A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Drug: SRP-5051
- Registration Number
- NCT03375255
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 15
- 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
- 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 participating in the study for any of the following: angiotensin-converting enzyme (ACE) inhibitors, angiotensin-receptor blocking agents (ARBs), beta-blockers, or potassium
- Requires antiarrhythmic and/or diuretic therapy for heart failure
- Forced vital capacity (FVC) <40% of predicted value within 3 months of Screening or at the Screening visit
- Known kidney disease or had an acute kidney injury within 6 months prior to Screening
- Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any experimental gene therapy for the treatment of DMD at any time
- Use of any herbal medication/supplement containing aristolochic acid
Other inclusion/exclusion criteria apply.
*The dose of steroids must remain constant except for modifications to accommodate changes in weight.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description SRP-5051 SRP-5051 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.
- Primary Outcome Measures
Name Time Method Number of Participants with Adverse Events (AEs) 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.
- Secondary Outcome Measures
Name Time Method Maximum Plasma concentration (Cmax) of SRP-5051 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.
Area under the plasma concentration versus time curve (AUC) of SRP-5051 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.
Trial Locations
- Locations (8)
Neuromuscular Research Center
🇺🇸Sacramento, California, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
NW FL Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, 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
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States