed
    5
    3
         Adverse event, non-fatal
    3
    1
         Other than specified
    1
    -
         Participant/Legal Guardian Decision
    1
    2
    Period 2
    Period 2 title
    OLE (Maximum Exposure: 93.4 Weeks)
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Pamrevlumab
    Arm description
    Participants received pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    Other name
    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.

    Arm title
    Placebo
    Arm description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    Other name
    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 [1]
    Pamrevlumab Placebo
    Started
    43
    43
    Received at least 1 dose of study drug
    43
    43
    Completed
    0
    0
    Not completed
    43
    43
         Sponsor Decision to Terminate Study
    40
    41
         Other than specified
    1
    -
         Participant/Legal Guardian Decision
    2
    2
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: 4 participants who completed the DB Treatment Period did not enter in to the OLE period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab intravenously (IV) every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Reporting group values
    Pamrevlumab Placebo Total
    Number of subjects
    49 49 98
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    15.6 ( 2.74 ) 15.5 ( 2.42 ) -
    Sex: Female, Male
    Units: participants
        Female
    0 0 0
        Male
    49 49 98
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0
        Asian
    6 9 15
        Native Hawaiian or Other Pacific Islander
    0 0 0
        Black or African American
    0 2 2
        White
    42 35 77
        More than one race
    1 3 4
        Unknown or Not Reported
    0 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    2 4 6
        Not Hispanic or Latino
    44 41 85
        Unknown or Not Reported
    3 4 7

    End points

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    End points reporting groups
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab intravenously (IV) every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants received pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period. After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Primary: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52

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    End point title
    Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52
    End point description
    The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items, 12 points), elbow (middle, 9 items, 17 points) and wrist/hand (distal, 7 items, 13 points). Higher scores indicate higher level of function. Total score ranges from 0-42 points and is the sum of the scores for the 3 subscales. Analysis was done using a random coefficient model (RCM), which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline ordinal PUL entry score as covariate. The modified intent-to-treat (mITT) population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    38
    40
    Units: units on a scale
        least squares mean (standard error)
    -2.036 ( 0.4471 )
    -2.119 ( 0.3367 )
    Statistical analysis title
    Statistical Analysis 1
    Comparison groups
    Pamrevlumab v Placebo
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.8802 [1]
    Method
    Random coefficient model
    Parameter type
    Least square (LS) Mean Difference
    Point estimate
    0.083
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.01
         upper limit
    1.176
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.5494
    Notes
    [1] - Threshold for significance at 0.05 level.

    Secondary: Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry

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    End point title
    Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry
    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 an RCM, which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline value as covariate. The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    38
    40
    Units: percentage of predicted FVC
        least squares mean (standard error)
    -8.349 ( 1.5760 )
    -5.989 ( 1.2233 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM)

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    End point title
    Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM)
    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. Grip Strength was analyzed using a MMRM with fixed effects for treatment, visit (as a factor), treatment-by-visit interaction, and covariates (baseline values). The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. 'n' = participants evaluable for specified category.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    37
    39
    Units: newton
    least squares mean (standard error)
        Grip Strength by Dominant Hand (n=37,39)
    -7.570 ( 2.0989 )
    -0.072 ( 2.2065 )
        Grip Strength by Nondominant Hand (n=36,39)
    -7.627 ( 1.8893 )
    -0.012 ( 1.8546 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Left Ventricular Ejection Fraction percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI)

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    End point title
    Change From Baseline in Left Ventricular Ejection Fraction percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI)
    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). The LVEF% was analyzed using an analysis of covariance (ANCOVA) model with treatment and baseline value. The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    33
    37
    Units: percentage of LVEF
        least squares mean (standard error)
    -0.499 ( 0.9750 )
    -1.114 ( 0.9204 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry

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    End point title
    Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry
    End point description
    The ppPEF 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. The ppFEV1 was analyzed using an RCM including fixed effects of time, treatment, and treatment-by-time interaction, with baseline as covariate. The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    46
    47
    Units: percentage of predicted PEF
        least squares mean (standard error)
    -4.921 ( 2.3086 )
    -4.516 ( 1.7663 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    DB treatment period: From first dose of study drug up to 61 weeks OLE period: From first dose of study drug up to study termination by Sponsor and safety follow-up (up to approximately 102 weeks)
    Adverse event reporting additional description
    The safety analysis set included all participants who received any dose of study medication.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    DB Period: Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period.

    Reporting group title
    OLE Period: Pamrevlumab
    Reporting group description
    After completing Week 52 of the DB treatment period, participants in the OLE period then moved to Week 2 of the OLE period and received pamrevlumab IV every 2 weeks.

    Reporting group title
    DB Period: Pamrevlumab
    Reporting group description
    Participants received pamrevlumab IV every 2 weeks for up to 52 weeks in the DB treatment period.

    Serious adverse events
    DB Period: Placebo OLE Period: Pamrevlumab DB Period: Pamrevlumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 49 (12.24%)
    5 / 86 (5.81%)
    8 / 48 (16.67%)
         number of deaths (all causes)
    0
    0
    1
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Fracture
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    3 / 49 (6.12%)
    2 / 86 (2.33%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ankle fracture
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 86 (1.16%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac arrest
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Myocarditis
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Loss of consciousness
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Food allergy
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Testicular necrosis
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Testicular torsion
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 86 (1.16%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Acute respiratory failure
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 86 (0.00%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumothorax
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 86 (1.16%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 49 (2.04%)
    1 / 86 (1.16%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 86 (0.00%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 86 (0.00%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin infection
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 86 (1.16%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    DB Period: Placebo OLE Period: Pamrevlumab DB Period: Pamrevlumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    44 / 49 (89.80%)
    53 / 86 (61.63%)
    42 / 48 (87.50%)
    Investigations
    Heart rate increased
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    3 / 48 (6.25%)
         occurrences all number
    0
    0
    7
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 49 (4.08%)
    4 / 86 (4.65%)
    4 / 48 (8.33%)
         occurrences all number
    4
    4
    4
    Limb injury
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 86 (3.49%)
    4 / 48 (8.33%)
         occurrences all number
    0
    3
    4
    Vascular access site bruising
         subjects affected / exposed
    4 / 49 (8.16%)
    1 / 86 (1.16%)
    3 / 48 (6.25%)
         occurrences all number
    4
    1
    3
    Ligament sprain
         subjects affected / exposed
    3 / 49 (6.12%)
    0 / 86 (0.00%)
    0 / 48 (0.00%)
         occurrences all number
    3
    0
    0
    Femur fracture
         subjects affected / exposed
    3 / 49 (6.12%)
    1 / 86 (1.16%)
    1 / 48 (2.08%)
         occurrences all number
    3
    1
    1
    Vascular disorders
    Flushing
         subjects affected / exposed
    2 / 49 (4.08%)
    3 / 86 (3.49%)
    3 / 48 (6.25%)
         occurrences all number
    2
    3
    3
    Cardiac disorders
    Cardiomyopathy
         subjects affected / exposed
    5 / 49 (10.20%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences all number
    5
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    2 / 49 (4.08%)
    1 / 86 (1.16%)
    4 / 48 (8.33%)
         occurrences all number
    2
    3
    4
    Headache
         subjects affected / exposed
    10 / 49 (20.41%)
    24 / 86 (27.91%)
    25 / 48 (52.08%)
         occurrences all number
    20
    50
    65
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 49 (8.16%)
    14 / 86 (16.28%)
    12 / 48 (25.00%)
         occurrences all number
    4
    15
    14
    Pain
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    4 / 48 (8.33%)
         occurrences all number
    0
    0
    10
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 86 (0.00%)
    3 / 48 (6.25%)
         occurrences all number
    0
    0
    3
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    4 / 49 (8.16%)
    4 / 86 (4.65%)
    5 / 48 (10.42%)
         occurrences all number
    4
    4
    8
    Nausea
         subjects affected / exposed
    3 / 49 (6.12%)
    5 / 86 (5.81%)
    8 / 48 (16.67%)
         occurrences all number
    3
    8
    13
    Vomiting
         subjects affected / exposed
    5 / 49 (10.20%)
    5 / 86 (5.81%)
    10 / 48 (20.83%)
         occurrences all number
    6
    8
    15
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 49 (8.16%)
    6 / 86 (6.98%)
    2 / 48 (4.17%)
         occurrences all number
    4
    6
    4
    Rhinorrhoea
         subjects affected / exposed
    3 / 49 (6.12%)
    0 / 86 (0.00%)
    1 / 48 (2.08%)
         occurrences all number
    3
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    3 / 49 (6.12%)
    1 / 86 (1.16%)
    3 / 48 (6.25%)
         occurrences all number
    3
    1
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    5 / 49 (10.20%)
    0 / 86 (0.00%)
    4 / 48 (8.33%)
         occurrences all number
    5
    0
    4
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    3 / 49 (6.12%)
    3 / 86 (3.49%)
    2 / 48 (4.17%)
         occurrences all number
    3
    3
    2
    Arthralgia
         subjects affected / exposed
    4 / 49 (8.16%)
    2 / 86 (2.33%)
    2 / 48 (4.17%)
         occurrences all number
    4
    2
    2
    Back pain
         subjects affected / exposed
    3 / 49 (6.12%)
    9 / 86 (10.47%)
    7 / 48 (14.58%)
         occurrences all number
    4
    11
    7
    Osteoporosis
         subjects affected / exposed
    1 / 49 (2.04%)
    6 / 86 (6.98%)
    1 / 48 (2.08%)
         occurrences all number
    1
    6
    1
    Myalgia
         subjects affected / exposed
    0 / 49 (0.00%)
    5 / 86 (5.81%)
    1 / 48 (2.08%)
         occurrences all number
    0
    5
    2
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    4 / 49 (8.16%)
    4 / 86 (4.65%)
    1 / 48 (2.08%)
         occurrences all number
    5
    4
    1
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 49 (6.12%)
    2 / 86 (2.33%)
    6 / 48 (12.50%)
         occurrences all number
    3
    3
    6
    Nasopharyngitis
         subjects affected / exposed
    8 / 49 (16.33%)
    8 / 86 (9.30%)
    3 / 48 (6.25%)
         occurrences all number
    9
    15
    8
    COVID-19
         subjects affected / exposed
    19 / 49 (38.78%)
    10 / 86 (11.63%)
    19 / 48 (39.58%)
         occurrences all number
    19
    10
    20

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Apr 2020
    It included following changes: - Muscle fibrosis MRI assessment was removed from inclusion criteria, and the fibrosis MRI assessment was added as an exploratory endpoint, per Food and Drug Administration (FDA) feedback.
    15 Sep 2020
    It included following changes: - Safety follow-up was extended to 60 days (+ 3 days) after the last infusion to align with 5 times the half-life of pamrevlumab (12.2 days). - Clarification was added that participants already on an approved DMD therapy should not discontinue that therapy to be eligible for the study. - Study drug administration window was extended from 24 to 48 hours to align with updated pamrevlumab stability information. - Electrocardiograms (ECGs) were removed to align with DMD standard of care. - Basophils were added to include all components of the lab testing panel. - Pulmonary function test (PFT) inclusion criteria were clarified. - Criteria for participants to continue into the OLE period were clarified. - Muscle fibrosis MRI assessment and fibrosis endpoint were removed from the OLE period.
    26 Oct 2020
    It included following changes: - Per investigator feedback, Inclusion Criterion was changed to average (of Screening and Day 0) ppFVC between 45 and 85, inclusive, to mitigate risk and safeguard the wellbeing of the DMD participants, who could have potentially suffered from exhaustion with the original ppFVC requirements.
    09 Jul 2021
    It included following changes: - Participation requirements for cardiac MRI, pneumonia and influenza vaccinations, and acceptable ranges for central laboratory assessments were clarified. - Inclusion Criterion was changed to remove the requirement for cardiomyopathy diagnosis. - The exclusion criterion for allergic reactions was expanded to include hypersensitivity to the study drug components or the gadolinium-based contrast agents required for the MRI. - Casimersen (amondys 45) was added to the prohibited concomitant medications, due to its recent drug approval. - Cardiac and pulmonary assessment exclusion criteria were expanded to exclude participants with abnormal glomerular filtration rate (GFR) or acute kidney injury. - Clarification was added to state that pamrevlumab would be permanently discontinued in the case of a serious or life-threatening allergic reaction. - Instruction was added for participants to speak to the study doctor and assess if an unscheduled visit is required after any adverse reaction during an home health care (HHC) visit. - Guidance regarding the time separation between study drug and COVID-19 vaccination was added. - Guidance regarding visit modality (in-person vs remote) and visit scheduling was added to align with FDA/European Medicines Agency (EMA) guidance regarding the COVID-19 pandemic and the resulting need for flexibility for visits and assessments. • Contraception requirements were added to include permitted methods, such as condoms and abstinence. • Clarification was added that subjects who discontinue from the Double-blind Treatment period were not eligible for OLE participation. • AE reporting and AE definition were updated to reflect International Council for Harmonisation (ICH) E2A standard language.
    01 Nov 2022
    It included following changes: - Bone fracture safety assessments were added to align with the pediatric investigational plan. - Secondary endpoints were rearranged to align with the revised analysis plan. - The exploratory endpoint of summarizing the subscores of the 3 regional dimensions of the PUL was added. - Withdrawal criteria were updated to include a description for participants discontinuing infusions. • Additional pharmacokinetics (PK), anti-drug antibodies (ADA), antidrug antibody neutralizing antibody (ADA-NA), and immunogenic reaction blood draws were added to increase the scope of specialty lab testing.

    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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