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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-002372-13 | EudraCT Number | ||
| 2024-512626-28-00 | Other Identifier | EU Clinical Trial Number |
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| Name | Class |
|---|---|
| Hoffmann-La Roche | INDUSTRY |
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The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec followed by Placebo | Experimental | Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks. |
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| 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 approximately 72 weeks. |
|
| 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 the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 | Baseline, Week 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72 | Baseline, Week 72 | |
| Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72 | Baseline, Week 72 |
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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 Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Lucile Packard Children's Hospital Stanford |
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| Label | URL |
|---|---|
| Click here to access the website, clinicaltrials.sarepta.com/ENVISION, for additional information for the study. | View source |
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|
| placebo | Genetic | Single IV infusion of matching placebo |
|
| Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot | Week 12 |
| Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72 | Baseline, Week 72 |
| Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) | Baseline up to Week 124 |
| Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72 | Baseline, Week 72 |
| Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72 | Baseline, Week 72 |
| Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72 | Baseline, Week 72 |
| Palo Alto |
| California |
| 94304 |
| United States |
| University of California at Davis Medical Center | Sacramento | California | 95817 | United States |
| Rady Children's Hospital-San Diego | San Diego | California | 92123 | United States |
| University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases | Gainesville | Florida | 32608 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University of St. Louis, St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| University of Rochester, Department of Neurology | Rochester | New York | 14642 | United States |
| Lenox Baker Children's Hospital (Duke University) | Durham | North Carolina | 27705 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23510 | United States |
| The Children's Hospital at Westmead | Westmead | New South Wales | 2145 | Australia |
| The Royal Children's Hospital | Parkville | Victoria | 3052 | Australia |
| Universitair Ziekenhuis Gent | Ghent | Oost-Vlaanderen | 9000 | Belgium |
| The Children's Hospital of Eastern Ontario | Ottawa | Ontario | K1H8L1 | Canada |
| Research Institute McGill University Health Centre | Montreal | Quebec | H4A3J1 | Canada |
| Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval) | Québec | G1V 4G2 | Canada |
| LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt | München | Bavaria | Germany |
| Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen | Essen | North Rhine-Westphalia | Germany |
| Universitatsklinikum Hamburg Eppendorf | Hamburg | Germany |
| Hong Kong Children's Hospital | Hong Kong | Hong Kong |
| Institute of Neruology, Schneider Children's Medical Center of Israel | Petah Tikva | Israel |
| Tel Aviv Sourasky Medical Center | Tel Aviv | 6423906 | Israel |
| U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico | Genova | 16147 | Italy |
| UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo | Milan | 20133 | Italy |
| UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS | Roma | 00168 | Italy |
| National Hospital Organization Osaka Toneyama Medical Center | Toyonaka-shi | Osaka | 560-8552 | Japan |
| National Center of Neurology and Psychiatry | Kodaira | Tokyo | 187-8551 | Japan |
| Tokyo Women's Medical University Hospital | Shinjuku-ku | Tokyo | 162-866 | Japan |
| Pusan National University Yangsan Hospital | Yangsan | Gyeongsangnam-do | 50612 | South Korea |
| Seoul National University Hospital | Seoul | NAP | 03080 | South Korea |
| Kyungpook National University Hospital | Daegu | 41944 | South Korea |
| Gangnam Severance Hospital, Yonsei University Health System | Seoul | 06273 | South Korea |
| Hospital Sant Joan de Deu | Esplugues de Llobregat | Barcelona | 08950 | Spain |
| Hospital Universitari Politecnic La Fe | Valencia | 46026 | Spain |
| Sahlgrenska Universitetssjukhuset | Gothenburg | 416 85 | Sweden |
| Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen | Solna | 171 76 | Sweden |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | Taiwan |
| National Taiwan University Hospital | Taipei | Taiwan |
| Great Ormond Street Hospital for Children Foundation Trust | London | Greater London | WC1N 3JH | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | Oxfordshire | United Kingdom |
| Institute of Translational and Clinical Research | Newcastle upon Tyne | NE13BZ | United Kingdom |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| 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 |
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