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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-503112-17-00 | Other Identifier | Clinical Trials Information System (CTIS) |
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This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-9003 | Experimental | Participants will receive a single intravenous (IV) infusion of SRP-9003. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-9003 | Biological | Solution for single IV infusion |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers | Baseline, Day 60 |
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay | Baseline, Day 60 | |
| Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI) |
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Inclusion Criteria:
Cohort 1, only ambulatory participants:
Cohort 2, only non-ambulatory participants:
Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
Able to cooperate with muscle testing
Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
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 |
|---|---|---|---|---|---|---|
| University of California, San Diego-Altman Clinical and Translational Research Institute | La Jolla | California | 92037 | United States | ||
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| Glucocorticoid | Drug | Oral tablet (prophylactic) |
|
|
| Baseline, Day 60 |
| Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF) | Baseline, Day 60 |
| Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score | Baseline through Month 60 |
| Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score | Baseline through Month 60 |
| Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test | Baseline through Month 60 |
| Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test | Baseline through Month 60 |
| Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test | Baseline through Month 60 |
| Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test | Baseline through Month 60 |
| Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test | Baseline through Month 60 |
| Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs) | Baseline through Month 60 |
| Change From Baseline Through Month 60 in Creatine Kinase Level | Baseline through Month 60 |
| Time to Change of Loss of Ambulation | Baseline through Month 60 |
| Nationwide Childrens Hospital |
| Columbus |
| Ohio |
| 43205 |
| United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of The King's Daughter | Norfolk | Virginia | 23507 | United States |
| University Hospital Leuven (UZ Leuven) | Leuven | Vlaams Brabant | 3000 | Belgium |
| NMRC Gent (UZ Gent) | Ghent | 9000 | Belgium |
| Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum | Essen | North Rhine-Westphalia | 45147 | Germany |
| Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Hospital Sant Joan de Deu | Barcelona | 8950 | Spain |
| Newcastle University | Newcastle upon Tyne | NE1 3BZ | United Kingdom |
| 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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| ID | Term |
|---|---|
| D005938 | Glucocorticoids |
| D011241 | Prednisone |
| ID | Term |
|---|---|
| D000305 | Adrenal Cortex Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D045505 | Physiological Effects of Drugs |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
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