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



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:12 - Any
Updated:12/20/2018
Start Date:February 5, 2018
End Date:September 1, 2019
Contact:Medical Information
Email:clinicaltrials@sarepta.com
Phone:+1-888-727-3782

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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

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.


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.
We found this trial at
12
sites
London, Ontario
Principal Investigator: Craig Campbell, MD
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barry Byrne, MD
Phone: 352-294-8705
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Hoda Z Abdel-Hamid, MD
Phone: 412-692-5176
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Pittsburgh, PA
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Atlanta, Georgia 30318
Principal Investigator: Han C Phan, MD
Phone: 678-883-6897
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Atlanta, GA
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 312-227-3019
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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Columbus, Ohio 43205
Principal Investigator: Jerry R Mendell, MD
Phone: 614-722-2699
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Columbus, OH
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1935 Medical District Dr
Dallas, Texas 75235
(214) 456-7000
Principal Investigator: Diana Castro, MD
Phone: 214-456-2463
Children's Medical Center of Dallas Children's Medical Center is private, not-for-profit, and is the fifth-largest...
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Dallas, TX
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Gulf Breeze, Florida 32561
Principal Investigator: Weldon Mauney, MD
Phone: 850-934-1299
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Jeffrey Statland, MD
Phone: 913-945-9922
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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10833 Le Conte Ave
Los Angeles, California 90095
(310) 825-4321
Principal Investigator: Perry Shieh, MD, PhD
Phone: 310-825-3264
David Geffen School of Medicine, UCLA In 2002 Mr. David Geffen announced a $200 million...
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Los Angeles, CA
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Sacramento, California 95817
Principal Investigator: Craig McDonald, MD
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Saint Louis, Missouri 63110
Principal Investigator: Anne Connolly, MD
Phone: 314-362-1146
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Saint Louis, MO
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