Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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, also known as vesleteplirsen, is an investigational small molecule being developed for the treatment of Duchenne muscular dystrophy (DMD). It is designed for patients with this genetic muscle-wasting condition. The drug is currently in clinical development and has not been approved by regulatory authorities.
SRP-5051 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 tolerability of this investigational drug for Duchenne muscular dystrophy.
SRP-5051 is in Phase 1 clinical development. A Phase 1 study has been completed to evaluate its safety, tolerability, and pharmacokinetics in patients with Duchenne muscular dystrophy. The drug remains investigational and is not yet approved for commercial use.
SRP-5051 has been studied in one completed Phase 1 clinical trial, identified as NCT03375255. This trial evaluated the safety, tolerability, and pharmacokinetics of a single dose of SRP-5051 in patients with Duchenne muscular dystrophy. The study enrolled 15 male participants aged 12 years and older in the United States and Canada.
Yes, SRP-5051 is also known as vesleteplirsen. Both names refer to the same investigational drug being developed by Sarepta Therapeutics for Duchenne muscular dystrophy. The alternative name may be used in clinical trial registries and scientific literature.