A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

Completed · Phase 1

Conditions studied: Muscular Dystrophy, Duchenne

In brief

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.

Key facts

Study ID

NCT03375255

Run by

Sarepta Therapeutics, Inc.

People needed

15

Starts

2018-02-05

Expected to finish

2019-08-19

Last updated by the study team

2022-07-06

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• 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 — 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

Full record on ClinicalTrials.gov

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