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Study is being terminated due to a business decision.
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The primary objective of this study is to evaluate the safety of SRP-9004.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-9004 | Experimental | Participants will receive a single dose of SRP-9004 on Day 1. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-9004 | Drug | Intravenous (IV) infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs) | Up to 60 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline Through Day 60 in Quantity of Alpha-sarcoglycan (α-SG) Protein Expression | Baseline, Day 60 | |
| Change from Baseline Through Month 24 in Quantity of α-SG Protein Expression | Baseline, Month 24 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other inclusion/exclusion criteria apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States | ||
| Children's Hospital of the King's Daughters |
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| Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Time to Rise From Floor | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Time to Ascend 4 Steps | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Time to Complete 10-meter Walk/Run (10MWR) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR) | Baseline, Month 60 |
| Change from Baseline through Month 60 in Creatine Kinase (CK) Level | Baseline, Month 60 |
| Time to Change of Disease Milestones (Loss of Ambulation) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imagining (MRI) Findings | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95% (SV95C) (Ambulatory Participants Only) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FVC Percentage (%) Predicted | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FEV1 % Predicted | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP) | Baseline, Month 60 |
| Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP) | Baseline, Month 60 |
| Norfolk |
| Virginia |
| 23510 |
| United States |
| ID | Term |
|---|---|
| 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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