Trial Outcomes & Findings for A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) (NCT NCT03769116)

Last Updated: 2024-11-14

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

41 participants

Primary outcome timeframe

Baseline, Week 12 (Part 1)

Results posted on

2024-11-14

Participant Flow

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.

No participants were excluded as all randomized participants received study drug in either Part 1 or Part 2.

Participant milestones

Participant milestones
Measure	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.	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.
Part 1 - Double Blind STARTED	20	21
Part 1 - Double Blind Received At Least 1 Dose Of Study Drug	20	21
Part 1 - Double Blind COMPLETED	20	21
Part 1 - Double Blind NOT COMPLETED	0	0
Part 2 - Double Blind STARTED	20	21
Part 2 - Double Blind Received At Least 1 Dose Of Study Drug	20	21
Part 2 - Double Blind COMPLETED	20	21
Part 2 - Double Blind NOT COMPLETED	0	0
Part 3 - Open Label STARTED	20	21
Part 3 - Open Label Received At Least 1 Dose Of Study Drug	0	0
Part 3 - Open Label COMPLETED	17	19
Part 3 - Open Label NOT COMPLETED	3	2

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	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.
Part 3 - Open Label Study Terminated by Sponsor	1	2
Part 3 - Open Label Physician Decision	1	0
Part 3 - Open Label Withdrawal by Subject	1	0

Baseline Characteristics

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=20 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	Total n=41 Participants Total of all reporting groups
Age, Continuous	6.29 years STANDARD_DEVIATION 1.19 • n=99 Participants	6.24 years STANDARD_DEVIATION 1.13 • n=107 Participants	6.27 years STANDARD_DEVIATION 1.15 • n=206 Participants
Age, Customized In utero	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized Preterm newborn infants (gestational age < 37 wks)	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized Newborns (0-27 days)	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized Infants and toddlers (28 days-23 months)	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized Children (2-11 years)	20 Participants n=99 Participants	21 Participants n=107 Participants	41 Participants n=206 Participants
Age, Customized Adolescents (12-17 years)	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized Adults (18-64 years)	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized From 65-84 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Customized 85 years and over	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Sex: Female, Male Female	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Sex: Female, Male Male	20 Participants n=99 Participants	21 Participants n=107 Participants	41 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=99 Participants	4 Participants n=107 Participants	5 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=99 Participants	16 Participants n=107 Participants	35 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	4 Participants n=99 Participants	1 Participants n=107 Participants	5 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	13 Participants n=99 Participants	17 Participants n=107 Participants	30 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=99 Participants	3 Participants n=107 Participants	6 Participants n=206 Participants
North Star Ambulatory Assessment (NSAA) Group NSAA baseline total score ≥ median score	8 Participants n=99 Participants	15 Participants n=107 Participants	23 Participants n=206 Participants
North Star Ambulatory Assessment (NSAA) Group NSAA baseline total score < median score	12 Participants n=99 Participants	6 Participants n=107 Participants	18 Participants n=206 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12 (Part 1)

Population: ITT Population: all randomized participants who received study treatment during Part 1.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=20 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content Change from Baseline	23.82 percent normal Standard Deviation 39.76 • Interval 39.76 to	0.14 percent normal Standard Deviation 1.24 • Interval 1.24 to	—	—	—
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content Baseline	4.23 percent normal Standard Deviation 6.83 • Interval 6.83 to	1.91 percent normal Standard Deviation 1.28 • Interval 1.28 to	—	—	—

PRIMARY outcome

Timeframe: Baseline, Week 48 (Part 1)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in NSAA Total Score	1.7 score on a scale Standard Error 0.6 • Interval 0.6 to	0.9 score on a scale Standard Error 0.6 • Interval 0.6 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 48 (Part 1)

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in Time to Rise From the Floor	-0.15 second Standard Error 0.25 • Interval 0.25 to	0.35 second Standard Error 0.23 • Interval 0.23 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 48 (Part 1)

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in Time to Ascend 4 Steps	0.17 second Standard Error 0.26 • Interval 0.26 to	0.03 second Standard Error 0.26 • Interval 0.26 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 48 (Part 1)

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in Time of 10-meter Timed Test	0.59 second Standard Error 0.20 • Interval 0.2 to	0.10 second Standard Error 0.19 • Interval 0.19 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 48 (Part 1)

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in Time of 100-meter Timed Test	4.29 second Standard Error 3.92 • Interval 3.92 to	6.28 second Standard Error 3.83 • Interval 3.83 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 12 (Part 1)

Population: ITT Population: all randomized participants who received study treatment during Part 1.

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).

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=20 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity	0.06 fiber intensity percent control Standard Deviation 0.11 • Interval 0.11 to	0.00 fiber intensity percent control Standard Deviation 0.02 • Interval 0.02 to	—	—	—

SECONDARY outcome

Timeframe: Baseline, Week 12 (Part 1)

Population: ITT Population: all randomized participants who received study treatment during Part 1.

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=20 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)	23.88 percent dystrophin positive fibers Standard Deviation 25.58 • Interval 25.58 to	5.09 percent dystrophin positive fibers Standard Deviation 12.96 • Interval 12.96 to	—	—	—

SECONDARY outcome

Timeframe: Day 1 through final study visit (approximately 4.6 years)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=41 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=41 Participants 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.	2 to 3 Years After Delandistrogene Moxeparvovec Infusion n=41 Participants 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.	3 to 4 Years After Delandistrogene Moxeparvovec Infusion n=30 Participants 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.	More Than 4 Years After Delandistrogene Moxeparvovec Infusion n=12 Participants 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.
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Treatment-emergent AE (TEAE)	41 Participants	37 Participants	33 Participants	17 Participants	3 Participants
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Serious AE (SAE)	5 Participants	1 Participants	2 Participants	0 Participants	0 Participants
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Treatment-related TEAE	37 Participants	2 Participants	1 Participants	0 Participants	0 Participants
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Treatment-related SAE	3 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Any AE leading to study discontinuation	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec Death	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline

Population: ITT Population: all randomized participants who received study treatment during Part 1.

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.

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=20 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Baseline NSAA Total Score by Age Group Age Group: 4-5 years old	20.1 score on a scale Standard Deviation 1.9	20.4 score on a scale Standard Deviation 2.7	—	—	—
Baseline NSAA Total Score by Age Group Age Group: 6-7 years old	19.6 score on a scale Standard Deviation 4.1	24.0 score on a scale Standard Deviation 2.9	—	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 48 (Part 1)

Outcome measures

Outcome measures
Measure	Delandistrogene Moxeparvovec Switched Over to Placebo n=19 Participants 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.	Placebo Switched Over to Delandistrogene Moxeparvovec n=21 Participants 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.	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.	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.	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.
Change From Baseline at Week 48 in NSAA Total Score by Age Group Age Group: 4-5 years old	4.3 score on a scale Standard Error 0.6	1.9 score on a scale Standard Error 0.6	—	—	—
Change From Baseline at Week 48 in NSAA Total Score by Age Group Age Group: 6-7 years old	-0.2 score on a scale Standard Error 0.7	0.5 score on a scale Standard Error 0.7	—	—	—

Adverse Events

Part 1: Delandistrogene Moxeparvovec

Serious events: 3 serious events

Other events: 20 other events

Deaths: 0 deaths

Part 1: Placebo

Serious events: 2 serious events

Other events: 21 other events

Deaths: 0 deaths

Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo

Serious events: 2 serious events

Other events: 20 other events

Deaths: 0 deaths

Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec

Serious events: 1 serious events

Other events: 21 other events

Deaths: 0 deaths

Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo

Serious events: 0 serious events

Other events: 18 other events

Deaths: 0 deaths

Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec

Serious events: 2 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part 1: Delandistrogene Moxeparvovec n=20 participants at risk Participants received delandistrogene moxeparvovec during Part 1.	Part 1: Placebo n=21 participants at risk Participants received placebo during Part 1.	Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo n=20 participants at risk Participants who received delandistrogene moxeparvovec during Part 1 received placebo during Part 2.	Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec n=21 participants at risk Participants who received placebo during Part 1 received delandistrogene moxeparvovec during Part 2.	Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo n=20 participants at risk 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.	Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec n=21 participants at risk 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.
Hepatobiliary disorders Hypertransaminasaemia	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Hepatobiliary disorders Liver injury	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Appendicitis	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Musculoskeletal and connective tissue disorders Rhabdomyolysis	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Femur fracture	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.

Other adverse events

Other adverse events
Measure	Part 1: Delandistrogene Moxeparvovec n=20 participants at risk Participants received delandistrogene moxeparvovec during Part 1.	Part 1: Placebo n=21 participants at risk Participants received placebo during Part 1.	Part 2: Delandistrogene Moxeparvovec Switched Over to Placebo n=20 participants at risk Participants who received delandistrogene moxeparvovec during Part 1 received placebo during Part 2.	Part 2: Placebo Switched Over to Delandistrogene Moxeparvovec n=21 participants at risk Participants who received placebo during Part 1 received delandistrogene moxeparvovec during Part 2.	Part 3: Delandistrogene Moxeparvovec Switched Over to Placebo n=20 participants at risk 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.	Part 3: Placebo Switched Over to Delandistrogene Moxeparvovec n=21 participants at risk 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.
Ear and labyrinth disorders Ear pain	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Vomiting	65.0% 13/20 • Number of events 26 • Day 1 through final study visit (approximately 4.6 years) 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.	33.3% 7/21 • Number of events 13 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	76.2% 16/21 • Number of events 33 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Nausea	35.0% 7/20 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	52.4% 11/21 • Number of events 14 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Abdominal pain	25.0% 5/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Abdominal pain upper	30.0% 6/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	38.1% 8/21 • Number of events 10 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Constipation	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Diarrhoea	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Upper respiratory tract infection	65.0% 13/20 • Number of events 27 • Day 1 through final study visit (approximately 4.6 years) 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.	61.9% 13/21 • Number of events 29 • Day 1 through final study visit (approximately 4.6 years) 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.	40.0% 8/20 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	33.3% 7/21 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	45.0% 9/20 • Number of events 18 • Day 1 through final study visit (approximately 4.6 years) 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.	42.9% 9/21 • Number of events 18 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Viral infection	40.0% 8/20 • Number of events 10 • Day 1 through final study visit (approximately 4.6 years) 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.	42.9% 9/21 • Number of events 11 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Gastroenteritis	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Sinusitis	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Otitis media	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Nasopharyngitis	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Procedural pain	25.0% 5/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	33.3% 7/21 • Number of events 7 • Day 1 through final study visit (approximately 4.6 years) 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.	30.0% 6/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	42.9% 9/21 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Skin abrasion	20.0% 4/20 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Limb injury	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Arthropod bite	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Post procedural contusion	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Investigations Gamma-glutamyltransferase increased	25.0% 5/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	28.6% 6/21 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Investigations Blood bilirubin increased	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Investigations Weight increased	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Metabolism and nutrition disorders Decreased appetite	40.0% 8/20 • Number of events 10 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	71.4% 15/21 • Number of events 18 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Metabolism and nutrition disorders Increased appetite	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Musculoskeletal and connective tissue disorders Arthralgia	25.0% 5/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 5/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	23.8% 5/21 • Number of events 7 • Day 1 through final study visit (approximately 4.6 years) 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.	35.0% 7/20 • Number of events 8 • Day 1 through final study visit (approximately 4.6 years) 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.	28.6% 6/21 • Number of events 7 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.
Nervous system disorders Headache	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	28.6% 6/21 • Number of events 13 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 7 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 7 • Day 1 through final study visit (approximately 4.6 years) 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.
Psychiatric disorders Irritability	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	25.0% 5/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	42.9% 9/21 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Psychiatric disorders Sleep disorder	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Renal and urinary disorders Dysuria	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Renal and urinary disorders Pollakiuria	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Respiratory, thoracic and mediastinal disorders Cough	45.0% 9/20 • Number of events 10 • Day 1 through final study visit (approximately 4.6 years) 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.	28.6% 6/21 • Number of events 8 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	20.0% 4/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	20.0% 4/20 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Respiratory, thoracic and mediastinal disorders Nasal congestion	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Respiratory, thoracic and mediastinal disorders Epistaxis	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Skin and subcutaneous tissue disorders Ecchymosis	35.0% 7/20 • Number of events 10 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	20.0% 4/20 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Skin and subcutaneous tissue disorders Dermatitis	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Skin and subcutaneous tissue disorders Dermatitis contact	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Skin and subcutaneous tissue disorders Rash	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Rhinovirus infection	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Craniocerebral injury	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Lip injury	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Blood and lymphatic system disorders Anaemia	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	23.8% 5/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Cardiac disorders Cardiomyopathy	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Endocrine disorders Cushingoid	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Eye disorders Cataract	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Gastrooesophageal reflux disease	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Gastrointestinal disorders Toothache	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations COVID-19	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	45.0% 9/20 • Number of events 9 • Day 1 through final study visit (approximately 4.6 years) 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.	42.9% 9/21 • Number of events 11 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Gastroenteritis viral	5.0% 1/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Influenza	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.
Infections and infestations Pharyngitis streptococcal	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Sunburn	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Injury, poisoning and procedural complications Incision site haemorrhage	30.0% 6/20 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	19.0% 4/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	23.8% 5/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Investigations Glutamate dehydrogenase increased	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Investigations White blood cell count decreased	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Metabolism and nutrition disorders Dehydration	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Metabolism and nutrition disorders Vitamin D deficiency	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
Musculoskeletal and connective tissue disorders Back pain	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 4 • Day 1 through final study visit (approximately 4.6 years) 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.
Musculoskeletal and connective tissue disorders Myalgia	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Nervous system disorders Lethargy	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Psychiatric disorders Affect lability	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Psychiatric disorders Anxiety	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.
Renal and urinary disorders Ketonuria	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
Renal and urinary disorders Proteinuria	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
General disorders Pyrexia	20.0% 4/20 • Number of events 5 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	28.6% 6/21 • Number of events 6 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.
General disorders Vessel puncture site haemorrhage	10.0% 2/20 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
General disorders Fatigue	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	15.0% 3/20 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	14.3% 3/21 • Number of events 3 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.
General disorders Infusion site extravasation	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.
General disorders Catheter site haemorrhage	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	4.8% 1/21 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	5.0% 1/20 • Number of events 1 • Day 1 through final study visit (approximately 4.6 years) 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.	9.5% 2/21 • Number of events 2 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/20 • Day 1 through final study visit (approximately 4.6 years) 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.	0.00% 0/21 • Day 1 through final study visit (approximately 4.6 years) 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.

Additional Information

Medical Director

Sarepta Therapeutics, Inc.

Phone: 888-727-3782

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER