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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-505043-39-00 | Other Identifier | Clinical Trials Information System (CTIS) |
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| Name | Class |
|---|---|
| Hoffmann-La Roche | INDUSTRY |
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The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants received delandistrogene moxeparvovec in a previous clinical study. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) | Up to 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years | |
| Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| UCLA Medical Center |
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| Baseline, up to 10 years |
| Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec | Baseline, up to 10 years |
| Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation | Baseline, up to 10 years |
| Los Angeles |
| California |
| 90095 |
| United States |
| Lucile Packard Children's Hospital Stanford (LPCH) | Palo Alto | California | 94304 | United States |
| University of California, Davis | Sacramento | California | 95817 | United States |
| Rady Children's Hospital | San Diego | California | 92123 | United States |
| Children's Hospital of Colorado | Aurora | Colorado | 80045-7106 | United States |
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Iowa ICTS Clinical Research Unit | Iowa City | Iowa | 52242 | United States |
| The Johns Hopkins Hospital, Rubenstein Child Health Bldg | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Columbia University Pediatric Neuromuscular Center | New York | New York | 10032 | United States |
| University of Rochester Medical Center | 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 |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Chiildren's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Health Specialty Center | Dallas | Texas | 75207 | United States |
| University of Utah Health | Salt Lake City | Utah | 84132 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23510 | United States |
| Children's Hospital Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC) | Ghent | East Flanders | 9000 | Belgium |
| Universitätsklinikum Essen - Klinik für Kinderheilkunde I | Essen | North Rhine-Westphalia | 45147 | Germany |
| Hong Kong Children's Hospital | Hong Kong | Hong Kong |
| UOC Neurologia Pediatrica e Malattie Muscolari, 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 |
| UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore | Roma | 00168 | Italy |
| Kobe University Hospital | Kobe | Hyōgo | 650-0017 | Japan |
| National Center of Neurology and Psychiatry | Kodaira | Tokyo | 187-8551 | Japan |
| Tokyo Women's Medical University Hospital | Tokyo | 1628666 | Japan |
| Hospital Sant Joan de Deu | Esplugues de Llobregat | Barcelona | 08950 | Spain |
| Hospital Universitario y Politecnico La fe. Neurology Department | Valencia | 46026 | Spain |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | 807 | Taiwan |
| National Taiwan University Hospital | Taipei | 100225 | Taiwan |
| Great Ormond Street Hospital For Children NHS Foundation Trust | London | Greater London | WC1N 1EJ | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | Oxfordshire | OX3 9DU | United Kingdom |
| Institute of Translational and Clinical Research | Newcastle upon Tyne | NE1 3BZ | 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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