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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

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. Identifier: NCT02420379
Recruitment Status : Completed
First Posted : April 17, 2015
Last Update Posted : February 26, 2019
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: eteplirsen Phase 2

Detailed Description:

Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
Actual Study Start Date : July 1, 2015
Actual Primary Completion Date : December 17, 2018
Actual Study Completion Date : December 17, 2018

Arm Intervention/treatment
Experimental: Open-Label
Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg .
Drug: eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.
Other Names:
  • AVI-4658
  • EXONDYS 51®

No Intervention: Control Group
Approximately 20 patients with DMD not amenable to exon 51 skipping will be observed for 96 weeks.

Primary Outcome Measures :
  1. Number of patients with treatment emergent adverse events [ Time Frame: 96 weeks ]

Secondary Outcome Measures :
  1. Change from baseline in percent of dystrophin-positive skeletal muscle fibers [ Time Frame: 96 weeks ]

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.

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Ages Eligible for Study:   4 Years to 6 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male 4-6 years of age.
  • 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:

  • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
  • 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.

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 identifier (NCT number): NCT02420379

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United States, Arizona
Neuromuscular Research Center of Arizona
Phoenix, Arizona, United States, 85028
United States, California
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
University of California, Davis Medical Center
Sacramento, California, United States, 95817
Stanford University Medical Center
Stanford, California, United States, 94305
United States, Florida
University of Florida, Shands Hospital
Gainesville, Florida, United States, 32610
United States, Georgia
Rare Disease Research Center
Atlanta, Georgia, United States, 30318
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30324
United States, Iowa
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
United States, Missouri
St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Shriners Hospital for Children
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc. Identifier: NCT02420379    
Other Study ID Numbers: 4658-203
First Posted: April 17, 2015    Key Record Dates
Last Update Posted: February 26, 2019
Last Verified: February 2019
Keywords provided by Sarepta Therapeutics, Inc.:
Duchenne muscular dystrophy
exon 51
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked