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An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

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ClinicalTrials.gov Identifier: NCT03675126
Recruitment Status : Active, not recruiting
First Posted : September 18, 2018
Last Update Posted : May 6, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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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

Study Design
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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

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Arms and Interventions
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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.


Outcome Measures
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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.


Eligibility Criteria
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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
Criteria

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.

Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT03675126


Locations
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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
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
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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: May 6, 2021
Last Verified: May 2021

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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
DMD
Exon 51
Ambulatory
 Pediatric
Nonambulatory
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Duchenne
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