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

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK)
of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable
to exon 51 skipping treatment.

Eligibility Criteria

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, or has not received corticosteroids for at least 12 weeks prior
to study drug administration

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 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 for any of the following: angiotensin
converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs),
beta-blockers, potassium

- Requires antiarrhythmic and/or antidiuretic 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

- Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any
experimental gene therapy for the treatment of DMD at any time

Principal Investigator

Jeffrey Statland, MD

Study Contact

Kiley Higss, ksims2@kumc.edu, 913-945-9922, Heather Minchew 913-945-9920 hminchew@kumc.edu

Estimated Completion Date

Tuesday, January 1, 2019

ClinicalTrials.gov #