An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03675126 |
Recruitment Status :
Terminated
(The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B.)
First Posted : September 18, 2018
Last Update Posted : November 24, 2021
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Condition or disease | Intervention/treatment | Phase |
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Muscular Dystrophy, Duchenne | Drug: SRP-5051 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 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 |
Actual Primary Completion Date : | August 25, 2021 |
Actual Study Completion Date : | August 25, 2021 |

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.
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Drug: SRP-5051
SRP-5051 administered as an IV infusion. |
- Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
- Pharmacokinetic (PK) Plasma Concentration of SRP-5051 [ Time Frame: End of infusion ]
- 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.

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.
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.

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 ClinicalTrials.gov identifier (NCT number): NCT03675126
United States, Connecticut | |
Connecticut Children's Medical Center | |
Hartford, Connecticut, United States, 06106 | |
United States, Florida | |
NW FL Clinical Research Group, LLC | |
Gulf Breeze, Florida, United States, 32561 | |
United States, Georgia | |
Center for Integrative Rare Disease Research (CIRDR) | |
Atlanta, Georgia, United States, 30318 | |
United States, Illinois | |
Ann & Robert H. Lurie Children's Hospital of Chicago | |
Chicago, Illinois, United States, 60611 | |
United States, Kansas | |
University of Kansas Medical Center | |
Kansas City, Kansas, United States, 66160 | |
United States, Pennsylvania | |
UPMC Children's Hospital of Pittsburgh | |
Pittsburgh, Pennsylvania, United States, 15224 | |
United States, Texas | |
Children's Medical Center Dallas | |
Dallas, Texas, United States, 75207 | |
Canada, Ontario | |
London Health Sciences Centre | |
London, Ontario, Canada, N6A 5W9 |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT03675126 |
Other Study ID Numbers: |
5051-102 |
First Posted: | September 18, 2018 Key Record Dates |
Last Update Posted: | November 24, 2021 |
Last Verified: | September 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
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