Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201
1 other identifier
interventional
12
1 country
11
Brief Summary
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2012
Longer than P75 for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 23, 2012
CompletedStudy Start
First participant enrolled
February 27, 2012
CompletedFirst Posted
Study publicly available on registry
February 28, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 15, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
August 16, 2017
CompletedResults Posted
Study results publicly available
July 10, 2019
CompletedMarch 30, 2020
March 1, 2020
4.1 years
February 23, 2012
June 20, 2019
March 26, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Change From Baseline in the 6 Minute Walk Test (6MWT) at 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).
Parent Baseline and Week 240
Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at 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).
Parent Baseline and Week 48
Study Arms (1)
AVI-4658 (Eteplirsen)
EXPERIMENTALMultiple-Dose Extension Study
Interventions
Eteplirsen will be administered once weekly via an IV infusion. There are two treatment groups, 30 mg/kg and 50 mg/kg.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- A subject who meets any of the following criteria will be excluded from this study.
- \. 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Miller Children's Hospital
Long Beach, California, 90806, United States
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Washington University Medical School
St Louis, Missouri, 63110, United States
Summerwood Pediatrics/Infusacare Medical Services
Liverpool, New York, 13088, United States
Levine Children's Hospital
Charlotte, North Carolina, 28203, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Children's Specialty Group, Pediatric Neurology
Norfolk, Virginia, 23510, United States
Osceola Medical Center
Osceola, Wisconsin, 54020, United States
Related Publications (1)
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.
PMID: 26573217DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Director
- Organization
- Sarepta Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Medical Director
Sarepta Therapeutics, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 23, 2012
First Posted
February 28, 2012
Study Start
February 27, 2012
Primary Completion
April 15, 2016
Study Completion
August 16, 2017
Last Updated
March 30, 2020
Results First Posted
July 10, 2019
Record last verified: 2020-03