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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 Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD)

    Summary
    EudraCT number
    2020-000699-39
    Trial protocol
    FR   BE   AT   NL   IT   ES  
    Global end of trial date
    14 Dec 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    31 Aug 2024
    First version publication date
    31 Aug 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    FGCL-3019-094
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04632940
    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., FG3019-094DMDStudy@fibrogen.com
    Scientific contact
    Clinical Trial Information Desk, FibroGen, Inc., FG3019-094DMDStudy@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
    14 Dec 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Dec 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 administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <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 IEC requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    03 Mar 2021
    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
    Austria: 2
    Country: Number of subjects enrolled
    Belgium: 4
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    China: 17
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Italy: 7
    Country: Number of subjects enrolled
    Netherlands: 2
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    United States: 30
    Worldwide total number of subjects
    73
    EEA total number of subjects
    24
    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)
    69
    Adolescents (12-17 years)
    4
    Adults (18-64 years)
    0
    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: a Double-blind (DB) period and an Open-label extension (OLE) period.

    Period 1
    Period 1 title
    DB 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 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    FG-3019
    Other name
    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 by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.
    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
    37
    36
    Received at least 1 dose of study drug
    36
    36
    Completed
    35
    36
    Not completed
    2
    0
         Participant/Legal Guardian Decision
    2
    -
    Period 2
    Period 2 title
    OLE Period (52 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 continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
    Arm type
    Experimental

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    FG-3019
    Other name
    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/Pamrevlumab
    Arm description
    Participants who received placebo in the DB period, received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
    Arm type
    Placebo

    Investigational medicinal product name
    Pamrevlumab
    Investigational medicinal product code
    FG-3019
    Other name
    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/Pamrevlumab
    Started
    34
    35
    Received at least 1 dose of study drug
    34
    34
    Completed
    0
    0
    Not completed
    34
    35
         Physician decision
    1
    -
         Adverse event, non-fatal
    1
    -
         Sponsor Decision to Terminate Study
    21
    19
         Other than specified
    -
    1
         Lost to follow-up
    -
    1
         Participant/Legal Guardian Decision
    11
    13
         Entered into OLE but not treated
    -
    1
    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: 2 participants who completed DB period, did not roll over into the OLE period.

    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 + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.

    Reporting group values
    Pamrevlumab Placebo Total
    Number of subjects
    37 36 73
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    9.1 ( 1.46 ) 9.0 ( 1.56 ) -
    Sex: Female, Male
    Units: participants
        Female
    0 0 0
        Male
    37 36 73
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    1 5 6
        Not Hispanic or Latino
    34 28 62
        Unknown or Not Reported
    2 3 5
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0
        Asian
    10 9 19
        Native Hawaiian or Other Pacific Islander
    0 0 0
        Black or African American
    1 1 2
        White
    23 22 45
        More than one race
    0 0 0
        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 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.
    Reporting group title
    Pamrevlumab
    Reporting group description
    Participants continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.

    Reporting group title
    Placebo/Pamrevlumab
    Reporting group description
    Participants who received placebo in the DB period, received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.

    Primary: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

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    End point title
    Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
    End point description
    The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM). The ITT set included all randomized participants. 'Overall number of participants analyzed' = participants evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    36
    36
    Units: units on a scale
        least squares mean (standard error)
    -3.022 ( 0.5505 )
    -2.494 ( 0.6962 )
    Statistical analysis title
    Statistical Analysis 1
    Comparison groups
    Pamrevlumab v Placebo
    Number of subjects included in analysis
    72
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.5553
    Method
    Mixed models analysis
    Parameter type
    LS Mean Difference
    Point estimate
    -0.528
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.308
         upper limit
    1.251
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.8912

    Secondary: Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52

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    End point title
    Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
    End point description
    The 4SCV (centimeters [cm]/second [sec]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb. The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    36
    36
    Units: cm/sec
        arithmetic mean (standard deviation)
    -1.858 ( 4.5459 )
    -3.797 ( 5.1899 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in the 10-Meter Walk/Run Test at Week 52

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    End point title
    Change From Baseline in the 10-Meter Walk/Run Test at Week 52
    End point description
    The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec). The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    36
    36
    Units: meters/sec
        arithmetic mean (standard deviation)
    -0.176 ( 0.2193 )
    -0.196 ( 0.3552 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Stand (TTSTAND) at Week 52

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    End point title
    Change From Baseline in Time to Stand (TTSTAND) at Week 52
    End point description
    The time (in sec) required for a participant to stand from a supine position has been reported. A longer time taken reflected a worse outcome. The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    36
    31
    Units: sec
        arithmetic mean (standard deviation)
    2.24 ( 3.353 )
    1.94 ( 3.688 )
    No statistical analyses for this end point

    Secondary: Time to Loss of Ambulation (LoA) From Baseline to Week 52

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    End point title
    Time to Loss of Ambulation (LoA) From Baseline to Week 52
    End point description
    Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates. The ITT set included all randomized participants. Here, '99999' signifies 'Due to insufficient number of participants with an event of LoA or death, median and 95% confidence interval (CI) could not be calculated.'
    End point type
    Secondary
    End point timeframe
    Baseline to Week 52
    End point values
    Pamrevlumab Placebo
    Number of subjects analysed
    37
    36
    Units: days
        median (confidence interval 95%)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    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 129
    Adverse event reporting additional description
    As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for 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: Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.

    Reporting group title
    OLE Period: Pamrevlumab
    Reporting group description
    Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.

    Reporting group title
    DB Period: Placebo
    Reporting group description
    Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.

    Serious adverse events
    DB Period: Pamrevlumab OLE Period: Pamrevlumab DB Period: Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 36 (8.33%)
    0 / 68 (0.00%)
    1 / 36 (2.78%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Cardiac disorders
    Myocarditis
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 68 (0.00%)
    0 / 36 (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
    Musculoskeletal and connective tissue disorders
    Knee deformity
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 68 (0.00%)
    0 / 36 (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
    Rhabdomyolysis
         subjects affected / exposed
    0 / 36 (0.00%)
    0 / 68 (0.00%)
    1 / 36 (2.78%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 68 (0.00%)
    0 / 36 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    DB Period: Pamrevlumab OLE Period: Pamrevlumab DB Period: Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    35 / 36 (97.22%)
    45 / 68 (66.18%)
    33 / 36 (91.67%)
    Investigations
    Blood pressure diastolic decreased
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 68 (0.00%)
    0 / 36 (0.00%)
         occurrences all number
    2
    0
    0
    SARS-CoV-2 test positive
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 68 (0.00%)
    1 / 36 (2.78%)
         occurrences all number
    2
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 36 (5.56%)
    1 / 68 (1.47%)
    1 / 36 (2.78%)
         occurrences all number
    3
    1
    1
    Ligament sprain
         subjects affected / exposed
    1 / 36 (2.78%)
    1 / 68 (1.47%)
    3 / 36 (8.33%)
         occurrences all number
    1
    1
    3
    Joint injury
         subjects affected / exposed
    3 / 36 (8.33%)
    0 / 68 (0.00%)
    0 / 36 (0.00%)
         occurrences all number
    3
    0
    0
    Fall
         subjects affected / exposed
    7 / 36 (19.44%)
    3 / 68 (4.41%)
    2 / 36 (5.56%)
         occurrences all number
    10
    3
    2
    Vascular access site pain
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 68 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    1
    0
    2
    Spinal compression fracture
         subjects affected / exposed
    0 / 36 (0.00%)
    1 / 68 (1.47%)
    2 / 36 (5.56%)
         occurrences all number
    0
    1
    2
    Nervous system disorders
    Hypokinesia
         subjects affected / exposed
    0 / 36 (0.00%)
    0 / 68 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    0
    2
    Headache
         subjects affected / exposed
    14 / 36 (38.89%)
    5 / 68 (7.35%)
    5 / 36 (13.89%)
         occurrences all number
    40
    6
    12
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    4 / 36 (11.11%)
    2 / 68 (2.94%)
    5 / 36 (13.89%)
         occurrences all number
    6
    2
    5
    Gait disturbance
         subjects affected / exposed
    0 / 36 (0.00%)
    0 / 68 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    0
    2
    Injection site bruising
         subjects affected / exposed
    0 / 36 (0.00%)
    1 / 68 (1.47%)
    2 / 36 (5.56%)
         occurrences all number
    0
    6
    13
    Pyrexia
         subjects affected / exposed
    9 / 36 (25.00%)
    10 / 68 (14.71%)
    7 / 36 (19.44%)
         occurrences all number
    16
    11
    8
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 36 (0.00%)
    0 / 68 (0.00%)
    2 / 36 (5.56%)
         occurrences all number
    0
    0
    2
    Abdominal pain
         subjects affected / exposed
    5 / 36 (13.89%)
    2 / 68 (2.94%)
    3 / 36 (8.33%)
         occurrences all number
    6
    5
    5
    Abdominal pain upper
         subjects affected / exposed
    2 / 36 (5.56%)
    3 / 68 (4.41%)
    1 / 36 (2.78%)
         occurrences all number
    2
    6
    1
    Constipation
         subjects affected / exposed
    0 / 36 (0.00%)
    3 / 68 (4.41%)
    2 / 36 (5.56%)
         occurrences all number
    0
    3
    2
    Diarrhoea
         subjects affected / exposed
    8 / 36 (22.22%)
    6 / 68 (8.82%)
    3 / 36 (8.33%)
         occurrences all number
    12
    6
    7
    Nausea
         subjects affected / exposed
    2 / 36 (5.56%)
    4 / 68 (5.88%)
    3 / 36 (8.33%)
         occurrences all number
    2
    6
    3
    Vomiting
         subjects affected / exposed
    10 / 36 (27.78%)
    12 / 68 (17.65%)
    7 / 36 (19.44%)
         occurrences all number
    10
    16
    11
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    6 / 36 (16.67%)
    10 / 68 (14.71%)
    4 / 36 (11.11%)
         occurrences all number
    6
    15
    4
    Epistaxis
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 68 (0.00%)
    3 / 36 (8.33%)
         occurrences all number
    10
    0
    4
    Nasal congestion
         subjects affected / exposed
    3 / 36 (8.33%)
    1 / 68 (1.47%)
    6 / 36 (16.67%)
         occurrences all number
    3
    1
    10
    Oropharyngeal pain
         subjects affected / exposed
    2 / 36 (5.56%)
    3 / 68 (4.41%)
    6 / 36 (16.67%)
         occurrences all number
    2
    3
    7
    Rhinorrhoea
         subjects affected / exposed
    4 / 36 (11.11%)
    4 / 68 (5.88%)
    3 / 36 (8.33%)
         occurrences all number
    6
    4
    8
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    3 / 36 (8.33%)
    0 / 68 (0.00%)
    0 / 36 (0.00%)
         occurrences all number
    3
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 36 (11.11%)
    1 / 68 (1.47%)
    2 / 36 (5.56%)
         occurrences all number
    5
    1
    2
    Back pain
         subjects affected / exposed
    4 / 36 (11.11%)
    2 / 68 (2.94%)
    2 / 36 (5.56%)
         occurrences all number
    4
    2
    2
    Myalgia
         subjects affected / exposed
    2 / 36 (5.56%)
    3 / 68 (4.41%)
    1 / 36 (2.78%)
         occurrences all number
    6
    3
    1
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 36 (0.00%)
    1 / 68 (1.47%)
    2 / 36 (5.56%)
         occurrences all number
    0
    1
    2
    Pain in extremity
         subjects affected / exposed
    4 / 36 (11.11%)
    3 / 68 (4.41%)
    2 / 36 (5.56%)
         occurrences all number
    4
    5
    3
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    2 / 36 (5.56%)
    3 / 68 (4.41%)
    1 / 36 (2.78%)
         occurrences all number
    3
    4
    1
    Ear infection
         subjects affected / exposed
    1 / 36 (2.78%)
    1 / 68 (1.47%)
    4 / 36 (11.11%)
         occurrences all number
    1
    1
    4
    COVID-19
         subjects affected / exposed
    16 / 36 (44.44%)
    4 / 68 (5.88%)
    8 / 36 (22.22%)
         occurrences all number
    17
    4
    9
    Influenza
         subjects affected / exposed
    2 / 36 (5.56%)
    4 / 68 (5.88%)
    1 / 36 (2.78%)
         occurrences all number
    2
    4
    1
    Nasopharyngitis
         subjects affected / exposed
    10 / 36 (27.78%)
    11 / 68 (16.18%)
    7 / 36 (19.44%)
         occurrences all number
    18
    17
    12
    Pharyngitis
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 68 (0.00%)
    0 / 36 (0.00%)
         occurrences all number
    2
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 36 (11.11%)
    6 / 68 (8.82%)
    8 / 36 (22.22%)
         occurrences all number
    4
    11
    10
    Rhinitis
         subjects affected / exposed
    3 / 36 (8.33%)
    5 / 68 (7.35%)
    3 / 36 (8.33%)
         occurrences all number
    4
    6
    6

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Oct 2020
    It included following changes: - The secondary endpoint of time to LoA was added to better assess the impact of pamrevlumab on LoA as a disease progression. - The exploratory endpoint of change in percent predicted forced vital capacity (ppFVC) and percent predicted peak expiratory flow (ppPEF) (assessed by spirometry) from baseline to Week 52 was added to assess the impact of pamrevlumab on pulmonary function. - The exploratory endpoint of change in left ventricular ejection fraction percentage (left ventricular ejection fraction percentage [LVEF%]; assessed by cardiac magnetic resonance imaging [MRI]) from baseline to Week 52 was added to assess the impact of pamrevlumab on cardiac fibrosis, as recommended by the European Medicines Agency (EMA). - The minimum distance requirement for Inclusion Criterion, ability to complete a certain distance during the 6-minute walking distance (6MWD), was changed from 330 meters to 270 meters to increase the population representatives and include a broad range of severities/disease progression rate. - The 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). - More benefit/risk statements were added to provide further information on the risk/benefit of studying pamrevlumab in ambulatory DMD. - The study drug administration time was updated from 24 to 48 hours to align with updated pamrevlumab stability information. - Genetic testing and electrocardiograms were removed. - Laboratory assessments and physical examinations were added to the OLE period. - The muscle fibrosis MRI assessment and endpoint were removed from the OLE period, as they were not a direct measure of participant function and were of uncertain clinical meaningfulness as a biomarker of disease progression.
    26 Oct 2020
    It included following changes: Inclusion criterion was changed to the average (of screening and Day 0) ppFVC above 45%, as opposed to an average ppFVC between 45% to 85%. This change was based on investigator feedback about forced vital capacity limit for ambulatory participants, especially with the shorter minimum distance for the 6MWD test implemented in Amendment 1.
    09 Aug 2021
    It included following changes: - Cardiac MRI was removed as an exploratory endpoint, as it was difficult to measure a meaningful result in this age group, and it was a worthwhile reduction in participant burden. - Acceptable ranges for central laboratory assessments and vaccination requirements were added to increase clarity and specificity related to eligibility. - The exclusion criterion for allergic reactions was expanded to include hypersensitivity to study drug, its components, or gadolinium-based contrast agents required for the MRI. - The recently approved Casimersen (amondys 45) was added to the prohibited medications list. - An exclusion criterion was added to include abnormal glomerular filtration rate (GFR) or evidence of acute kidney injury. - Time points for vital signs were corrected, and methods for temperature collection were defined. A physical examination was added at Week 36 during the OLE period. - Contraception methods were expanded to include condoms. - Clarification was added that the Duchenne Video Assessment (DVA) would not be conducted in Belgium.
    28 Oct 2022
    It included following changes: - Bone fracture and height velocity safety assessments were added to align with the pediatric investigational plan. - The inclusion criterion requiring pneumococcal vaccination was changed from a requirement to a suggestion, as vaccine recommendations varied by country and depended on participant health status. - Removed exclusion criterion of hypersensitivity reaction to gadolinium-based contrast agents required for MRI acquisition as well as language pertaining to contrast MRI, as the cardiac MRI was removed in Amendment 3. Additionally, LVEF% was removed throughout the protocol, as it only pertained to the removed cardiac MRI. - The collection of specific DMD genetic mutation was added to support characterization of the most recent DMD mutation landscape and potentially evaluate possible relationships between participant genotype and clinical outcomes. - Clarification was added that the DVA would not be conducted in China. - Additional pharmacokinetic (PK), antidrug antibody (ADA), and antidrug antibody neutralizing antibody (ADA-NA) blood draws, along with immunogenic reaction blood draws, were added to meet regulatory recommendations for immunogenicity testing, and clarification was added for investigators to follow local guidance for allowable total blood volume.

    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.
    The OLE was discontinued due to study termination by the Sponsor.
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