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Study is being terminated due to a business decision.
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The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-6004 | Experimental | Participants will receive single IV infusion of SRP-6004 on Day 1. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-6004 | Genetic | Single IV infusion of SRP-6004 |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) | Baseline up to Month 60 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot | Baseline, Day 90 and Month 15 | |
| Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity | Baseline, Day 90 and Month 15 |
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Inclusion Criteria:
Exclusion Criteria:
Note: 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 |
|---|---|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
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| Change from Baseline in Percent of Normal DYSF Protein Expression as Assessed by IF Percent DYSF Positive Fibers (PPF: DYSF) | Baseline, Day 90 and Month 15 |
| ID | Term |
|---|---|
| D049288 | Muscular Dystrophies, Limb-Girdle |
| C535899 | Limb-girdle muscular dystrophy, type 2B |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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