SRP-5051
DrugSingle dose of SRP-5051 administered as an intravenous (IV) infusion.
Other names: vesleteplirsen
NCT Number: NCT03375255
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.
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Notify Me12 year and older
Male
Interventional
Phase 1
London Health Sciences Centre, London, Ontario, Canada
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Other inclusion/exclusion criteria apply.
*The dose of steroids must remain constant except for modifications to accommodate changes in weight.
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
Other names: vesleteplirsen
Time frame: From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks)
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: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)
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)
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Sarepta Therapeutics, Inc.
Industry
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
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View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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