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    Clinical Trial Results:
    A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001

    Summary
    EudraCT number
    		 2021-000078-27
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    16 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

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    SRP-9001-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03769116
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Sarepta Therapeutics, Inc.
    Sponsor organisation address
    215 First Street, Cambridge, MA, United States, 02142
    Public contact
    Medical Director, Sarepta Therapeutics, Inc., 1 888-727-3782, SareptAlly@sarepta.com
    Scientific contact
    Medical Director, Sarepta Therapeutics, Inc., 1 888-727-3782, SareptAlly@sarepta.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002677-PIP01-19
    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
    16 Aug 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Aug 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objectives of this study are the assessments of the safety and efficacy of intravenous administration of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy participants.
    Protection of trial subjects
    Written informed consent from each participant or participant's parent(s) or legal guardian(s), if applicable, and written assent from each participant, if applicable, were obtained before any study-specific screening or baseline period evaluations were performed. The anonymity of participating participant will be maintained to the extent required by applicable laws and in accordance with current Health Insurance Portability and Accountability Act (HIPAA) standards. This study was designed and monitored in accordance with Sponsor procedures, which complied with the ethical principles of Good Clinical Practice (GCP) as required by the major regulatory authorities, and in accordance with the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Dec 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 41
    Worldwide total number of subjects
    41
    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)
    41
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 This was a 3-part clinical study of a single dose of delandistrogene moxeparvovec. Participants were randomized to receive delandistrogene moxeparvovec in one of two 48-week periods (Part 1 or Part 2) via a crossover study design. Part 3 was an open-label follow up.

    Pre-assignment
    Screening details
    		 No participants were excluded as all randomized participants received study drug in either Part 1 or Part 2.

    Period 1
    Period 1 title
    Part 1 - Double Blind
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Carer, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Delandistrogene Moxeparvovec Switched Over to Placebo
    Arm description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    delandistrogene moxeparvovec
    Investigational medicinal product code
    SRP-9001
    Other name
    SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Single intravenous (IV) infusion

    Arm title
    		 Placebo Switched Over to Delandistrogene Moxeparvovec
    Arm description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Lactated Ringer’s solution
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Single IV infusion

    Number of subjects in period 1
    		Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Started
    20
    21
    Received At Least 1 Dose Of Study Drug
    20
    21
    Completed
    20
    21
    Period 2
    Period 2 title
    Part 2 - Double Blind
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Delandistrogene Moxeparvovec Switched Over to Placebo
    Arm description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Lactated Ringer’s solution
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Single IV infusion

    Arm title
    		 Placebo Switched Over to Delandistrogene Moxeparvovec
    Arm description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 Experimental

    Investigational medicinal product name
    delandistrogene moxeparvovec
    Investigational medicinal product code
    SRP-9001
    Other name
    SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Single IV infusion

    Number of subjects in period 2
    		Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Started
    20
    21
    Received At Least 1 Dose Of Study Drug
    20
    21
    Completed
    20
    21
    Period 3
    Period 3 title
    Part 3 - Open Label
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Delandistrogene Moxeparvovec Switched Over to Placebo
    Arm description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 Placebo Switched Over to Delandistrogene Moxeparvovec
    Arm description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 3
    		Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Started
    20
    21
    Completed
    17
    19
    Not completed
    3
    2
         Physician decision
    1
    -
         Consent withdrawn by subject
    1
    -
         Study Terminated by Sponsor
    1
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec Total
    Number of subjects
    		 20 21 41
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 20 21 41
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 0 0 0
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 6.29 ( 1.19 ) 6.24 ( 1.13 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 0 0 0
        Male
    		 20 21 41
    Race (NIH/OMB)
    NIH/OMB = National Institutes of Health/Office of Management and Budget
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 4 1 5
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 13 17 30
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 3 3 6
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 1 4 5
        Not Hispanic or Latino
    		 19 16 35
        Unknown or Not Reported
    		 0 1 1
    North Star Ambulatory Assessment (NSAA) Group
    Median score = 21
    Units: Subjects
        NSAA baseline total score ≥ median score
    		 8 15 23
        NSAA baseline total score < median score
    		 12 6 18

    End points

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    End points reporting groups
    Reporting group title
    Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Reporting group title
    Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.
    Reporting group title
    Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants received delandistrogene moxeparvovec at Part 1 followed by placebo at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Subject analysis set title
    Less than 1 Year after Delandistrogene Moxeparvovec Infusion
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2.

    Subject analysis set title
    1 to 2 Years after Delandistrogene Moxeparvovec Infusion
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2.

    Subject analysis set title
    2 to 3 Years after Delandistrogene Moxeparvovec Infusion
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2.

    Subject analysis set title
    3 to 4 Years after Delandistrogene Moxeparvovec Infusion
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2 and who were evaluable.

    Subject analysis set title
    More than 4 Years after Delandistrogene Moxeparvovec Infusion
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2 and who were evaluable.

    Subject analysis set title
    Intent-to-Treat (ITT) Population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    All randomized participants who received study treatment (delandistrogene moxeparvovec or placebo) during Part 1.

    Subject analysis set title
    Safety Population
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All participants who received study treatment during Part 1 or Part 2, with treatment group designated according to the treatment that they actually received.

    Primary: Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content

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    End point title
    		 Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
    End point description
    Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Week 12) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined by Western Blot. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1.
    End point type
    Primary
    End point timeframe
    Baseline, Week 12 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    20
    21
    Units: Percent Normal
    arithmetic mean (standard deviation)
        Baseline
    4.23 ( 6.83 )
    1.91 ( 1.28 )
        Change from Baseline
    23.82 ( 39.76 )
    0.14 ( 1.24 )
    Statistical analysis title
    		 Western Blot Adjusted by Muscle Content Analysis
    Statistical analysis description
    Analysis conducted on changes from baseline.
    Comparison groups
    Delandistrogene Moxeparvovec Switched Over to Placebo v Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001
    Method
    		 Wilcoxon rank sum test
    Parameter type
    		 Hodges-Lehmann treatment diff
    Point estimate
    6.11
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.08
         upper limit
    		 12.58

    Primary: Change From Baseline at Week 48 in NSAA Total Score

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    End point title
    		 Change From Baseline at Week 48 in NSAA Total Score
    End point description
    The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
    End point type
    Primary
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19 [1]
    21 [2]
    Units: Score on a scale
        least squares mean (standard error)
    1.7 ( 0.6 )
    0.9 ( 0.6 )
    Notes
    [1] - ITT - those who were evaluable for this end point
    [2] - ITT
    Statistical analysis title
    		 NSAA Total Score Analysis
    Comparison groups
    Delandistrogene Moxeparvovec Switched Over to Placebo v Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.373
    Method
    		 Mixed-model for Repeated Measures
    Parameter type
    		 LSM Change Difference
    Point estimate
    0.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1
         upper limit
    		 2.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.9

    Secondary: Change From Baseline at Week 48 in Time to Ascend 4 Steps

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    End point title
    		 Change From Baseline at Week 48 in Time to Ascend 4 Steps
    End point description
    This functional assessment measures the time taken to climb 4 steps. All functional assessments were administered by a clinical evaluator. A decrease in time indicates an improvement in motor function. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this end point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19
    21
    Units: second
        least squares mean (standard error)
    0.17 ( 0.26 )
    0.03 ( 0.26 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Week 48 in Time of 100-meter Timed Test

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    End point title
    		 Change From Baseline at Week 48 in Time of 100-meter Timed Test
    End point description
    This functional assessment measures the time needed to move 100 meters. All functional assessments were administered by a clinical evaluator. A decrease in time indicates an improvement in motor function. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this end point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19
    21
    Units: second
        least squares mean (standard error)
    4.29 ( 3.92 )
    6.28 ( 3.83 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Week 48 in Time of 10-meter Timed Test

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    End point title
    		 Change From Baseline at Week 48 in Time of 10-meter Timed Test
    End point description
    This functional assessment measures the time needed to move 10 meters. All functional assessments were administered by a clinical evaluator. A decrease in time indicates an improvement in motor function. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this end point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19
    21
    Units: second
        least squares mean (standard error)
    0.59 ( 0.20 )
    0.10 ( 0.19 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Week 48 in Time to Rise From the Floor

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    End point title
    		 Change From Baseline at Week 48 in Time to Rise From the Floor
    End point description
    This functional assessment was performed as a part of the NSAA and measures the time taken to rise from supine to standing. All functional assessments were administered by a clinical evaluator. A decrease in time indicates an improvement in motor function. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this end point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19
    21
    Units: second
        least squares mean (standard error)
    -0.15 ( 0.25 )
    0.35 ( 0.23 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)

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    End point title
    		 Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)
    End point description
    Baseline muscle biopsies were performed pre-treatment and post-treatment (Week 12) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined by IF PDPF. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing dystrophin was quantified by independent trained evaluators. An increase in IF PDPF indicates increased delandistrogene moxeparvovec dystrophin expression. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    20
    21
    Units: percent dystrophin positive fibers
        arithmetic mean (standard deviation)
    23.88 ( 25.58 )
    5.09 ( 12.96 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity

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    End point title
    		 Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity
    End point description
    Baseline muscle biopsies were performed pre-treatment and post-treatment (Week 12) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined using IF fiber intensity. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing dystrophin was quantified by independent trained evaluators. An increase in IF fiber intensity indicates increased delandistrogene moxeparvovec dystrophin expression. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    20
    21
    Units: percent fluorescent expression
        arithmetic mean (standard deviation)
    0.06 ( 0.11 )
    0.00 ( 0.02 )
    No statistical analyses for this end point

    Secondary: Participants Experiencing Adverse Events (AEs) Since Treatment with Delandistrogene Moxeparvovec

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    End point title
    		 Participants Experiencing Adverse Events (AEs) Since Treatment with Delandistrogene Moxeparvovec
    End point description
    Participants experiencing AEs are reported based upon the time to AE onset after delandistrogene moxeparvovec infusion. Each analysis set includes data from both arms and is based upon the Delandistrogene Moxeparvovec-treated Population: all participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2. ‘Delandistrogene Moxeparvovec switched over to Placebo’ participants received delandistrogene moxeparvovec in Part 1, followed by placebo in Part 2. AEs experienced by participants during Parts 1, 2, and 3 of the study are reported. ‘Placebo switched over to Delandistrogene Moxeparvovec’ participants received placebo in Part 1, followed by delandistrogene moxeparvovec in Part 2. AEs experienced by participants during Parts 2 and 3 of the study are reported. No intervention was administered during Part 3. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
    End point type
    Secondary
    End point timeframe
    Day 1 through Week 130
    End point values
    		 Less than 1 Year after Delandistrogene Moxeparvovec Infusion 1 to 2 Years after Delandistrogene Moxeparvovec Infusion 2 to 3 Years after Delandistrogene Moxeparvovec Infusion 3 to 4 Years after Delandistrogene Moxeparvovec Infusion More than 4 Years after Delandistrogene Moxeparvovec Infusion
    Number of subjects analysed
    41
    41
    41
    30
    12
    Units: participants
        Treatment-emergent AE (TEAE)
    41
    37
    33
    17
    3
        Serious AE (SAE)
    5
    1
    2
    0
    0
        Treatment-related TEAE
    37
    2
    1
    0
    0
        Treatment-related SAE
    3
    0
    0
    0
    0
        Any AE leading to study discontinuation
    0
    0
    0
    0
    0
        Death
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Other pre-specified: Baseline NSAA Total Score by Age Group

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    End point title
    		 Baseline NSAA Total Score by Age Group
    End point description
    The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). This end point presents only the baseline NSAA total score of the ITT population by the following age groups: 4-5 years old; 6-7 years old. Reported results based upon the ITT Population: all randomized participants who received study treatment during Part 1.
    End point type
    Other pre-specified
    End point timeframe
    Baseline
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    20
    21
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Age Group: 4-5 years old
    20.1 ( 1.9 )
    20.4 ( 2.7 )
        Age Group: 6-7 years old
    19.6 ( 4.1 )
    24.0 ( 2.9 )
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline at Week 48 in NSAA Total Score by Age Group

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    End point title
    		 Change From Baseline at Week 48 in NSAA Total Score by Age Group
    End point description
    The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 48 (Part 1)
    End point values
    		 Delandistrogene Moxeparvovec Switched Over to Placebo Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects analysed
    19 [3]
    21 [4]
    Units: Score on a scale
    least squares mean (standard error)
        Age Group: 4-5 years old
    4.3 ( 0.6 )
    1.9 ( 0.6 )
        Age Group: 6-7 years old
    -0.2 ( 0.7 )
    0.5 ( 0.7 )
    Notes
    [3] - ITT - those who were evaluable for this end point
    [4] - ITT
    Statistical analysis title
    		 NSAA Total Score Analysis: 6-7 Years Old
    Statistical analysis description
    Change from baseline - Age Group: 6-7 years old
    Comparison groups
    Delandistrogene Moxeparvovec Switched Over to Placebo v Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.5384
    Method
    		 Mixed-model for Repeated Measures
    Parameter type
    		 LSM Change Difference
    Point estimate
    -0.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3
         upper limit
    		 1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.1
    Statistical analysis title
    		 NSAA Total Score Analysis: 4-5 Years Old
    Statistical analysis description
    Change from baseline - Age Group: 4-5 years old
    Comparison groups
    Delandistrogene Moxeparvovec Switched Over to Placebo v Placebo Switched Over to Delandistrogene Moxeparvovec
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0172
    Method
    		 Mixed-model for Repeated Measures
    Parameter type
    		 LSM Change Difference
    Point estimate
    2.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.5
         upper limit
    		 4.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.9

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 through Week 130
    Adverse event reporting additional description
    This was a 3-part clinical study of a single dose of delandistrogene moxeparvovec. Participants were randomized to receive delandistrogene moxeparvovec in one of two 48-week periods (Part 1 or Part 2) via a crossover study design. Part 3 was an open-label follow up. All reported adverse events are based upon the Safety Population.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    Part 1: Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received delandistrogene moxeparvovec during Part 1.

    Reporting group title
    Part 1: Placebo
    Reporting group description
    		 Participants received placebo during Part 1.

    Reporting group title
    Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants received matching placebo during Part 1 followed by delandistrogene moxeparvovec during Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec
    Reporting group description
    		 Participants who received placebo during Part 1 received delandistrogene moxeparvovec during Part 2.

    Reporting group title
    Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants received delandistrogene moxeparvovec during Part 1 followed by matching placebo during Part 2 followed by an open-label extension at Part 3. No intervention was administered during Part 3.

    Reporting group title
    Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo
    Reporting group description
    		 Participants who received delandistrogene moxeparvovec during Part 1 received placebo during Part 2.

    Serious adverse events
    		 Part 1: Delandistrogene Moxeparvovec Part 1: Placebo Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 20 (15.00%)
    2 / 21 (9.52%)
    2 / 21 (9.52%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Humerus fracture
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Liver injury
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Rhabdomyolysis
         subjects affected / exposed
    2 / 20 (10.00%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    1 / 1
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part 1: Delandistrogene Moxeparvovec Part 1: Placebo Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    20 / 20 (100.00%)
    21 / 21 (100.00%)
    19 / 21 (90.48%)
    21 / 21 (100.00%)
    18 / 20 (90.00%)
    20 / 20 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    2
    0
    0
    2
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 20 (20.00%)
    1 / 21 (4.76%)
    3 / 21 (14.29%)
    6 / 21 (28.57%)
    1 / 20 (5.00%)
    2 / 20 (10.00%)
         occurrences all number
    5
    1
    3
    6
    1
    2
    Vessel puncture site haemorrhage
         subjects affected / exposed
    2 / 20 (10.00%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    1
    0
    2
    0
    0
    Infusion site extravasation
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    3 / 21 (14.29%)
    0 / 20 (0.00%)
    3 / 20 (15.00%)
         occurrences all number
    1
    0
    2
    3
    0
    3
    Catheter site haemorrhage
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    0
    2
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    9 / 20 (45.00%)
    6 / 21 (28.57%)
    4 / 21 (19.05%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
    3 / 20 (15.00%)
         occurrences all number
    10
    8
    4
    1
    2
    3
    Rhinorrhoea
         subjects affected / exposed
    4 / 20 (20.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    3 / 20 (15.00%)
    4 / 20 (20.00%)
         occurrences all number
    5
    3
    0
    2
    5
    4
    Nasal congestion
         subjects affected / exposed
    3 / 20 (15.00%)
    2 / 21 (9.52%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    3
    2
    2
    0
    0
    0
    Epistaxis
         subjects affected / exposed
    2 / 20 (10.00%)
    3 / 21 (14.29%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    6
    1
    6
    1
    0
    Psychiatric disorders
    Irritability
         subjects affected / exposed
    3 / 20 (15.00%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    9 / 21 (42.86%)
    1 / 20 (5.00%)
    5 / 20 (25.00%)
         occurrences all number
    3
    1
    2
    9
    1
    5
    Sleep disorder
         subjects affected / exposed
    3 / 20 (15.00%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    3
    1
    1
    1
    0
    2
    Anxiety
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    1
    2
    0
    1
    Affect lability
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    1
    1
    2
    0
    0
    Investigations
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    5 / 20 (25.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    6 / 21 (28.57%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    5
    0
    0
    6
    0
    0
    Blood bilirubin increased
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    2
    0
    0
    Weight increased
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    2
    0
    0
    2
    0
    2
    Glutamate dehydrogenase increased
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    3 / 21 (14.29%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    White blood cell count decreased
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    0
    5
    0
    0
    Injury, poisoning and procedural complications
    Procedural pain
         subjects affected / exposed
    5 / 20 (25.00%)
    7 / 21 (33.33%)
    2 / 21 (9.52%)
    9 / 21 (42.86%)
    0 / 20 (0.00%)
    6 / 20 (30.00%)
         occurrences all number
    5
    7
    2
    9
    0
    6
    Skin abrasion
         subjects affected / exposed
    4 / 20 (20.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    4
    3
    0
    1
    0
    2
    Limb injury
         subjects affected / exposed
    3 / 20 (15.00%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    3
    1
    1
    0
    0
    1
    Incision site haemorrhage
         subjects affected / exposed
    6 / 20 (30.00%)
    4 / 21 (19.05%)
    0 / 21 (0.00%)
    5 / 21 (23.81%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    6
    5
    0
    5
    0
    2
    Arthropod bite
         subjects affected / exposed
    1 / 20 (5.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    3
    0
    1
    0
    1
    Post procedural contusion
         subjects affected / exposed
    1 / 20 (5.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    1
    2
    0
    2
    0
    2
    Craniocerebral injury
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Lip injury
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Sunburn
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Cardiac disorders
    Cardiomyopathy
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    0
    2
    0
    2
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 20 (15.00%)
    6 / 21 (28.57%)
    3 / 21 (14.29%)
    3 / 21 (14.29%)
    1 / 20 (5.00%)
    3 / 20 (15.00%)
         occurrences all number
    3
    13
    7
    5
    1
    7
    Lethargy
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    3 / 21 (14.29%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    1
    2
    0
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    5 / 21 (23.81%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    0
    5
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    2 / 20 (10.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    2
    4
    0
    0
    0
    1
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    7 / 20 (35.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    11 / 21 (52.38%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    9
    3
    0
    14
    0
    3
    Vomiting
         subjects affected / exposed
    13 / 20 (65.00%)
    7 / 21 (33.33%)
    3 / 21 (14.29%)
    16 / 21 (76.19%)
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    26
    13
    3
    33
    1
    0
    Diarrhoea
         subjects affected / exposed
    3 / 20 (15.00%)
    2 / 21 (9.52%)
    2 / 21 (9.52%)
    3 / 21 (14.29%)
    2 / 20 (10.00%)
    2 / 20 (10.00%)
         occurrences all number
    3
    2
    2
    3
    2
    2
    Constipation
         subjects affected / exposed
    3 / 20 (15.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    3
    0
    0
    2
    2
    0
    Abdominal pain upper
         subjects affected / exposed
    6 / 20 (30.00%)
    4 / 21 (19.05%)
    0 / 21 (0.00%)
    8 / 21 (38.10%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    6
    4
    0
    10
    0
    2
    Abdominal pain
         subjects affected / exposed
    5 / 20 (25.00%)
    2 / 21 (9.52%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    6
    5
    1
    2
    1
    1
    Toothache
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    4 / 21 (19.05%)
    2 / 20 (10.00%)
    2 / 20 (10.00%)
         occurrences all number
    1
    0
    1
    5
    2
    2
    Skin and subcutaneous tissue disorders
    Ecchymosis
         subjects affected / exposed
    7 / 20 (35.00%)
    4 / 21 (19.05%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    4 / 20 (20.00%)
         occurrences all number
    10
    4
    0
    2
    0
    4
    Dermatitis
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Urticaria
         subjects affected / exposed
    0 / 20 (0.00%)
    3 / 21 (14.29%)
    1 / 21 (4.76%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    3
    1
    3
    0
    0
    Rash
         subjects affected / exposed
    1 / 20 (5.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    3
    0
    2
    0
    1
    Dermatitis contact
         subjects affected / exposed
    0 / 20 (0.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    3
    0
    1
    0
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Pollakiuria
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    0
    0
    0
    1
    0
    Proteinuria
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 21 (4.76%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    1
    0
    2
    0
    0
    Ketonuria
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    0
    0
    3
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    1 / 21 (4.76%)
    1 / 20 (5.00%)
    2 / 20 (10.00%)
         occurrences all number
    0
    0
    2
    1
    1
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    5 / 20 (25.00%)
    1 / 21 (4.76%)
    3 / 21 (14.29%)
    2 / 21 (9.52%)
    0 / 20 (0.00%)
    2 / 20 (10.00%)
         occurrences all number
    6
    1
    3
    2
    0
    2
    Pain in extremity
         subjects affected / exposed
    5 / 20 (25.00%)
    5 / 21 (23.81%)
    4 / 21 (19.05%)
    6 / 21 (28.57%)
    3 / 20 (15.00%)
    7 / 20 (35.00%)
         occurrences all number
    6
    7
    4
    7
    3
    8
    Back pain
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 21 (4.76%)
    3 / 21 (14.29%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    1
    4
    1
    2
    1
    Myalgia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    0
    2
    1
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    13 / 20 (65.00%)
    13 / 21 (61.90%)
    9 / 21 (42.86%)
    7 / 21 (33.33%)
    9 / 20 (45.00%)
    8 / 20 (40.00%)
         occurrences all number
    27
    29
    18
    9
    18
    9
    Viral infection
         subjects affected / exposed
    8 / 20 (40.00%)
    9 / 21 (42.86%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    2 / 20 (10.00%)
    3 / 20 (15.00%)
         occurrences all number
    10
    11
    3
    0
    2
    4
    Gastroenteritis
         subjects affected / exposed
    3 / 20 (15.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    3
    2
    0
    1
    0
    0
    Sinusitis
         subjects affected / exposed
    2 / 20 (10.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
    0 / 20 (0.00%)
         occurrences all number
    2
    2
    0
    0
    1
    0
    Otitis media
         subjects affected / exposed
    1 / 20 (5.00%)
    3 / 21 (14.29%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    1
    3
    0
    0
    1
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Rhinovirus infection
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    2
    0
    0
    0
    0
    1
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    1
    2
    0
    0
    0
    Influenza
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 21 (0.00%)
    3 / 21 (14.29%)
    1 / 21 (4.76%)
    2 / 20 (10.00%)
    0 / 20 (0.00%)
         occurrences all number
    1
    0
    3
    1
    3
    0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 21 (4.76%)
    4 / 21 (19.05%)
    3 / 21 (14.29%)
    3 / 20 (15.00%)
    2 / 20 (10.00%)
         occurrences all number
    3
    1
    5
    3
    4
    3
    COVID-19
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    9 / 21 (42.86%)
    2 / 21 (9.52%)
    9 / 20 (45.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    11
    2
    9
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    8 / 20 (40.00%)
    0 / 21 (0.00%)
    0 / 21 (0.00%)
    15 / 21 (71.43%)
    0 / 20 (0.00%)
    1 / 20 (5.00%)
         occurrences all number
    10
    0
    0
    18
    0
    1
    Increased appetite
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    4 / 21 (19.05%)
    0 / 20 (0.00%)
    3 / 20 (15.00%)
         occurrences all number
    0
    2
    0
    4
    0
    3
    Dehydration
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 21 (0.00%)
    2 / 21 (9.52%)
    1 / 21 (4.76%)
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         occurrences all number
    0
    0
    2
    1
    0
    0
    Vitamin D deficiency
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 21 (4.76%)
    2 / 21 (9.52%)
    0 / 21 (0.00%)
    1 / 20 (5.00%)
    1 / 20 (5.00%)
         occurrences all number
    0
    1
    2
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 Jun 2019
    - Increased the sample size from 24 participants to 44 participants in order to increase the power of the study regarding the NSAA end point. Note: This amendment (Amendment 3) was internally finalized on 04 June 2019 and was sent to sites; however, this amendment was not implemented or submitted to any regulatory agency. Before submitting to the Food and Drug Administration, the Sponsor decided that additional changes were needed, and since the site had already received a version labeled Amendment 3, to avoid confusion the updated version was labeled Amendment 4 when submitted and included additional changes.
    02 Jul 2019
    - Removed the requirement to use an enzyme-linked immunospot assay as a means of guiding the post-infusion steroid taper - Updated the sample size determination text - Added a Week 10 visit to both parts of the study
    25 Sep 2019
    - Elevated the assessment of ambulatory function as measured by NSAA (total score) to a co-primary end point - Updated language around steroid dose tapering - Added language regarding adverse events of special interest - Clarified the dose concentration to align with the Investigator’s Brochure and added explanation of the update to the quantitative polymerase chain reaction method for titering (Sponsor’s method using a linear standard)
    27 Aug 2020
    - Extended the long-term safety follow-up to 5 years and split the study into 3 parts (two 48-week double-blind periods and one open-label follow-up period) - Defined evaluation of delandistrogene moxeparvovec dystrophin expression from delandistrogene moxeparvovec at 12 weeks post-dosing and effect of delandistrogene moxeparvovec on physical functional assessments as assessed by the NSAA as primary end points (instead of co-primary) - Added a long-term safety and efficacy objective with corresponding end points for Part 3 (open-label extension) of the study
    12 Apr 2023
    - Provided participants in Part 3 of the study with the option of continuing their follow-up in a long-term extension study. The long-term extension study was designed to provide a uniform approach to monitoring long-term safety and efficacy in participants who received an infusion of delandistrogene moxeparvovec in a clinical trial. Participants must have completed the Week 130 visit at a minimum before rolling over into the long-term extension study. After completion of the Week 130 visit, if participants decided not to continue their follow-up in the long-term extension study, participants completed an end of study visit.

    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.
    Caution should be taken in interpreting the treatment effect on the 6-7 years-old age group due to differences in baseline prognostic functional characteristics for certain assessments in treated versus placebo in Part 1.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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