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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-003374-91 | EudraCT Number |
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| Name | Class |
|---|---|
| Hoffmann-La Roche | INDUSTRY |
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The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec followed by Placebo | Experimental | Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2. |
|
| Placebo followed by Delandistrogene Moxeparvovec | Placebo Comparator | Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | Single IV infusion of delandistrogene moxeparvovec. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function. | Baseline, Week 52 (Part 1) |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content | Quantity of delandistrogene moxeparvovec dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Part 1, Week 12. For this endpoint, 2 blocks of tissues were analyzed by Western blot, each with 2 technical replicates to determine the delandistrogene moxeparvovec dystrophin protein level (percent control). The block average value from 2 technical replicates was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for the analysis. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein. |
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Inclusion Criteria:
Exclusion Criteria:
Other inclusion or exclusion criteria could apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's | Little Rock | Arkansas | 72202 | United States | ||
| UC San Diego Altman Clinical and Translational Research Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39385046 | Result | Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9. | |
| 41518520 |
| Label | URL |
|---|---|
| SRP-9001-301 Plain Language Study Summary | View source |
Not provided
126 participants were enrolled in the study. 1 of the 126 participants was enrolled via a country-specific protocol addendum. Data are presented below for the 125 participants enrolled via the global protocol.
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| ID | Title | Description |
|---|---|---|
| FG000 | Delandistrogene Moxeparvovec Followed by Placebo | Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2. |
| FG001 | Placebo Followed by Delandistrogene Moxeparvovec |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1 |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | May 28, 2024 |
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|
| placebo | Genetic | Single IV infusion of matching placebo. |
|
| Week 12 |
| Part 1: Change From Baseline in Time to Rise From the Floor at Week 52 | The time to rise from the floor test quantifies the time required for the participant to stand in an upright position with arms by sides, starting from the supine position with arms by sides. Data are presented for the change from baseline to Week 52 in the time taken (in seconds) to rise from the floor. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52 | The timed 10MWR quantifies the time required for the participant to run or walk 10 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 10MWR. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52 | The timed 100MWR quantifies the time required for the participant to run or walk 100 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 100MWR. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52 | The timed stair ascend 4 steps test quantifies the time required for the participant to ascend 4 standard steps (each step was 6 inches in height). Data presented is the change from baseline to Week 52 in the time (in seconds) to ascend 4 steps. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52 | Each participant was provided with wearable devices to collect data on stride velocity. Participants wore a device on each ankle. SV95C data was derived based on stride velocity. Data are presented for change from baseline to Week 52 in SV95C. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52 | PROMIS is a family of instruments developed and validated to assess health-related quality of life, including mobility. Mobility function scores ranged from 1 (worst mobility function) to 5 (best mobility function), where higher scores indicate a better clinical outcome. | Baseline, Week 52 (Part 1) |
| Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52 | PROMIS is a family of instruments developed and validated to assess health-related quality of life, including upper extremity function. Upper extremity function scores ranged from 1 (not able to do so [worst upper extremity function]) to 5 (no trouble [best upper extremity function]), where higher scores indicate a better clinical outcome. | Baseline, Week 52 (Part 1) |
| Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA | The NSAA is a clinician-administered scale that rates performance on various functional activities. As measured by the NSAA, data was collected for the number of skills gained (the average item score was 0 at Baseline and > 0 at Part 1 Week 52) or improved (the average item score at Baseline was > 0 but less than the average item score at Part 1 Week 52). As pre-specified, data are presented for the combined number of skills gained or improved at Week 52. | Week 52 (Part 1) |
| Parts 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | TEAEs were defined as an adverse event (AE) that emerged during treatment, having been absent pre-treatment, or worsened relative to the pretreatment state. SAEs were defined as any adverse event that resulted in death, was life threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or was an important medical event. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Up to 104 weeks |
| Parts 1 and 2: Number of Participants With Adverse Events of Special Interest (AESI) | AESIs were defined as adverse events (AEs) of special interest, based on pre-specified criteria and pertain to categories labeled hepatotoxicity, immune-mediated myositis, thrombotic microangiopathy (TMA), hypersensitivity, thrombocytopenia, rhabdomyolysis, and troponin I elevations. The data represent the number of participants who experienced an event within the specified AESI category as observed by the principal investigator (PI), after adjudication. Per prespecified analysis, AESI data were only collected based on the specified AESI categories. A Summary of all SAEs and nonserious AEs ("Other"), regardless of causality and regardless of the AESI category, is located in the "Reported Adverse Events" section. | Up to 104 weeks |
| La Jolla |
| California |
| 92037 |
| United States |
| UCLA Medical Center | Los Angeles | California | 90095 | United States |
| Lucile Packard Children's Hospital at Stanford | Palo Alto | California | 94304 | United States |
| University of California, Davis | Sacramento | California | 95817 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| University of Florida | Gainesville | Florida | 32608 | United States |
| Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | 52242 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University of St. Louis | St Louis | Missouri | 63110 | United States |
| Columbia University/NYPH | New York | New York | 10032 | United States |
| University of Rochester | Rochester | New York | 14642 | United States |
| Duke University Medical Center, Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43210 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| University of Texas Southwestern | Dallas | Texas | 75390 | United States |
| University of Utah Hospital | Salt Lake City | Utah | 84132 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23510 | United States |
| Children's Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| University Hospital Ghent | Ghent | 9000 | Belgium |
| LMU - Klinikum der Universitaet Muenchen - Kinderklinik und | Bayern | 80337 | Germany |
| Universitätsklinikum Essen - Klinik für Kinderheilkunde I | Essen | 45147 | Germany |
| University Hospital Hamburg- Eppendorf | Hamburg | 20251 | Germany |
| Hong Kong Children's Hospital | Kowloon | Hong Kong |
| IRCCS Istituto G.Gaslini, U.O. | Genoa | 16147 | Italy |
| UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS | Roma | 00168 | Italy |
| Kobe University Hospital | Kobe | 650-0017 | Japan |
| National Center for Child Health and Development | Tokyo | 157-8535 | Japan |
| Tokyo Women's Medical University Hospital - Pediatrics | Tokyo | 162-8666 | Japan |
| National Center of Neurology and Psychiatry | Tokyo | 187-8551 | Japan |
| Hospital Universitari i Politécnico La Fe | Valencia | Comunidad Valencia | Spain |
| Hospital Sant Joan de Déu | Barcelona | 08950 | Spain |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | 807 | Taiwan |
| National Taiwan University Hospital | Taipei | 100225 | Taiwan |
| Oxford University Hospitals NHS Foundation Trust | Headington | Oxford | OX3 9DU | United Kingdom |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | WC1N3JK | United Kingdom |
| The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Derived |
| Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Furgerson M, Ding K, Singh P, Potter R, Asher DR, Murphy AP, Reid C, Hooper G, Torre CO, Manfrini M, Rodino-Klapac LR. Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. Neurol Ther. 2026 Apr;15(2):545-559. doi: 10.1007/s40120-025-00879-8. Epub 2026 Jan 10. |
| 41182527 | Derived | Audhya I, Nacson AB, Gooch K, Basnyat B, Slota C, Martin S, Murphy A, Lansdall CJ, Ciobanu T, Nascimento A, Veerapandiyan A. Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial. Neurol Ther. 2026 Feb;15(1):41-60. doi: 10.1007/s40120-025-00842-7. Epub 2025 Nov 3. |
| 40354061 | Derived | Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR. Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial. JAMA Neurol. 2025 Jul 1;82(7):734-744. doi: 10.1001/jamaneurol.2025.0992. |
| 39589719 | Derived | McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I; EMBARK Study Group. Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. Neurol Ther. 2025 Feb;14(1):211-225. doi: 10.1007/s40120-024-00685-8. Epub 2024 Nov 26. |
Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
| Received at Least 1 Dose of Study Drug in Part 1 |
|
| COMPLETED |
|
| NOT COMPLETED |
|
| Part 2 |
|
|
Measured in the Modified Intent-to-Treat (mITT) Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Delandistrogene Moxeparvovec Followed by Placebo | Participants received a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2. |
| BG001 | Placebo Followed by Delandistrogene Moxeparvovec | Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | 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. | Measured in the Modified Intent-to-Treat (mITT) Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | scores on a scale | Baseline, Week 52 (Part 1) |
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content | Quantity of delandistrogene moxeparvovec dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Part 1, Week 12. For this endpoint, 2 blocks of tissues were analyzed by Western blot, each with 2 technical replicates to determine the delandistrogene moxeparvovec dystrophin protein level (percent control). The block average value from 2 technical replicates was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for the analysis. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Percent control | Week 12 |
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| Secondary | Part 1: Change From Baseline in Time to Rise From the Floor at Week 52 | The time to rise from the floor test quantifies the time required for the participant to stand in an upright position with arms by sides, starting from the supine position with arms by sides. Data are presented for the change from baseline to Week 52 in the time taken (in seconds) to rise from the floor. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Seconds | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52 | The timed 10MWR quantifies the time required for the participant to run or walk 10 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 10MWR. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Seconds | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52 | The timed 100MWR quantifies the time required for the participant to run or walk 100 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 100MWR. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Seconds | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52 | The timed stair ascend 4 steps test quantifies the time required for the participant to ascend 4 standard steps (each step was 6 inches in height). Data presented is the change from baseline to Week 52 in the time (in seconds) to ascend 4 steps. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Seconds | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52 | Each participant was provided with wearable devices to collect data on stride velocity. Participants wore a device on each ankle. SV95C data was derived based on stride velocity. Data are presented for change from baseline to Week 52 in SV95C. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | meters (m)/second (s) | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52 | PROMIS is a family of instruments developed and validated to assess health-related quality of life, including mobility. Mobility function scores ranged from 1 (worst mobility function) to 5 (best mobility function), where higher scores indicate a better clinical outcome. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline, Week 52 (Part 1) |
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| Secondary | Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52 | PROMIS is a family of instruments developed and validated to assess health-related quality of life, including upper extremity function. Upper extremity function scores ranged from 1 (not able to do so [worst upper extremity function]) to 5 (no trouble [best upper extremity function]), where higher scores indicate a better clinical outcome. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | scores on a scale | Baseline, Week 52 (Part 1) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA | The NSAA is a clinician-administered scale that rates performance on various functional activities. As measured by the NSAA, data was collected for the number of skills gained (the average item score was 0 at Baseline and > 0 at Part 1 Week 52) or improved (the average item score at Baseline was > 0 but less than the average item score at Part 1 Week 52). As pre-specified, data are presented for the combined number of skills gained or improved at Week 52. | Measured in the mITT Population, which included all randomized participants who received study treatment, with treatment group designated according to randomization. Here, "Number of Participants Analyzed"= number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | number of skills | Week 52 (Part 1) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Parts 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | TEAEs were defined as an adverse event (AE) that emerged during treatment, having been absent pre-treatment, or worsened relative to the pretreatment state. SAEs were defined as any adverse event that resulted in death, was life threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or was an important medical event. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Measured in the Safety Population in Part 1, which included all participants who received study treatment, with treatment group designated according to the treatment they received. Measured in the SRP-treated population in Part 2, which included all participants who received investigational study treatment (delandistrogene moxeparvovec) in the study. | Posted | Count of Participants | Participants | Up to 104 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Parts 1 and 2: Number of Participants With Adverse Events of Special Interest (AESI) | AESIs were defined as adverse events (AEs) of special interest, based on pre-specified criteria and pertain to categories labeled hepatotoxicity, immune-mediated myositis, thrombotic microangiopathy (TMA), hypersensitivity, thrombocytopenia, rhabdomyolysis, and troponin I elevations. The data represent the number of participants who experienced an event within the specified AESI category as observed by the principal investigator (PI), after adjudication. Per prespecified analysis, AESI data were only collected based on the specified AESI categories. A Summary of all SAEs and nonserious AEs ("Other"), regardless of causality and regardless of the AESI category, is located in the "Reported Adverse Events" section. | Measured in the Safety Population in Part 1, which included all participants who received study treatment, with treatment group designated according to the treatment they received. Measured in the SRP-treated population in Part 2, which included all participants who received investigational study treatment (delandistrogene moxeparvovec) in the study. | Posted | Count of Participants | Participants | Up to 104 weeks |
|
Up to approximately 104 weeks
Part 1: data are reported in the Safety Population, which included all participants who received study treatment, with treatment group designated according to the treatment they received. Part 2: data are reported in the SRP-treated population, which included all participants who received investigational study treatment (delandistrogene moxeparvovec) in the study.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part 1: Delandistrogene Moxeparvovec | Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 of Part 1. | 0 | 63 | 14 | 63 | 62 | 63 |
| EG001 | Part 1: Placebo | Participants received a single IV infusion of matching placebo on Day 1 of Part 1. | 0 | 62 | 5 | 62 | 57 | 62 |
| EG002 | Part 2: Placebo | Participants who received delandistrogene moxeparvovec on Day 1 of Part 1 received a single IV infusion of matching placebo on Day 1 in Part 2. | 0 | 63 | 5 | 63 | 52 | 63 |
| EG003 | Part 2: Delandistrogene Moxeparvovec | Participants who received matching placebo on Day 1 in Paty 1 received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. | 0 | 60 | 8 | 60 | 55 | 60 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Toxic shock syndrome streptococcal | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Hepatotoxicity | Hepatobiliary disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Liver injury | Hepatobiliary disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Prescription drug used without a prescription | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Arterial injury | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Myocarditis | Cardiac disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Left ventricular dysfunction | Cardiac disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Forearm fracture | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA Version 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Glutamate dehydrogenase increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Cushingoid | Endocrine disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Troponin I increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA Version 26.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA Version 26.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Attention deficit hyperactivity disorder | Psychiatric disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Ketonuria | Renal and urinary disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA Version 26.0 | Systematic Assessment |
| |
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 26.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sarepta Therapeutics Inc. | Sarepta Therapeutics Inc. | 1-888-727-3782 | SareptAlly@sarepta.com |
| Nov 22, 2024 |
| Prot_SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009136 | Muscular Dystrophies |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black of African American |
|
| White |
|
| More than One Race |
|
| Other or Not Reported |
|
Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
|
|
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| Participants |
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| Participants |
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| Participants |
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| Units |
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| Counts |
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| Participants |
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| Units | Counts |
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| Participants |
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| Units | Counts |
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| Participants |
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|
|
|
|
Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2.
| OG002 | Part 2: Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 | Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2. |
| OG003 | Part 2: Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2 | Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
|
|
Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
| OG002 | Part 2: Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 | Participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 of Part 1. Then, participants received a single IV infusion of matching placebo on Day 1 in Part 2. |
| OG003 | Part 2: Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2 | Participants received a single IV infusion of matching placebo on Day 1 of Part 1. Then, participants received a single IV infusion of delandistrogene moxeparvovec on Day 1 in Part 2. |
|
|