Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
NCT ID: NCT01540409
Last Updated: 2020-03-30
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
12 participants
INTERVENTIONAL
2012-02-27
2017-08-16
Brief Summary
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Detailed Description
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Subjects will have the opportunity to enroll in this study during the last visit of Study 4658-us-201 (Week 28). Eligible subjects will receive once weekly IV infusions of eteplirsen (50 or 30 mg/kg) for an additional 212 weeks. Subjects will receive the same dose of eteplirsen they received in Study 4658-us-201. Subjects will thereafter continue to receive once weekly IV infusions of eteplirsen for up to an additional 72 week period (through week 284). If commercial eteplirsen becomes available during this additional 72 week period, participation in the study will be discontinued as subjects transition to commercial eteplirsen.
Safety, efficacy, pharmacokinetic (PK), and biomarker assessments will be performed at scheduled visits; adverse events (AEs) and concomitant medications and therapies will be continuously monitored.
If review of data from this open label study suggests that continued treatment with eteplirsen is warranted, this study may be extended by protocol amendment or subjects who successfully complete this study may have the opportunity to participate in a separate follow on, open label eteplirsen study.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion. There are two treatment groups, 30 mg/kg and 50 mg/kg.
Interventions
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AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion. There are two treatment groups, 30 mg/kg and 50 mg/kg.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.
Exclusion Criteria
1\. The subject has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the subject or make it unlikely that the course of treatment or follow-up would be completed or impair the assessment of study results.
7 Years
13 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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Miller Children's Hospital
Long Beach, California, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
Rush University Medical Center
Chicago, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Washington University Medical School
St Louis, Missouri, United States
Summerwood Pediatrics/Infusacare Medical Services
Liverpool, New York, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Children's Specialty Group, Pediatric Neurology
Norfolk, Virginia, United States
Osceola Medical Center
Osceola, Wisconsin, United States
Countries
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References
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Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016 Feb;79(2):257-71. doi: 10.1002/ana.24555. Epub 2016 Jan 8.
Related Links
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Mendell, 2013, Annals of Neurology
Mendell, 2016, Annals of Neurology
Kinane, 2018, Journal of Neuromuscular Diseases
Charleston, 2018, Neurology
Other Identifiers
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4658-us-202
Identifier Type: -
Identifier Source: org_study_id
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