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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, also known as patidistrogene bexoparvovec, is an investigational gene therapy being studied for the treatment of Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D). It is designed to address the underlying genetic cause of this condition, though it is not yet approved and remains in clinical development.
SRP-9004 is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SRPT. The company is conducting clinical research to evaluate the safety and efficacy of this gene therapy for Limb-Girdle Muscular Dystrophy, Type 2D.
SRP-9004 is in Phase 1 clinical development. It is an investigational gene therapy for Limb-Girdle Muscular Dystrophy, Type 2D, and is not yet approved by regulatory authorities. The Phase 1 study has been completed, and the therapy is still in the early stages of clinical evaluation.
SRP-9004 has been studied in one clinical trial, identified as NCT01976091. This Phase 1 study, 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),' has been completed and enrolled 6 participants.
Yes, SRP-9004 is also known as patidistrogene bexoparvovec. This alternative name is used in clinical trial documentation, including the study NCT01976091, which evaluates the safety of SRP-9004 in participants with Limb-Girdle Muscular Dystrophy, Type 2D.
SRP-9004 is a gene therapy, a modality that involves delivering genetic material to cells to treat or prevent disease. In the context of Limb-Girdle Muscular Dystrophy, Type 2D, this approach aims to address the genetic defect underlying the condition, though specific details of its mechanism are not provided.