The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
Neuromuscular Research Center
Sacramento, California, United States
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Number of Participants with Adverse Events (AEs)
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.
Time frame: From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks)
Maximum Plasma concentration (Cmax) of SRP-5051
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Time frame: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)
Area under the plasma concentration versus time curve (AUC) of SRP-5051
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Time frame: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Children's Medical Center Dallas
Dallas, Texas, United States
London Health Sciences Centre
London, Ontario, Canada