We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01540409
First Posted: February 28, 2012
Last Update Posted: November 25, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sarepta Therapeutics
  Purpose
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.

Condition Intervention Phase
Duchenne Muscular Dystrophy (DMD) Drug: AVI-4658 (Eteplirsen) Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • Percent of dystrophin positive fibers [ Time Frame: Baseline to Week 20 ]
    The primary biological efficacy endpoint will be the change from baseline at Week 20 in the percent of dystrophin positive fibers (type = anti-dystrophin antibody MANDYS106) in muscle biopsy tissue as measured by immunohistochemistry (IHC).

  • 6 Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 212 ]
    The primary functional efficacy endpoint will be the change from baseline to Week 212 on the 6 Minute Walk Test (6MWT).


Other Outcome Measures:
  • Muscle Biopsy Tests [ Time Frame: Baseline to Week 20, Week 140 (optional) ]
    • Dystrophin intensity per fiber in muscle biopsy tissue as determined by IHC
    • CD3, CD4, and CD8 lymphocyte count in muscle biopsy tissue
    • Total dystrophin protein in muscle biopsy tissue as determined by Western blot analysis
    • Exon skipping in muscle biopsy tissue as assessed by reverse transcriptase polymerase chain reaction (RT PCR).

  • Timed 4 Step Test results [ Time Frame: Baseline to Week 212 ]
  • North Star Ambulatory Assessment (NSAA) results [ Time Frame: Baseline to Week 212 ]
  • Maximum voluntary isometric contraction test (MVICT) results [ Time Frame: Baseline to Week 212 ]
  • 9 Hole Peg Test results [ Time Frame: Baseline to Week 212 ]
  • Pediatric Quality of Life Inventory (PedsQL) results, including the neuromuscular module (NMM) [ Time Frame: Baseline to Week 212 ]
  • Pulmonary function test results [ Time Frame: Baseline to Week 212 ]
    including forced vital capacity (FVC), percent predicted FVC, forced expiratory volume in 1 second (FEV1), FEV1%, FEV1/FVC ratio, maximum inspiratory pressure (MIP), and maximum expiratory pressure (MEP).


Enrollment: 12
Study Start Date: February 2012
Estimated Study Completion Date: February 2017
Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

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
Criteria

Inclusion Criteria:

A subject must meet all of the following criteria to be eligible for this study.

  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:

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.

  Contacts and Locations
Information from the National Library of Medicine

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


Locations
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
St. 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
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Principal Investigator: Jerry R Mendell, MD Nationwide Children's Hospital
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT01540409     History of Changes
Other Study ID Numbers: 4658-us-202
First Submitted: February 23, 2012
First Posted: February 28, 2012
Last Update Posted: November 25, 2016
Last Verified: November 2016

Keywords provided by Sarepta Therapeutics:
DMD, Duchenne, Eteplirsen, dystrophy, dystrophin, exon 51

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked