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Trial record 1 of 1 for:    NCT02420379
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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Sarepta Therapeutics Identifier:
First received: April 10, 2015
Last updated: May 31, 2017
Last verified: May 2017
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 Intervention Phase
Duchenne Muscular Dystrophy (DMD) Drug: eteplirsen Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by Sarepta Therapeutics:

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

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

Estimated Enrollment: 40
Study Start Date: May 2015
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.


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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT02420379

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
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
Study Chair: Catherine Stehman-Breen, MD Sarepta Therapeutics, Inc.
  More Information

Responsible Party: Sarepta Therapeutics Identifier: NCT02420379     History of Changes
Other Study ID Numbers: 4658-203
Study First Received: April 10, 2015
Last Updated: May 31, 2017

Keywords provided by Sarepta Therapeutics:
Duchenne muscular dystrophy
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 processed this record on August 18, 2017