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Also known as patidistrogene bexoparvovec
SRP-9004 · 1 trial · 1 indication
An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug related. An AE was considered serious if, in the view of the investigator or sponsor, it resulted in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Treatment-related Treatment Emergent Adverse Event (TEAE) is defined as an TEAE that was classified by the investigator as related to treatment.
| Arm | Type | Description |
|---|---|---|
| Cohort 1A: SRP-9004 Low Dose (Single Limb Perfusion) | EXPERIMENTAL | Non-ambulant participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to a single limb on Day 0. |
| Cohort 1B Low Dose (Bilateral Limb Perfusion) | EXPERIMENTAL | Participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to both limbs on Day 0. |
| Cohort 2 High Dose (Bilateral Limb Perfusion) | EXPERIMENTAL | Participants with LGMD2D will receive 1 high dose of SRP-9004 via ILI to both limbs on Day 0. |
| Name | Type | Description |
|---|---|---|
| SRP-9004 | GENETIC | Isolated Limb Infusion (ILI) |
Key Inclusion Criteria: * Cohort 1A must be adult and wheelchair-dependent; Cohorts 1B and 2 will be participants of age 7 or older. * Confirmed alpha-sarcoglycan deficiency or identified sarcoglycan alpha (SGCA) deoxyribonucleic acid (DNA) mutation. * Participants enrolled in Cohorts 1B or 2 must ...
SRP-9004 is an investigational gene therapy developed by Sarepta Therapeutics for Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D). It is designed to deliver a functional copy of the sarcoglycan gene to muscle cells. It has been studied in a single Phase 1 clinical trial and is not approved for commercial use.
SRP-9004 is being developed as a treatment for Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D), a genetic muscle-wasting disorder. The goal of the gene therapy is to address the underlying genetic defect that causes the condition. It remains investigational and has not been approved for any indication.
SRP-9004 targets sarcoglycan. LGMD2D is caused by mutations affecting the sarcoglycan protein complex in muscle. The gene therapy is designed to deliver genetic material intended to restore sarcoglycan expression in affected muscle tissue. This target-directed approach is the basis of its development as a gene transfer therapy.
SRP-9004 is being developed by Sarepta Therapeutics, Inc., which trades on the Nasdaq under the ticker SRPT. Sarepta is a biopharmaceutical company focused on genetic medicines, including gene therapies for rare neuromuscular disorders. SRP-9004 is part of its pipeline for Limb-Girdle Muscular Dystrophy, Type 2D.
SRP-9004 has completed a Phase 1 clinical trial. It is an investigational gene therapy and has not been approved by the FDA for any indication. The Phase 1 study has been completed, and no active trials for SRP-9004 are currently listed in the clinical trial registry.
SRP-9004 has been evaluated in one clinical trial, NCT01976091, titled A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D). The Phase 1 trial enrolled 6 participants, was controlled, and has been completed.
Yes, SRP-9004 is also known as patidistrogene bexoparvovec. The full name appears in the title of its Phase 1 clinical trial, NCT01976091, which studied the gene transfer therapy in participants with Limb-Girdle Muscular Dystrophy, Type 2D. Both names refer to the same investigational Sarepta Therapeutics gene therapy candidate.