Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
109 participants
INTERVENTIONAL
2014-11-17
2019-06-14
Brief Summary
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Detailed Description
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Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Treated Group
Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping will receive 30 mg/kg of eteplirsen weekly for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Untreated Group
Approximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
No interventions assigned to this group
Interventions
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eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Diagnosed with DMD, genotypically confirmed
* Stable dose of corticosteroids for at least 24 weeks
* Have intact right and left alternative upper muscle groups
* Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
* Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
Exclusion Criteria
* Participation in any other DMD interventional clinical study within 12 weeks
* Major surgery within 3 months
* Presence of other clinically significant illness
* Major change in the physical therapy regime within 3 months
7 Years
16 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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Neuromuscular Research Center
Phoenix, Arizona, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
University of California, Davis Medical Center
Sacramento, California, United States
Rady Children's Hospital, U.C. San Diego
San Diego, California, United States
Stanford University School of Medicine/Medical Center
Stanford, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Children's National Health System
Washington D.C., District of Columbia, United States
The University of Florida, Powell Gene Therapy Center
Gainesville, Florida, United States
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Rare Disease Research Center
Atlanta, Georgia, United States
Emory University
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Children's Hospital Boston
Boston, Massachusetts, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States
Columbia University Medical Center
New York, New York, United States
University of Rochester Clinical Research Center
Rochester, New York, United States
Levine Childrens Hospital, Carolinas Medical Center
Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center (CCHMC)
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Shriners Hospital for Children
Portland, Oregon, United States
Penn State Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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References
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Brogna C, Coratti G, Pane M, Ricotti V, Messina S, D'Amico A, Bruno C, Vita G, Berardinelli A, Mazzone E, Magri F, Ricci F, Mongini T, Battini R, Bello L, Pegoraro E, Baranello G, Previtali SC, Politano L, Comi GP, Sansone VA, Donati A, Bertini E, Muntoni F, Goemans N, Mercuri E; on behalf on the International DMD group. Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53. PLoS One. 2019 Jun 25;14(6):e0218683. doi: 10.1371/journal.pone.0218683. eCollection 2019.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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4658-301
Identifier Type: -
Identifier Source: org_study_id
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