Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT01540409|
Recruitment Status : Completed
First Posted : February 28, 2012
Results First Posted : July 10, 2019
Last Update Posted : March 30, 2020
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|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy (DMD)||Drug: AVI-4658 (Eteplirsen)||Phase 2|
This is an open label, multiple dose extension study to assess the ongoing efficacy, safety, and tolerability of weekly intravenous (IV) infusions of eteplirsen in DMD subjects who have successfully completed Study 4658-us 201.
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201|
|Actual Study Start Date :||February 27, 2012|
|Actual Primary Completion Date :||April 15, 2016|
|Actual Study Completion Date :||August 16, 2017|
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Drug: 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 Name: EXONDYS 51
- Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240 [ Time Frame: Parent Baseline and Week 240 ]This study used a modified version of the 6MWT test procedure described in American Thoracic Society (ATS) 2002 guidelines, specifically adapted for patients with Duchenne muscular dystrophy. The participant was asked to walk a set course of 25 meters for 6 minutes (timed) and the distance walked in meters was recorded. Increases from baseline in 6MWT distance are indicative of improvement and decreases from baseline indicate worsening. Baseline here corresponds to the baseline in the parent study (4658-us-201, NCT01396239).
- Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48 [ Time Frame: Parent Baseline and Week 48 ]Dystrophin expression as assessed by percent dystrophin positive fibers was measured by immunohistochemistry (IHC) technique using primary anti-dystrophin antibody. Percent change from baseline is the arithmetic difference of the treatment time point minus baseline divided by baseline calculated for individual subjects. Baseline here corresponds to the baseline in the parent study (4658-us-201, NCT01396239).
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:||7 Years to 13 Years (Child)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
A subject must meet all of the following criteria to be eligible for this study.
- The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
- The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
- The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.
A subject who meets any of the following criteria will be excluded from this study.
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.
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): NCT01540409
|United States, California|
|Miller Children's Hospital|
|Long Beach, California, United States, 90806|
|United States, Florida|
|University of Florida Clinical Research Center|
|Gainesville, Florida, United States, 32610|
|United States, Illinois|
|Rush University Medical Center|
|Chicago, Illinois, United States, 60612|
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, Missouri|
|Washington University Medical School|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|Summerwood Pediatrics/Infusacare Medical Services|
|Liverpool, New York, United States, 13088|
|United States, North Carolina|
|Levine Children's Hospital|
|Charlotte, North Carolina, United States, 28203|
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15224|
|United States, Virginia|
|Children's Specialty Group, Pediatric Neurology|
|Norfolk, Virginia, United States, 23510|
|United States, Wisconsin|
|Osceola Medical Center|
|Osceola, Wisconsin, United States, 54020|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Sarepta Therapeutics, Inc.|
|Other Study ID Numbers:||
|First Posted:||February 28, 2012 Key Record Dates|
|Results First Posted:||July 10, 2019|
|Last Update Posted:||March 30, 2020|
|Last Verified:||March 2020|
DMD, Duchenne, Eteplirsen, dystrophy, dystrophin, exon 51
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked