Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
NCT ID: NCT02420379
Last Updated: 2021-01-25
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
33 participants
INTERVENTIONAL
2015-06-30
2018-12-17
Brief Summary
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Detailed Description
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Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy.
Population and serial PK will be collected.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Control Group
Approximately 20 patients with DMD not amenable to exon 51 skipping will be observed for 96 weeks.
No interventions assigned to this group
Open-Label
Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg .
eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.
Interventions
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eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Diagnosis of DMD, genotypically confirmed.
* Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
* Intact right and left biceps muscles or two alternative upper arm muscle groups.
* Parent that is willing to provide consent and comply with study procedures.
Exclusion Criteria
* Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
* Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
* Presence of other clinically significant illness.
4 Years
6 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 of Arizona
Phoenix, Arizona, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
University of California, Davis Medical Center
Sacramento, California, United States
Stanford University Medical Center
Stanford, California, United States
University of Florida, Shands Hospital
Gainesville, Florida, United States
Rare Disease Research Center
Atlanta, Georgia, United States
Children's Hospital of Atlanta
Atlanta, Georgia, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Shriners Hospital for Children
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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Provided Documents
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Document Type: Statistical Analysis Plan
Document Type: Study Protocol
Other Identifiers
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4658-203
Identifier Type: -
Identifier Source: org_study_id
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