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Trial record 1 of 3 for:    SRP-5051
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An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03675126
Recruitment Status : Recruiting
First Posted : September 18, 2018
Last Update Posted : June 18, 2020
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: SRP-5051 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Actual Study Start Date : December 19, 2018
Estimated Primary Completion Date : July 31, 2024
Estimated Study Completion Date : July 31, 2024

Arm Intervention/treatment
Experimental: SRP-5051
Patients will receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 studies.
Drug: SRP-5051
SRP-5051 administered as an IV infusion.

Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]

Secondary Outcome Measures :
  1. Pharmacokinetic (PK) Plasma Concentration of SRP-5051 [ Time Frame: End of infusion ]
  2. Number of participants with clinically relevant abnormalities, as assessed by vital sign measurements, physical examination findings, clinical laboratory tests and electrocardiograms (ECGs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
    *A clinically relevant abnormality is an abnormality confirmed by repeat testing that is changed sufficiently from screening/baseline so that, in the judgment of the Investigator, a change in management is warranted.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.

Exclusion Criteria:

  • Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) since completing a study administering SRP-5051 and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids*.
  • Requires antiarrhythmic and/or diuretic therapy for heart failure.
  • Use of any herbal medication/supplement containing aristolochic acid.
  • Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time.
  • Participation in an interventional clinical trial since completing original study.

Other inclusion/exclusion criteria apply.

* The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03675126

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Contact: Medical information +1-800-690-2003

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United States, Florida
NW FL Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
Contact: Shezsay Colbert    850-934-1299   
Principal Investigator: Weldon Mauney, MD         
United States, Georgia
Center for Integrative Rare Disease Research (CIRDR) Recruiting
Atlanta, Georgia, United States, 30318
Contact    678-883-6897   
Principal Investigator: Han C Phan, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Bobby Lindquist    312-227-4449   
Principal Investigator: Nancy Kuntz, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Katherine Roath    913-945-9928   
Principal Investigator: Jeffrey Statland, MD         
United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer Monahan, RN    412-692-6106   
Principal Investigator: Hoda Abdel-Hamid, MD         
United States, Texas
Children's Medical Center Dallas Recruiting
Dallas, Texas, United States, 75207
Contact: Kristy Riddle    214-456-9501   
Principal Investigator: Diana Castro, MD         
Canada, Ontario
London Health Sciences Centre Recruiting
London, Ontario, Canada, N6A 5W9
Contact: Maysaa Assaf    519-685-8500 ext 75556   
Principal Investigator: Craig Campbell, MD         
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
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Study Director: Medical Director Sarepta Therapeutics, Inc.
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Responsible Party: Sarepta Therapeutics, Inc. Identifier: NCT03675126    
Other Study ID Numbers: 5051-102
First Posted: September 18, 2018    Key Record Dates
Last Update Posted: June 18, 2020
Last Verified: June 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
Duchenne muscular dystrophy
Exon Skipping
Exon 51
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked