A Randomized, Double-blind, Placebo-controlled Study of D... | NCT03769116 | Trialant
NCT03769116
Sponsor
Sarepta Therapeutics, Inc.
Status
Completed
Last Update Posted
Nov 14, 2024Actual
Enrollment
41Actual
Phase
Phase 1Phase 2
Conditions
Muscular Dystrophy, Duchenne
Interventions
delandistrogene moxeparvovec
placebo
Countries
United States
Protocol Section
Identification Module
NCT ID
NCT03769116
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
SRP-9001-102
Secondary IDs
ID
Type
Description
Link
2021-000078-27
EudraCT Number
Brief Title
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
Official Title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
Acronym
Not provided
Organization
Sarepta Therapeutics, Inc.INDUSTRY
Status Module
Record Verification Date
Nov 2024
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Dec 5, 2018Actual
Primary Completion Date
Dec 8, 2020Actual
Completion Date
Aug 16, 2023Actual
First Submitted Date
Dec 6, 2018
First Submission Date that Met QC Criteria
Dec 6, 2018
First Posted Date
Dec 7, 2018Actual
Results Waived
Not provided
Results First Submitted Date
Jul 5, 2023
Results First Submitted that Met QC Criteria
Sep 13, 2023
Results First Posted Date
Sep 15, 2023Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Dec 1, 2021
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Sep 15, 2023Actual
Last Update Submitted Date
Nov 12, 2024
Last Update Posted Date
Nov 14, 2024Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Sarepta Therapeutics, Inc.INDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2.
In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).
Detailed Description
Not provided
Conditions Module
Conditions
Muscular Dystrophy, Duchenne
Keywords
Duchenne Muscular Dystrophy
Gene-Delivery
DMD
Ambulatory
Pediatric
North Star Ambulatory Assessment (NSAA)
Percent Dystrophin Positive Fibers (PDPF)
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 1Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
41Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2
Experimental
Participant will receive delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Genetic: delandistrogene moxeparvovec
Genetic: placebo
Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Experimental
Participant will receive matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Genetic: delandistrogene moxeparvovec
Genetic: placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
delandistrogene moxeparvovec
Genetic
Single IV infusion of delandistrogene moxeparvovec
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2
Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
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.
Baseline, Week 12 (Part 1)
Change From Baseline at Week 48 in NSAA Total Score
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.
Baseline, Week 48 (Part 1)
Secondary Outcomes
Measure
Description
Time Frame
Change From Baseline at Week 48 in Time to Rise From the Floor
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.
Baseline, Week 48 (Part 1)
Other Outcomes
Measure
Description
Time Frame
Baseline NSAA Total Score by Age Group
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 outcome measure presents only the baseline NSAA total score of the ITT population by the following age groups: 4-5 years old; 6-7 years old.
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
Indication of symptomatic muscular dystrophy by protocol-specified criteria.
Ability to cooperate with motor assessment testing.
Stable dose equivalent of oral corticosteroids for at least 12 weeks.
A frameshift mutation contained between exons 18 and 58 (inclusive).
Exclusion Criteria:
Impaired cardiovascular function on echocardiogram.
Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results.
Exposure to another investigational drug or exon skipping medication within 6 months of screening.
Exposure to an investigational or commercial gene therapy product.
Abnormal liver or renal function by protocol-specified criteria.
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
No participants were excluded as all randomized participants received study drug in either Part 1 or Part 2.
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.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
FG001
Placebo Switched Over to Delandistrogene Moxeparvovec
Periods
Title
Milestones
Reasons Not Completed
Part 1 - Double Blind
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot_SAP
Yes
Yes
No
Study Protocol and Statistical Analysis Plan
Apr 12, 2023
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
MeSH Terms
Intervention Browse Module
No data available
No data is available for this block.
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Parallel up to the measurement of the primary outcome at Week 48. At the beginning of Part 2, participants who were originally assigned to placebo will have the opportunity to receive delandistrogene moxeparvovec. All participants will be followed for 5 years following treatment with delandistrogene moxeparvovec.
Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2
Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2
Change From Baseline at Week 48 in Time to Ascend 4 Steps
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.
Baseline, Week 48 (Part 1)
Change From Baseline at Week 48 in Time of 10-meter Timed Test
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.
Baseline, Week 48 (Part 1)
Change From Baseline at Week 48 in Time of 100-meter Timed Test
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.
Baseline, Week 48 (Part 1)
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity
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 and reviewed by trained evaluators. An increase in IF fiber intensity (percent control) indicates increased delandistrogene moxeparvovec dystrophin expression, relative to non-dystrophic muscle (the control).
Baseline, Week 12 (Part 1)
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)
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. The number of muscle fibers expressing dystrophin was quantified by independent trained evaluators. An increase in IF PDPF indicates the number of muscle fibers with increased delandistrogene moxeparvovec dystrophin expression.
Baseline, Week 12 (Part 1)
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec
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.
Day 1 through final study visit (approximately 4.6 years)
Baseline
Change From Baseline at Week 48 in NSAA Total Score by Age Group
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.
Baseline, Week 48 (Part 1)
Columbus
Ohio
43205
United States
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.
FG00020 subjects
FG00121 subjects
Received At Least 1 Dose Of Study Drug
FG00020 subjects
FG00121 subjects
COMPLETED
FG00020 subjects
FG00121 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
Part 2 - Double Blind
Type
Comment
Milestone Data
STARTED
FG00020 subjects
FG00121 subjects
Received At Least 1 Dose Of Study Drug
FG00020 subjects
FG00121 subjects
COMPLETED
FG00020 subjects
FG00121 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
Part 3 - Open Label
Type
Comment
Milestone Data
STARTED
FG00020 subjects
FG00121 subjects
Received At Least 1 Dose Of Study Drug
FG0000 subjects
FG0010 subjects
COMPLETED
FG00017 subjects
FG00119 subjects
NOT COMPLETED
FG0003 subjects
FG0012 subjects
Type
Comment
Reasons
Study Terminated by Sponsor
FG0001 subjects
FG0012 subjects
Physician Decision
FG0001 subjects
FG001
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
BG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
BG002
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00020
BG00121
BG00241
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG0006.29± 1.19
BG0016.24± 1.13
BG0026.27± 1.15
Age, Customized
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
In utero
BG0000
BG0010
BG002
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0000
BG0010
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0001
BG0014
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG002
North Star Ambulatory Assessment (NSAA) Group
Median score = 21
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
NSAA baseline total score ≥ median score
BG0008
BG00115
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
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.
ITT Population: all randomized participants who received study treatment during Part 1.
Posted
Mean
Standard Deviation
percent normal
Baseline, Week 12 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Participants
OG00020
OG00121
Title
Denominators
Categories
Baseline
Title
Measurements
OG0004.23± 6.83(6.83 to )
OG0011.91± 1.28(1.28 to )
Change from Baseline
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Analysis conducted on changes from baseline.
Wilcoxon rank sum test
< 0.0001
Hodges-Lehmann treatment diff
6.11
2-Sided
95
2.08
12.58
Other
Primary
Change From Baseline at Week 48 in NSAA Total Score
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Least Squares Mean
Standard Error
score on a scale
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
Secondary
Change From Baseline at Week 48 in Time to Rise From the Floor
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Least Squares Mean
Standard Error
second
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Secondary
Change From Baseline at Week 48 in Time to Ascend 4 Steps
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Least Squares Mean
Standard Error
second
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Secondary
Change From Baseline at Week 48 in Time of 10-meter Timed Test
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Least Squares Mean
Standard Error
second
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Secondary
Change From Baseline at Week 48 in Time of 100-meter Timed Test
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Least Squares Mean
Standard Error
second
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Secondary
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity
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 and reviewed by trained evaluators. An increase in IF fiber intensity (percent control) indicates increased delandistrogene moxeparvovec dystrophin expression, relative to non-dystrophic muscle (the control).
ITT Population: all randomized participants who received study treatment during Part 1.
Posted
Mean
Standard Deviation
fiber intensity percent control
Baseline, Week 12 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Secondary
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)
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. The number of muscle fibers expressing dystrophin was quantified by independent trained evaluators. An increase in IF PDPF indicates the number of muscle fibers with increased delandistrogene moxeparvovec dystrophin expression.
ITT Population: all randomized participants who received study treatment during Part 1.
Posted
Mean
Standard Deviation
percent dystrophin positive fibers
Baseline, Week 12 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Other Pre-specified
Baseline NSAA Total Score by Age Group
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 outcome measure presents only the baseline NSAA total score of the ITT population by the following age groups: 4-5 years old; 6-7 years old.
ITT Population: all randomized participants who received study treatment during Part 1.
Posted
Mean
Standard Deviation
score on a scale
Baseline
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Other Pre-specified
Change From Baseline at Week 48 in NSAA Total Score by Age Group
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.
ITT Population: all randomized participants who received study treatment during Part 1 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure, and number analyzed = those who were evaluable for the specific category.
Posted
Least Squares Mean
Standard Error
score on a scale
Baseline, Week 48 (Part 1)
ID
Title
Description
OG000
Delandistrogene Moxeparvovec Switched Over to Placebo
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.
Secondary
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec
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.
Delandistrogene Moxeparvovec-treated Population: all participants who were treated with delandistrogene moxeparvovec in Part 1 or Part 2 and who were evaluable for this outcome measure. Here, overall number of participants analyzed = the number of participants with evaluable data for the outcome measure.
Posted
Count of Participants
Participants
Day 1 through final study visit (approximately 4.6 years)
ID
Title
Description
OG000
Less Than 1 Year After Delandistrogene Moxeparvovec Infusion
Arm 1: Participants who received a single infusion of delandistrogene moxeparvovec in Part 1, followed by a single infusion of placebo in Part 2, followed by an open-label extension in Part 3.
Arm 2: Participants who received a single infusion of delandistrogene moxeparvovec in Part 2, followed by an open-label extension in Part 3.
Time Frame
Day 1 through final study visit (approximately 4.6 years)
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.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Part 1: Delandistrogene Moxeparvovec
Participants received delandistrogene moxeparvovec during Part 1.
0
20
3
20
20
20
EG001
Part 1: Placebo
Participants received placebo during Part 1.
0
21
2
21
21
21
EG002
Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo
Participants who received delandistrogene moxeparvovec during Part 1 received placebo during Part 2.
0
20
2
20
20
20
EG003
Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec
Participants who received placebo during Part 1 received delandistrogene moxeparvovec during Part 2.
0
21
1
21
21
21
EG004
Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo
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.
0
20
0
20
18
20
EG005
Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec
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.
0
21
2
21
19
21
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Liver injury
Hepatobiliary disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG0030 events0 affected21 at risk
EG0040 events0 affected20 at risk
EG0050 events0 affected21 at risk
Humerus fracture
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Appendicitis
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Rhabdomyolysis
Musculoskeletal and connective tissue disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Hypertransaminasaemia
Hepatobiliary disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Femur fracture
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Ear pain
Ear and labyrinth disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0014 events3 affected21 at risk
EG0021 events1 affected20 at risk
EG0030 events0 affected21 at risk
EG0040 events0 affected20 at risk
EG0050 events0 affected21 at risk
Vomiting
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG00026 events13 affected20 at risk
EG00113 events7 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0009 events7 affected20 at risk
EG0013 events2 affected21 at risk
EG0023 events2 affected20 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0006 events5 affected20 at risk
EG0015 events2 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0006 events6 affected20 at risk
EG0014 events4 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0012 events2 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG00027 events13 affected20 at risk
EG00129 events13 affected21 at risk
EG0029 events8 affected20 at risk
EG003
Viral infection
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG00010 events8 affected20 at risk
EG00111 events9 affected21 at risk
EG0024 events3 affected20 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Sinusitis
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Otitis media
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0013 events3 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Procedural pain
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0005 events5 affected20 at risk
EG0017 events7 affected21 at risk
EG0026 events6 affected20 at risk
EG003
Skin abrasion
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0004 events4 affected20 at risk
EG0013 events2 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Limb injury
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0011 events1 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Arthropod bite
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0013 events3 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Post procedural contusion
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0012 events2 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA 24.1
Systematic Assessment
EG0005 events5 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Weight increased
Investigations
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA 24.1
Systematic Assessment
EG00010 events8 affected20 at risk
EG0010 events0 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Increased appetite
Metabolism and nutrition disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0012 events2 affected21 at risk
EG0023 events3 affected20 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 24.1
Systematic Assessment
EG0006 events5 affected20 at risk
EG0011 events1 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 24.1
Systematic Assessment
EG0006 events5 affected20 at risk
EG0017 events5 affected21 at risk
EG0028 events7 affected20 at risk
EG003
Headache
Nervous system disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG00113 events6 affected21 at risk
EG0027 events3 affected20 at risk
EG003
Irritability
Psychiatric disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0011 events1 affected21 at risk
EG0025 events5 affected20 at risk
EG003
Sleep disorder
Psychiatric disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0011 events1 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 24.1
Systematic Assessment
EG00010 events9 affected20 at risk
EG0018 events6 affected21 at risk
EG0023 events3 affected20 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 24.1
Systematic Assessment
EG0005 events4 affected20 at risk
EG0013 events3 affected21 at risk
EG0024 events4 affected20 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 24.1
Systematic Assessment
EG0003 events3 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0016 events3 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA 24.1
Systematic Assessment
EG00010 events7 affected20 at risk
EG0014 events4 affected21 at risk
EG0024 events4 affected20 at risk
EG003
Dermatitis
Skin and subcutaneous tissue disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Dermatitis contact
Skin and subcutaneous tissue disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0013 events3 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0013 events3 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0013 events3 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Rhinovirus infection
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Craniocerebral injury
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Lip injury
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Skin papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0010 events0 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Cardiomyopathy
Cardiac disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Cushingoid
Endocrine disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Cataract
Eye disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Gastrooesophageal reflux disease
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
COVID-19
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0003 events1 affected20 at risk
EG0011 events1 affected21 at risk
EG0023 events2 affected20 at risk
EG003
Influenza
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Pharyngitis streptococcal
Infections and infestations
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Sunburn
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Incision site haemorrhage
Injury, poisoning and procedural complications
MedDRA 24.1
Systematic Assessment
EG0006 events6 affected20 at risk
EG0015 events4 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Glutamate dehydrogenase increased
Investigations
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
White blood cell count decreased
Investigations
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Vitamin D deficiency
Metabolism and nutrition disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0011 events1 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0011 events1 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Lethargy
Nervous system disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Affect lability
Psychiatric disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0011 events1 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Ketonuria
Renal and urinary disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0010 events0 affected21 at risk
EG0021 events1 affected20 at risk
EG003
Proteinuria
Renal and urinary disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Pyrexia
General disorders
MedDRA 24.1
Systematic Assessment
EG0005 events4 affected20 at risk
EG0011 events1 affected21 at risk
EG0022 events2 affected20 at risk
EG003
Vessel puncture site haemorrhage
General disorders
MedDRA 24.1
Systematic Assessment
EG0002 events2 affected20 at risk
EG0011 events1 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Fatigue
General disorders
MedDRA 24.1
Systematic Assessment
EG0001 events1 affected20 at risk
EG0010 events0 affected21 at risk
EG0023 events3 affected20 at risk
EG003
Infusion site extravasation
General disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0012 events2 affected21 at risk
EG0020 events0 affected20 at risk
EG003
Catheter site haemorrhage
General disorders
MedDRA 24.1
Systematic Assessment
EG0000 events0 affected20 at risk
EG0011 events1 affected21 at risk
EG0021 events1 affected20 at risk
EG003
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.
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Browse Leaves
Not provided
Browse Branches
Not provided
0 subjects
Withdrawal by Subject
FG0001 subjects
FG0010 subjects
0
Preterm newborn infants (gestational age < 37 wks)
BG0000
BG0010
BG0020
Newborns (0-27 days)
BG0000
BG0010
BG0020
Infants and toddlers (28 days-23 months)
BG0000
BG0010
BG0020
Children (2-11 years)
BG00020
BG00121
BG00241
Adolescents (12-17 years)
BG0000
BG0010
BG0020
Adults (18-64 years)
BG0000
BG0010
BG0020
From 65-84 years
BG0000
BG0010
BG0020
85 years and over
BG0000
BG0010
BG0020
0
Male
BG00020
BG00121
BG00241
5
Not Hispanic or Latino
BG00019
BG00116
BG00235
Unknown or Not Reported
BG0000
BG0011
BG0021
0
Asian
BG0004
BG0011
BG0025
Native Hawaiian or Other Pacific Islander
BG0000
BG0010
BG0020
Black or African American
BG0000
BG0010
BG0020
White
BG00013
BG00117
BG00230
More than one race
BG0000
BG0010
BG0020
Unknown or Not Reported
BG0003
BG0013
BG0026
23
NSAA baseline total score < median score
BG00012
BG0016
BG00218
23.82
± 39.76
(39.76 to )
OG0010.14± 1.24(1.24 to )
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Participants
OG00019
OG00121
Title
Denominators
Categories
Title
Measurements
OG0001.7± 0.6(0.6 to )
OG0010.9± 0.6(0.6 to )
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mixed-model for Repeated Measures
= 0.3730
LSM Change Difference
0.8
Standard Error of the Mean
0.9
2-Sided
95
-1.0
2.7
Other
Participants
OG00019
OG00121
Title
Denominators
Categories
Title
Measurements
OG000-0.15± 0.25(0.25 to )
OG0010.35± 0.23(0.23 to )
Participants
OG00019
OG00121
Title
Denominators
Categories
Title
Measurements
OG0000.17± 0.26(0.26 to )
OG0010.03± 0.26(0.26 to )
Participants
OG00019
OG00121
Title
Denominators
Categories
Title
Measurements
OG0000.59± 0.20(0.20 to )
OG0010.10± 0.19(0.19 to )
Participants
OG00019
OG00121
Title
Denominators
Categories
Title
Measurements
OG0004.29± 3.92(3.92 to )
OG0016.28± 3.83(3.83 to )
Units
Counts
Participants
OG00020
OG00121
Title
Denominators
Categories
Title
Measurements
OG0000.06± 0.11(0.11 to )
OG0010.00± 0.02(0.02 to )
Units
Counts
Participants
OG00020
OG00121
Title
Denominators
Categories
Title
Measurements
OG00023.88± 25.58(25.58 to )
OG0015.09± 12.96(12.96 to )
Units
Counts
Participants
OG00020
OG00121
Title
Denominators
Categories
Age Group: 4-5 years old
ParticipantsOG0008
ParticipantsOG0018
Title
Measurements
OG00020.1± 1.9
OG00120.4± 2.7
Age Group: 6-7 years old
ParticipantsOG00012
ParticipantsOG00113
Title
Measurements
OG00019.6± 4.1
OG001
OG001
Placebo Switched Over to Delandistrogene Moxeparvovec
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.
Units
Counts
Participants
OG00019
OG00121
Title
Denominators
Categories
Age Group: 4-5 years old
ParticipantsOG0008
ParticipantsOG0018
Title
Measurements
OG0004.3± 0.6
OG0011.9± 0.6
Age Group: 6-7 years old
ParticipantsOG00011
ParticipantsOG00113
Title
Measurements
OG000-0.2± 0.7
OG001
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Change from baseline - Age Group: 4-5 years old
Mixed-model for Repeated Measures
= 0.0172
LSM Change Difference
2.5
Standard Error of the Mean
0.9
2-Sided
95
0.5
4.4
Other
OG000
OG001
Change from baseline - Age Group: 6-7 years old
Mixed-model for Repeated Measures
= 0.5384
LSM Change Difference
-0.7
Standard Error of the Mean
1.1
2-Sided
95
-3.0
1.6
Other
OG001
1 to 2 Years After Delandistrogene Moxeparvovec Infusion
Arm 1: Participants who received a single infusion of delandistrogene moxeparvovec in Part 1, followed by a single infusion of placebo in Part 2, followed by an open-label extension in Part 3.
Arm 2: Participants who received a single infusion of delandistrogene moxeparvovec in Part 2, followed by an open-label extension in Part 3.
OG002
2 to 3 Years After Delandistrogene Moxeparvovec Infusion
Arm 1: Participants who received a single infusion of delandistrogene moxeparvovec in Part 1, followed by a single infusion of placebo in Part 2, followed by an open-label extension in Part 3.
Arm 2: Participants who received a single infusion of delandistrogene moxeparvovec in Part 2, followed by an open-label extension in Part 3.
OG003
3 to 4 Years After Delandistrogene Moxeparvovec Infusion
Arm 1: Participants who received a single infusion of delandistrogene moxeparvovec in Part 1, followed by a single infusion of placebo in Part 2, followed by an open-label extension in Part 3.
Arm 2: Participants who received a single infusion of delandistrogene moxeparvovec in Part 2, followed by an open-label extension in Part 3.
OG004
More Than 4 Years After Delandistrogene Moxeparvovec Infusion
Arm 1: Participants who received a single infusion of delandistrogene moxeparvovec in Part 1, followed by a single infusion of placebo in Part 2, followed by an open-label extension in Part 3.
Arm 2: Participants who received a single infusion of delandistrogene moxeparvovec in Part 2, followed by an open-label extension in Part 3.