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Also known as vesleteplirsen
SRP-5051 · 1 trial · 1 indication
An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug.
| Arm | Type | Description |
|---|---|---|
| SRP-5051 | EXPERIMENTAL | Patients will be sequentially assigned to receive 1 of the 5 escalating dose levels of SRP-5051 on Day 1. Patients who complete the study and continue to meet safety eligibility criteria will have the opportunity to enroll in an open-label extension study to continue to receive SRP-5051. |
| Name | Type | Description |
|---|---|---|
| SRP-5051 | DRUG | Single dose of SRP-5051 administered as an intravenous (IV) infusion. |
Inclusion Criteria: * Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping treatment * Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration with continued dosing of oral corticosteroid...
SRP-5051 is an investigational therapy being developed for Duchenne muscular dystrophy, a genetic muscle-wasting disorder. It is designed for patients whose disease is amenable to exon 51 skipping. It has been studied in a Phase 1 clinical trial and is not approved for commercial use.
SRP-5051 targets exon 51 of the dystrophin gene. By promoting skipping of exon 51 during dystrophin pre-mRNA splicing, the drug aims to restore the reading frame and enable production of a shortened but functional dystrophin protein in patients with applicable Duchenne muscular dystrophy mutations.
SRP-5051 is being developed by Sarepta Therapeutics, Inc., which trades on the Nasdaq under the ticker symbol SRPT. Sarepta is a biopharmaceutical company focused on precision genetic medicines for rare diseases, including Duchenne muscular dystrophy.
SRP-5051 is in Phase 1 clinical development. It is an investigational agent and has not been approved by the FDA or any other regulatory authority. The completed Phase 1 study evaluated safety, tolerability, and pharmacokinetics in patients with Duchenne muscular dystrophy.
SRP-5051 has been evaluated in the Phase 1 trial NCT03375255, titled a study to evaluate the safety, tolerability, and pharmacokinetics of a single dose of SRP-5051 in patients with Duchenne muscular dystrophy. The trial enrolled 15 male participants aged 12 years and older in the United States and Canada and is completed.
Yes, vesleteplirsen is another name for SRP-5051. Both names refer to the same investigational exon 51 skipping agent being developed by Sarepta Therapeutics for Duchenne muscular dystrophy. Searches for either name refer to the same Phase 1 drug candidate.