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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-05

Study Completion Date

2019-08-19

Brief Summary

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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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Detailed Description

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Conditions

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Muscular Dystrophy, Duchenne

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

SRP-5051

Intervention Type DRUG

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

Interventions

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SRP-5051

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

Intervention Type DRUG

Other Intervention Names

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vesleteplirsen

Eligibility Criteria

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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

\*The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Minimum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No