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

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03375255
• Other study ID #: 5051-101
• Status: Completed
• Phase: Phase 1
• Start date: February 5, 2018
• Est. completion date: August 19, 2019

Study information

• Verified date: June 2022
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: August 19, 2019
• Est. primary completion date: August 19, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• 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 Criteria:

• 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.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• SRP-5051
Single dose of SRP-5051 administered as an intravenous (IV) infusion.

Locations

Country	Name	City	State
Canada	London Health Sciences Centre	London	Ontario
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Children's Medical Center Dallas	Dallas	Texas
United States	NW FL Clinical Research Group, LLC	Gulf Breeze	Florida
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Neuromuscular Research Center	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks)
Secondary	Maximum Plasma concentration (Cmax) of SRP-5051	Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.	Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)
Secondary	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.	Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)