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Study was Withdrawn per Sponsor decision.
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This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-9005 | Experimental | Participants will receive a single intravenous (IV) infusion of SRP-9005. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-9005 | Biological | Solution for single IV infusion |
|
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| Measure | Description | Time Frame |
|---|---|---|
| Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs) | Baseline through Month 60 | |
| Part A and B: Change from Baseline in gamma-Sarcoglycan (SG) Expression at Day 60 Post-dose in Ambulatory Participants as Measured by Immunofluorescence (IF) Percent Positive Fibers (PPF) | Baseline, Day 60 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A and B: Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score | Baseline through Month 60 | |
| Part A and B: Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23510 | United States |
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| ID | Term |
|---|---|
| D049288 | Muscular Dystrophies, Limb-Girdle |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D000305 | Adrenal Cortex Hormones |
| D011241 | Prednisone |
| ID | Term |
|---|---|
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
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This study comprises 2 parts: Part A is the Phase 1 portion, Part B is the Phase 3 expansion portion.
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| Corticosteroid | Drug | Oral tablet (prophylactic) |
|
|
| Baseline through Month 60 |
| Part A and B: Change from Baseline through Month 60 in Time to Rise From Floor | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Time to Ascend 4 Steps | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Time to Complete the 10-meter Walk/Run (10MWR) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Timed Up and Go | Baseline through Month 60 |
| Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by IF Percent Fiber Intensity (PFI) | Baseline, Day 60 |
| Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by Western Assay | Baseline, Day 60 |
| Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PPF | Baseline to Day 60 |
| Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PFI | Baseline to Day 60 |
| Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by Western Assay | Baseline to Day 60 |
| Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PPF | Baseline to Month 24 |
| Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PFI | Baseline to Month 24 |
| Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by Western Assay | Baseline to Month 24 |
| Part A and B: Change from Baseline Through Month 60 in Creatine Kinase Levels | Baseline through Month 60 |
| Part A and B: Loss of Ambulation | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imaging (MRI) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95th Percentile (SV95C) (Ambulatory Participants Only) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity Percent Predicted (FVC%p) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second % Predicted (FEV1%p) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR) | Baseline through Month 60 |
| Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Inspiratory Pressure (MIP%p) | Baseline through Month 60 |
| Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP) | Baseline through Month 60 |
| Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Expiratory Pressure (MEP%p) | Baseline through Month 60 |
| Part B: Number of Participants with TEAEs | Baseline through Month 60 |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |