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| ID | Type | Description | Link |
|---|---|---|---|
| IRB17-00253 | Other Identifier | Sarepta Therapeutics |
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Study is being terminated due to a business decision.
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The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: SRP-9003 | Experimental | Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose. |
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| Cohort 2: SRP-9003 | Experimental | Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-9003 | Genetic | SRP-9003 will be administered through a single systemic injection. |
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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 7 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot | β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies. | Baseline, Day 60 |
| Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence |
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INCLUSION CRITERIA
EXCLUSION CRITERIA
Other inclusion/exclusion criteria 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 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38177855 | Derived | Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4. |
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β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies. |
| Baseline, Day 60 |
| Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers | Baseline, Day 60 |
| ID | Term |
|---|---|
| C535902 | Limb-girdle muscular dystrophy, type 2E |
| D049288 | Muscular Dystrophies, Limb-Girdle |
| 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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