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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-000691-19 | EudraCT Number | ||
| 2023-509901-57-00 | EU Trial (CTIS) Number |
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| Name | Class |
|---|---|
| Sarepta Therapeutics, Inc. | INDUSTRY |
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This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | Single IV infusion of delandistrogene moxeparvovec |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) | Baseline up to Week 260 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot | Baseline, Week 12 |
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Inclusion Criteria:
Exclusion Criteria:
Other inclusion or exclusion criteria could apply
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chr de La Citadelle | Liège | 3500 | Belgium | |||
| Hôpital Necker-Enfants Malades |
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
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| Paris |
| 75015 |
| France |
| Universitätsklinikum Essen | Essen | 45147 | Germany |
| PU A. Gemelli, Università Cattolica del Sacro Cuore | Rome | Lazio | 00168 | Italy |
| Hospital Sant Joan De Deu | Esplugues de Llobregas | Barcelona | 08950 | Spain |
| Great Ormond Street Hospital for Children | London | WC1N 3JH | United Kingdom |
| John Radcliffe Hospital | Oxford | OX3 9DU | 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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