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 | 
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.
			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
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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
	
			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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								Atlanta, Georgia 30318			
	
			
					Principal Investigator: Han C Phan, MD
			
						
										Phone: 678-883-6897
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									225 E Chicago Ave
Chicago, Illinois 60611
	
			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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								Columbus, Ohio 43205			
	
			
					Principal Investigator: Jerry R Mendell, MD
			
						
										Phone: 614-722-2699
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									1935 Medical District Dr
Dallas, Texas 75235
	
			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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								Gulf Breeze, Florida 32561			
	
			
					Principal Investigator: Weldon Mauney, MD
			
						
										Phone: 850-934-1299
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									3901 Rainbow Blvd
Kansas City, Kansas 66160
	
			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
	
			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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