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Clinical Trial Results:
A Phase 3 Multinational, Randomized, Double-Blind, Placebo Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP 9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)

Summary
EudraCT number	2019-003374-91
Trial protocol	FR BE ES DE IT
Global end of trial date	25 Oct 2024

Results information
Results version number	v1(current)
This version publication date	09 May 2025
First version publication date	09 May 2025
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

SRP-9001-301

ISRCTN number

US NCT number

NCT05096221

WHO universal trial number (UTN)

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

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?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

25 Oct 2024

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

25 Oct 2024

Was the trial ended prematurely?

Main objective of the trial

The primary objective of the trial was to evaluate the effect of SRP 9001 on physical function as assessed by the North Star Ambulatory Assessment (NSAA) score.

Protection of trial subjects

This study was conducted in compliance with the Institutional Review Board (IRB)/Independent Ethics Committee (IEC) regulations, Good Clinical Practice (GCP) Guidelines described in the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) E6 (R2) and 21 Code of Federal Regulations, and applicable national regulations and directives including the European Union (EU) Clinical Practice Directive 2005/28/EC, EU No 536/2014, and Japanese GCP Regulation.

Background therapy

Evidence for comparator

Actual start date of recruitment

27 Oct 2021

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

Number of subjects enrolled per country

Country: Number of subjects enrolled

United States: 80

Country: Number of subjects enrolled

Spain: 15

Country: Number of subjects enrolled

Taiwan: 5

Country: Number of subjects enrolled

Italy: 12

Country: Number of subjects enrolled

United Kingdom: 3

Country: Number of subjects enrolled

Japan: 4

Country: Number of subjects enrolled

Hong Kong: 3

Country: Number of subjects enrolled

Belgium: 2

Country: Number of subjects enrolled

Germany: 1

Worldwide total number of subjects

125

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

125

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Screening details

126 participants were enrolled in the study. 1 of the 126 participants was enrolled via a country-specific protocol addendum. Data were collected but not analyzed for the participant enrolled via the country-specific protocol addendum. Data are presented below for the 125 participants enrolled via the global protocol.

Period 1 title

Part 1

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Delandistrogene Moxeparvovec followed by Placebo

Arm description

Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2.

Arm type

Experimental

Investigational medicinal product name

Delandistrogene moxeparvovec

Investigational medicinal product code

Other name

SRP-9001

Pharmaceutical forms

Solution for infusion

Routes of administration

Intravenous use

Dosage and administration details

Administration per details specified in the arm description.

Placebo followed by Delandistrogene Moxeparvovec

Arm description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Arm type

Experimental

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Saline, 0.9% sodium chloride solution

Pharmaceutical forms

Solution for infusion

Routes of administration

Intravenous use

Dosage and administration details

Administration per details specified in the arm description.

Number of subjects in period 1	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Started	63	62
Received at Least 1 Dose in Part 1	63	62
Completed	63	62

Period 2 title

Part 2

Is this the baseline period?

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Delandistrogene Moxeparvovec followed by Placebo

Arm description

Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2.

Arm type

Experimental

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Saline, 0.9% sodium chloride solution

Pharmaceutical forms

Solution for infusion

Routes of administration

Intravenous use

Dosage and administration details

Administration per details specified in the arm description.

Placebo followed by Delandistrogene Moxeparvovec

Arm description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Arm type

Experimental

Investigational medicinal product name

Delandistrogene moxeparvovec

Investigational medicinal product code

Other name

SRP-9001

Pharmaceutical forms

Solution for infusion

Routes of administration

Intravenous use

Dosage and administration details

Administration per details specified in the arm description.

Number of subjects in period 2 ^[1]	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Started	63	60
Received at Least 1 Dose in Part 2	62 ^[2]	60
Completed	63	59
Not completed	0	1
Physician decision	-	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: Two participants who completed Part 1 discontinued prior to Part 2.
[2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Number of participants who received at least 1 dose in Part 2.

Baseline characteristics

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Reporting group title

Delandistrogene Moxeparvovec followed by Placebo

Reporting group description

Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2.

Reporting group title

Placebo followed by Delandistrogene Moxeparvovec

Reporting group description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Reporting group values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec	Total
Number of subjects	63	62	125
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)	63	62	125
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)	5.98 ( 1.06 )	6.08 ( 1.05 )	-
Sex: Female, Male
Units: participants
Female	0	0	0
Male	63	62	125
Race/Ethnicity, Customized
Units: Subjects
American Indian or Alaska Native	0	0	0
Asian	8	11	19
Native Hawaiian or Other Pacific Islander	0	0	0
Black of African American	0	2	2
White	49	46	95
More than One Race	1	0	1
Other or Not Reported	5	3	8
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	15	8	23
Not Hispanic or Latino	47	53	100
Unknown or Not Reported	1	1	2

End points

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Reporting group title

Delandistrogene Moxeparvovec followed by Placebo

Reporting group description

Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2.

Reporting group title

Placebo followed by Delandistrogene Moxeparvovec

Reporting group description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Reporting group title

Delandistrogene Moxeparvovec followed by Placebo

Reporting group description

Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2.

Reporting group title

Placebo followed by Delandistrogene Moxeparvovec

Reporting group description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

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End point title

Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

End point description

The NSAA is a healthcare provider administered scale. During assessment, participants were asked to perform 17 functional activities 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 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. An increase in score=improvement in motor function. Modified Intent-to-Treat (mITT) Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Number of Subjects Analyzed= number of participants evaluable for this outcome measure.

End point type

Primary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	61
Units: scores on a scale
least squares mean (standard error)	2.57 ( 0.39 )	1.92 ( 0.39 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2441

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

0.65

Confidence interval

level

95%

sides

2-sided

lower limit

-0.45

upper limit

1.74

Variability estimate

Standard error of the mean

Dispersion value

0.55

Secondary: Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content

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End point title

Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content

End point description

Quantity of delandistrogene moxeparvovec dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Part 1, Week 12. 2 blocks of tissues were analyzed by Western blot, each with 2 technical replicates to determine the delandistrogene moxeparvovec dystrophin protein level (percent control). The block average value from 2 technical replicates was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for the analysis. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression =production of the delandistrogene moxeparvovec dystrophin protein. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable.

End point type

Secondary

End point timeframe

Week 12

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	17	14
Units: Percent control
arithmetic mean (standard deviation)	34.29 ( 41.04 )	0.00 ( 0.00 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Re-randomization test

Confidence interval

Secondary: Part 1: Change From Baseline in Time to Rise From the Floor at Week 52

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End point title

Part 1: Change From Baseline in Time to Rise From the Floor at Week 52

End point description

The time to rise from the floor test quantifies the time required for the participant to stand in an upright position with arms by sides, starting from the supine position with arms by sides. Data is presented for the change from baseline to Week 52 in the time taken (in seconds) to rise from the floor. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Subjects"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	61
Units: Seconds
least squares mean (standard error)	-0.27 ( 0.15 )	0.37 ( 0.15 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0025

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

-0.64

Confidence interval

level

95%

sides

2-sided

lower limit

-1.06

upper limit

-0.23

Variability estimate

Standard error of the mean

Dispersion value

0.21

Secondary: Part 1: Change from Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52

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End point title

Part 1: Change from Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52

End point description

The timed 10MWR quantifies the time required for the participant to run or walk 10 meters (on a straight walkway) from a standing position. Data is presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 10MWR. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	61
Units: Seconds
least squares mean (standard error)	-0.34 ( 0.10 )	0.08 ( 0.10 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0048

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

-0.42

Confidence interval

level

95%

sides

2-sided

lower limit

-0.71

upper limit

-0.13

Variability estimate

Standard error of the mean

Dispersion value

0.15

Secondary: Part 1: Change from Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52

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End point title

Part 1: Change from Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52

End point description

The timed 100MWR quantifies the time required for the participant to run or walk 100 meters (on a straight walkway) from a standing position. Data is presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 100MWR. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Subjects"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	59	57
Units: Seconds
least squares mean (standard error)	-6.57 ( 1.76 )	-3.28 ( 1.80 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

116

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1942

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

-3.29

Confidence interval

level

95%

sides

2-sided

lower limit

-8.28

upper limit

1.7

Variability estimate

Standard error of the mean

Dispersion value

2.52

Secondary: Part 1: Change from Baseline in the Timed Stair Ascend 4 Steps Test at Week 52

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End point title

Part 1: Change from Baseline in the Timed Stair Ascend 4 Steps Test at Week 52

End point description

The timed stair ascend 4 steps test quantifies the time required for the participant to ascend 4 standard steps (each step was 6 inches in height). Data presented is the change from baseline to Week 52 in the time (in seconds) to ascend 4 steps. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	62	60
Units: Seconds
least squares mean (standard error)	-0.44 ( 0.12 )	-0.08 ( 0.13 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

122

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0412

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

-0.36

Confidence interval

level

95%

sides

2-sided

lower limit

-0.71

upper limit

-0.01

Variability estimate

Standard error of the mean

Dispersion value

0.18

Secondary: Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52

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End point title

Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52

End point description

Each participant was provided with wearable devices to collect data on stride velocity. Participants wore a device on each ankle. SV95C data was derived based on stride velocity. Data is presented for change from baseline to Week 52 in SV95C. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	57	61
Units: meters (m)/second (s)
least squares mean (standard error)	0.06 ( 0.03 )	-0.03 ( 0.03 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

118

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0402

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

0.1

Confidence interval

level

95%

sides

2-sided

lower limit

upper limit

0.19

Variability estimate

Standard error of the mean

Dispersion value

0.05

Secondary: Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52

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End point title

Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52

End point description

PROMIS is a family of instruments developed and validated to assess health-related quality of life, including mobility. Mobility function scores ranged from 1 (worst mobility function) to 5 (best mobility function), where higher scores indicate a better clinical outcome. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	57	59
Units: Scores on a scale
least squares mean (standard error)	0.05 ( 0.05 )	-0.01 ( 0.05 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

116

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4272

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

0.05

Confidence interval

level

95%

sides

2-sided

lower limit

-0.08

upper limit

0.19

Variability estimate

Standard error of the mean

Dispersion value

0.07

Secondary: Part 1: Change from Baseline in PROMIS Score in Upper Extremity Function to Week 52

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End point title

Part 1: Change from Baseline in PROMIS Score in Upper Extremity Function to Week 52

End point description

PROMIS is a family of instruments developed and validated to assess health-related quality of life, including upper extremity function. Upper extremity function scores ranged from 1 (not able to do so [worst upper extremity function]) to 5 (no trouble [best upper extremity function]), where higher scores indicate a better clinical outcome. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Baseline, Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	57	58
Units: scores on a scale
least squares mean (standard error)	0.19 ( 0.07 )	0.23 ( 0.07 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

115

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7324

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

-0.04

Confidence interval

level

95%

sides

2-sided

lower limit

-0.24

upper limit

0.17

Variability estimate

Standard error of the mean

Dispersion value

0.1

Secondary: Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA

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End point title

Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA

End point description

The NSAA is a clinician-administered scale that rates performance on various functional activities. As measured by the NSAA, data was collected for the number of skills gained (the average item score was 0 at Baseline and > 0 at Part 1 Week 52) or improved (the average item score at Baseline was > 0 but less than the average item score at Part 1 Week 52). As pre-specified, data are presented for the combined number of skills gained or improved at Week 52. Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Subjects Analyzed"= number of participants evaluable for this outcome measure.

End point type

Secondary

End point timeframe

Week 52 (Part 1)

End point values	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	61
Units: number of skills
least squares mean (standard error)	4.18 ( 0.31 )	3.99 ( 0.31 )

Delandistrogene Moxeparvovec vs Placebo

Comparison groups

Delandistrogene Moxeparvovec followed by Placebo v Placebo followed by Delandistrogene Moxeparvovec

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6554

Method

Mixed model of repeated measures

Parameter type

Least squares mean change difference

Point estimate

0.19

Confidence interval

level

95%

sides

2-sided

lower limit

-0.67

upper limit

1.06

Variability estimate

Standard error of the mean

Dispersion value

0.44

Secondary: Parts 1 and 2: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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End point title

Parts 1 and 2: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

End point description

TEAEs were defined as an adverse event (AE) that emerged during treatment, having been absent pre-treatment, or worsened relative to the pretreatment state. SAEs were defined as any adverse event that resulted in death, was life threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or was an important medical event. A Summary of all SAEs and nonserious AEs ("Other"), regardless of causality and regardless of the AESI category, is located in the "Reported Adverse Events" section. Measured in the Safety Population in Part 1, which included all participants who received study treatment, with treatment group designated according to the treatment they received. Measured in the SRP-treated population in Part 2, which included all participants who received investigational study treatment (delandistrogene moxeparvovec) in the study.

End point type

Secondary

End point timeframe

Up to 104 weeks

End point values	Delandistrogene Moxeparvovec followed by Placebo	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	63	62	60
Units: participants
TEAEs	62	53	57	56
SAEs	14	5	5	8

No statistical analyses for this end point

Secondary: Parts 1 and 2: Number of Participants with Adverse Events of Special Interest (AESI)

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End point title

Parts 1 and 2: Number of Participants with Adverse Events of Special Interest (AESI)

End point description

AESIs defined as adverse events (AEs) of special interest, based on prespecified criteria and pertain to categories labeled hepatotoxicity, immune-mediated myositis, thrombotic microangiopathy (TMA), hypersensitivity, thrombocytopenia, rhabdomyolysis, and troponin elevations. Data represent number of participants who experienced an event within specified AESI category as observed by principal investigator (PI), after adjudication. Per prespecified analysis, AESI data were only collected based on specified AESI categories. Summary of all SAEs and nonserious AEs ("Other"), regardless of causality and regardless of the AESI category, is located in the "Reported Adverse Events" section. Measured in Safety Population in Part 1, which included all participants who received study treatment, with treatment group designated according to treatment received. Measured in SRP-treated population in Part 2, which included all participants who received delandistrogene moxeparvovec in the study.

End point type

Secondary

End point timeframe

Up to 104 weeks

End point values	Delandistrogene Moxeparvovec followed by Placebo	Delandistrogene Moxeparvovec followed by Placebo	Placebo followed by Delandistrogene Moxeparvovec	Placebo followed by Delandistrogene Moxeparvovec
Number of subjects analysed	63	63	62	60
Units: participants
Hepatotoxicity	12	0	0	14
Immune-mediated Myositis	0	0	0	0
TMA	0	0	0	0
Hypersensitivity	1	1	0	0
Thrombocytopenia	2	1	0	0
Rhabdomyolysis	1	0	1	0
Troponin I Elevations	2	14	1	8

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Up to approximately 104 weeks

Adverse event reporting additional description

Part 1: Safety Population, which included all participants who received study treatment, with treatment group designated according to the treatment they received. Part 2: SRP-treated population, which included all participants who received investigational study treatment (delandistrogene moxeparvovec) in the study.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

26.0

Reporting group title

Part 1: Delandistrogene Moxeparvovec

Reporting group description

Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 of Part 1.

Reporting group title

Part 2: Delandistrogene Moxeparvovec

Reporting group description

Participants who received matching placebo on Day 1 in Paty 1 received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.

Reporting group title

Part 2: Placebo

Reporting group description

Participants who received delandistrogene moxeparvovec on Day 1 of Part 1 received a single IV infusion of matching placebo on Day 1 in Part 2.

Reporting group title

Part 1: Placebo

Reporting group description

Participants received a single IV infusion of matching placebo on Day 1 of Part 1.

Serious adverse events	Part 1: Delandistrogene Moxeparvovec	Part 2: Delandistrogene Moxeparvovec	Part 2: Placebo	Part 1: Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	14 / 63 (22.22%)	8 / 60 (13.33%)	5 / 63 (7.94%)	5 / 62 (8.06%)
number of deaths (all causes)	0	0	0	0
number of deaths resulting from adverse events
Investigations
Hepatic enzyme increased
subjects affected / exposed	1 / 63 (1.59%)	1 / 60 (1.67%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Transaminases increased
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Gamma-glutamyltransferase increased
subjects affected / exposed	1 / 63 (1.59%)	1 / 60 (1.67%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Injury, poisoning and procedural complications
Craniocerebral injury
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Prescription drug used without a prescription
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Arterial injury
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Upper limb fracture
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Forearm fracture
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Cardiac disorders
Left ventricular dysfunction
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Myocarditis
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Nervous system disorders
Haemorrhage intracranial
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Headache
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	1 / 63 (1.59%)	2 / 60 (3.33%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	1 / 1	2 / 2	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Vomiting
subjects affected / exposed	2 / 63 (3.17%)	2 / 60 (3.33%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	1 / 2	2 / 2	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Abdominal pain
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Nausea
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Faecaloma
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Hepatobiliary disorders
Hepatotoxicity
subjects affected / exposed	1 / 63 (1.59%)	2 / 60 (3.33%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	4 / 4	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Liver injury
subjects affected / exposed	1 / 63 (1.59%)	1 / 60 (1.67%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 1	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	1 / 2	0 / 0	2 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Pneumonia
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Appendicitis
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
COVID-19
subjects affected / exposed	2 / 63 (3.17%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 2	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Rotavirus infection
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Toxic shock syndrome streptococcal
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Anal abscess
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Influenza
subjects affected / exposed	0 / 63 (0.00%)	0 / 60 (0.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Gastroenteritis
subjects affected / exposed	0 / 63 (0.00%)	1 / 60 (1.67%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	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 2: Delandistrogene Moxeparvovec	Part 2: Placebo	Part 1: Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	62 / 63 (98.41%)	55 / 60 (91.67%)	52 / 63 (82.54%)	57 / 62 (91.94%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
subjects affected / exposed	1 / 63 (1.59%)	3 / 60 (5.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences all number	1	3	0	1
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	19 / 63 (30.16%)	12 / 60 (20.00%)	5 / 63 (7.94%)	14 / 62 (22.58%)
occurrences all number	24	14	8	19
Fatigue
subjects affected / exposed	9 / 63 (14.29%)	10 / 60 (16.67%)	1 / 63 (1.59%)	6 / 62 (9.68%)
occurrences all number	10	10	1	8
Immune system disorders
Seasonal allergy
subjects affected / exposed	3 / 63 (4.76%)	0 / 60 (0.00%)	4 / 63 (6.35%)	2 / 62 (3.23%)
occurrences all number	3	0	4	2
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	12 / 63 (19.05%)	20 / 60 (33.33%)	12 / 63 (19.05%)	19 / 62 (30.65%)
occurrences all number	15	29	15	30
Oropharyngeal pain
subjects affected / exposed	0 / 63 (0.00%)	2 / 60 (3.33%)	2 / 63 (3.17%)	4 / 62 (6.45%)
occurrences all number	0	2	3	4
Nasal congestion
subjects affected / exposed	2 / 63 (3.17%)	6 / 60 (10.00%)	4 / 63 (6.35%)	7 / 62 (11.29%)
occurrences all number	3	8	7	11
Rhinorrhoea
subjects affected / exposed	5 / 63 (7.94%)	6 / 60 (10.00%)	7 / 63 (11.11%)	7 / 62 (11.29%)
occurrences all number	13	9	8	9
Epistaxis
subjects affected / exposed	2 / 63 (3.17%)	3 / 60 (5.00%)	1 / 63 (1.59%)	3 / 62 (4.84%)
occurrences all number	2	4	3	3
Psychiatric disorders
Aggression
subjects affected / exposed	1 / 63 (1.59%)	6 / 60 (10.00%)	2 / 63 (3.17%)	4 / 62 (6.45%)
occurrences all number	1	6	2	4
Irritability
subjects affected / exposed	9 / 63 (14.29%)	3 / 60 (5.00%)	2 / 63 (3.17%)	4 / 62 (6.45%)
occurrences all number	10	3	2	6
Attention deficit hyperactivity disorder
subjects affected / exposed	4 / 63 (6.35%)	1 / 60 (1.67%)	3 / 63 (4.76%)	1 / 62 (1.61%)
occurrences all number	4	1	3	1
Investigations
Blood creatine phosphokinase increased
subjects affected / exposed	1 / 63 (1.59%)	1 / 60 (1.67%)	1 / 63 (1.59%)	4 / 62 (6.45%)
occurrences all number	1	1	1	4
Gamma-glutamyltransferase increased
subjects affected / exposed	5 / 63 (7.94%)	12 / 60 (20.00%)	2 / 63 (3.17%)	0 / 62 (0.00%)
occurrences all number	6	17	2	0
Glutamate dehydrogenase increased
subjects affected / exposed	18 / 63 (28.57%)	12 / 60 (20.00%)	1 / 63 (1.59%)	2 / 62 (3.23%)
occurrences all number	22	13	1	2
Troponin I increased
subjects affected / exposed	2 / 63 (3.17%)	5 / 60 (8.33%)	6 / 63 (9.52%)	2 / 62 (3.23%)
occurrences all number	3	7	10	3
Blood bilirubin increased
subjects affected / exposed	0 / 63 (0.00%)	3 / 60 (5.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences all number	0	5	0	0
Injury, poisoning and procedural complications
Skin abrasion
subjects affected / exposed	1 / 63 (1.59%)	1 / 60 (1.67%)	1 / 63 (1.59%)	4 / 62 (6.45%)
occurrences all number	3	1	1	5
Fall
subjects affected / exposed	5 / 63 (7.94%)	6 / 60 (10.00%)	5 / 63 (7.94%)	7 / 62 (11.29%)
occurrences all number	14	7	10	9
Contusion
subjects affected / exposed	7 / 63 (11.11%)	5 / 60 (8.33%)	5 / 63 (7.94%)	9 / 62 (14.52%)
occurrences all number	13	5	7	17
Ligament sprain
subjects affected / exposed	3 / 63 (4.76%)	6 / 60 (10.00%)	0 / 63 (0.00%)	1 / 62 (1.61%)
occurrences all number	4	7	0	1
Joint injury
subjects affected / exposed	0 / 63 (0.00%)	3 / 60 (5.00%)	0 / 63 (0.00%)	2 / 62 (3.23%)
occurrences all number	0	3	0	2
Nervous system disorders
Headache
subjects affected / exposed	8 / 63 (12.70%)	10 / 60 (16.67%)	9 / 63 (14.29%)	8 / 62 (12.90%)
occurrences all number	12	16	17	8
Blood and lymphatic system disorders
Thrombocytopenia
subjects affected / exposed	1 / 63 (1.59%)	4 / 60 (6.67%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences all number	1	4	1	0
Gastrointestinal disorders
Nausea
subjects affected / exposed	24 / 63 (38.10%)	23 / 60 (38.33%)	5 / 63 (7.94%)	8 / 62 (12.90%)
occurrences all number	30	38	7	10
Abdominal pain upper
subjects affected / exposed	10 / 63 (15.87%)	11 / 60 (18.33%)	1 / 63 (1.59%)	9 / 62 (14.52%)
occurrences all number	14	15	1	14
Diarrhoea
subjects affected / exposed	6 / 63 (9.52%)	8 / 60 (13.33%)	0 / 63 (0.00%)	13 / 62 (20.97%)
occurrences all number	8	9	0	15
Vomiting
subjects affected / exposed	39 / 63 (61.90%)	43 / 60 (71.67%)	9 / 63 (14.29%)	11 / 62 (17.74%)
occurrences all number	93	103	11	14
Abdominal pain
subjects affected / exposed	5 / 63 (7.94%)	8 / 60 (13.33%)	0 / 63 (0.00%)	7 / 62 (11.29%)
occurrences all number	6	13	0	9
Constipation
subjects affected / exposed	5 / 63 (7.94%)	6 / 60 (10.00%)	4 / 63 (6.35%)	5 / 62 (8.06%)
occurrences all number	5	6	4	6
Skin and subcutaneous tissue disorders
Ecchymosis
subjects affected / exposed	0 / 63 (0.00%)	4 / 60 (6.67%)	1 / 63 (1.59%)	1 / 62 (1.61%)
occurrences all number	0	5	1	1
Rash
subjects affected / exposed	6 / 63 (9.52%)	8 / 60 (13.33%)	2 / 63 (3.17%)	3 / 62 (4.84%)
occurrences all number	8	9	2	5
Renal and urinary disorders
Proteinuria
subjects affected / exposed	1 / 63 (1.59%)	2 / 60 (3.33%)	4 / 63 (6.35%)	0 / 62 (0.00%)
occurrences all number	1	3	4	0
Ketonuria
subjects affected / exposed	0 / 63 (0.00%)	3 / 60 (5.00%)	0 / 63 (0.00%)	0 / 62 (0.00%)
occurrences all number	0	3	0	0
Endocrine disorders
Cushingoid
subjects affected / exposed	4 / 63 (6.35%)	3 / 60 (5.00%)	0 / 63 (0.00%)	4 / 62 (6.45%)
occurrences all number	4	3	0	4
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	4 / 63 (6.35%)	5 / 60 (8.33%)	5 / 63 (7.94%)	4 / 62 (6.45%)
occurrences all number	7	5	7	5
Arthralgia
subjects affected / exposed	7 / 63 (11.11%)	8 / 60 (13.33%)	3 / 63 (4.76%)	3 / 62 (4.84%)
occurrences all number	8	21	3	5
Pain in extremity
subjects affected / exposed	7 / 63 (11.11%)	10 / 60 (16.67%)	8 / 63 (12.70%)	12 / 62 (19.35%)
occurrences all number	15	23	8	20
Myalgia
subjects affected / exposed	4 / 63 (6.35%)	4 / 60 (6.67%)	1 / 63 (1.59%)	1 / 62 (1.61%)
occurrences all number	4	4	3	1
Muscle spasms
subjects affected / exposed	2 / 63 (3.17%)	2 / 60 (3.33%)	2 / 63 (3.17%)	4 / 62 (6.45%)
occurrences all number	2	2	5	4
Rhabdomyolysis
subjects affected / exposed	1 / 63 (1.59%)	0 / 60 (0.00%)	0 / 63 (0.00%)	4 / 62 (6.45%)
occurrences all number	1	0	0	4
Infections and infestations
Nasopharyngitis
subjects affected / exposed	9 / 63 (14.29%)	5 / 60 (8.33%)	4 / 63 (6.35%)	12 / 62 (19.35%)
occurrences all number	11	6	7	15
Ear infection
subjects affected / exposed	6 / 63 (9.52%)	4 / 60 (6.67%)	2 / 63 (3.17%)	6 / 62 (9.68%)
occurrences all number	6	6	3	7
Enterobiasis
subjects affected / exposed	5 / 63 (7.94%)	1 / 60 (1.67%)	5 / 63 (7.94%)	0 / 62 (0.00%)
occurrences all number	6	1	7	0
Viral infection
subjects affected / exposed	5 / 63 (7.94%)	2 / 60 (3.33%)	5 / 63 (7.94%)	5 / 62 (8.06%)
occurrences all number	6	2	8	5
Gastroenteritis viral
subjects affected / exposed	4 / 63 (6.35%)	1 / 60 (1.67%)	1 / 63 (1.59%)	1 / 62 (1.61%)
occurrences all number	6	1	1	1
Conjunctivitis
subjects affected / exposed	2 / 63 (3.17%)	1 / 60 (1.67%)	1 / 63 (1.59%)	4 / 62 (6.45%)
occurrences all number	2	1	1	4
COVID-19
subjects affected / exposed	15 / 63 (23.81%)	6 / 60 (10.00%)	6 / 63 (9.52%)	10 / 62 (16.13%)
occurrences all number	15	6	6	11
Influenza
subjects affected / exposed	9 / 63 (14.29%)	5 / 60 (8.33%)	2 / 63 (3.17%)	3 / 62 (4.84%)
occurrences all number	9	5	2	3
Upper respiratory tract infection
subjects affected / exposed	13 / 63 (20.63%)	13 / 60 (21.67%)	10 / 63 (15.87%)	17 / 62 (27.42%)
occurrences all number	16	30	21	27
Gastroenteritis
subjects affected / exposed	0 / 63 (0.00%)	2 / 60 (3.33%)	4 / 63 (6.35%)	2 / 62 (3.23%)
occurrences all number	0	2	5	2
Metabolism and nutrition disorders
Decreased appetite
subjects affected / exposed	20 / 63 (31.75%)	15 / 60 (25.00%)	1 / 63 (1.59%)	3 / 62 (4.84%)
occurrences all number	21	19	1	4
Vitamin D deficiency
subjects affected / exposed	0 / 63 (0.00%)	3 / 60 (5.00%)	0 / 63 (0.00%)	2 / 62 (3.23%)
occurrences all number	0	3	0	2
Dehydration
subjects affected / exposed	0 / 63 (0.00%)	3 / 60 (5.00%)	1 / 63 (1.59%)	0 / 62 (0.00%)
occurrences all number	0	3	1	0

More information

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Were there any global substantial amendments to the protocol? Yes

02 Aug 2021

Added a blinded crossover design.

30 Aug 2021

Updated/refined the exon language for inclusion criterion #2; and safety monitoring and adverse events of special interest language.

29 Aug 2022

Updated the randomization language.

28 May 2024

Added mITT as the analysis population. Updated safety information for delandistrogene moxeparvovec. Removed glutamate dehydrogenase (GLDH) from safety monitoring and reporting.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical Trial Results: A Phase 3 Multinational, Randomized, Double-Blind, Placebo Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP 9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)

Trial information

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Subject disposition

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Baseline characteristics

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End points

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Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

Secondary: Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content

Secondary: Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content

Secondary: Part 1: Change From Baseline in Time to Rise From the Floor at Week 52

Secondary: Part 1: Change From Baseline in Time to Rise From the Floor at Week 52

Secondary: Part 1: Change from Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52

Secondary: Part 1: Change from Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52

Secondary: Part 1: Change from Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52

Secondary: Part 1: Change from Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52

Secondary: Part 1: Change from Baseline in the Timed Stair Ascend 4 Steps Test at Week 52

Secondary: Part 1: Change from Baseline in the Timed Stair Ascend 4 Steps Test at Week 52

Secondary: Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52

Secondary: Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52

Secondary: Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52

Secondary: Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52

Secondary: Part 1: Change from Baseline in PROMIS Score in Upper Extremity Function to Week 52

Secondary: Part 1: Change from Baseline in PROMIS Score in Upper Extremity Function to Week 52

Secondary: Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA

Secondary: Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA

Secondary: Parts 1 and 2: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary: Parts 1 and 2: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary: Parts 1 and 2: Number of Participants with Adverse Events of Special Interest (AESI)

Secondary: Parts 1 and 2: Number of Participants with Adverse Events of Special Interest (AESI)

Adverse events

Adverse events

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Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Clinical Trial Results:
A Phase 3 Multinational, Randomized, Double-Blind, Placebo Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP 9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)