A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
Active, not recruiting · NCT05876780 · Registry updated 2026-02-19Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, limb-girdle muscular dystrophy, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 1 | 2026-02-19 | NCT05876780 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Sarepta Therapeutics, Inc. | 15 |
2 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Nationwide Children's Hospital | Columbus | United States | 309 |
| St. Jude Children's Research Hospital | Memphis | United States | 194 |