A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment
Sponsor: Sarepta Therapeutics, Inc.
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Number of Participants with Adverse Events (AEs)
Time frame: From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks)
Secondary Endpoints
Maximum Plasma concentration (Cmax) of SRP-5051
Area under the plasma concentration versus time curve (AUC) of SRP-5051
Eligibility Criteria
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 corticosteroids while participating in the study\*, or has not received corticosteroids for at least 12 weeks prior to study drug administration and will not initiate dosing of oral corticosteroids while participating in the study Exclusion Criteria: * Has a left ventricular ejection fraction (LVEF) less than (\<) 40 percent (%) based on an echocardiogram (ECHO) performed within 3 months prior to Screening or at the Screening visit * Has a QT interval corrected with Fridericia's method (QTcF) \>= 450 millisecond (msec) on the Screening electrocardiogram (ECG) * Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening and while particip…
Read full criteria on CT.gov →✦ Analyst Commentary
Expert commentary on why this trial matters and what to watch for.
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