Status:
COMPLETED
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
12+ years
Phase:
PHASE1
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 amena...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 5 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 19 2019
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT03375255
Start Date
February 5 2018
End Date
August 19 2019
Last Update
July 6 2022
Active Locations (8)
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1
Neuromuscular Research Center
Sacramento, California, United States, 95817
2
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
3
Rare Disease Research, LLC
Atlanta, Georgia, United States, 30318
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611