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
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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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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Gainesville, FL
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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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Gulf Breeze, Florida 32561
Principal Investigator: Weldon Mauney, MD
Phone: 850-934-1299
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Gulf Breeze, FL
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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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London, Ontario
Principal Investigator: Craig Campbell, MD
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London,
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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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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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