Summary

for males ages 12 years and up (full criteria)
at Sacramento, California and other locations
study started
estimated completion

Description

Summary

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.

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

Keywords

Muscular Dystrophy, Duchenne Duchenne Muscular Dystrophy Exon Skipping DMD Exon 51 Ambulatory Pediatric Nonambulatory Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) Duchenne Muscular Dystrophies SRP-5051

Eligibility

You can join if…

Open to males ages 12 years and up

  • 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 CAN'T join 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.

Locations

  • Neuromuscular Research Center accepting new patients
    Sacramento California 95817 United States
  • University of Kansas Medical Center accepting new patients
    Kansas City Kansas 66160 United States
  • Children's Medical Center Dallas accepting new patients
    Dallas Texas 75207 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics
Links
Sign up for this study
ID
NCT03375255
Phase
Phase 1
Study Type
Interventional
Last Updated