Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT02286947 |
Recruitment Status :
Completed
First Posted : November 10, 2014
Last Update Posted : May 11, 2018
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Condition or disease | Intervention/treatment | Phase |
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Muscular Dystrophy, Duchenne | Drug: Eteplirsen | Phase 2 |
This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.
Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 20 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy |
Study Start Date : | November 2014 |
Actual Primary Completion Date : | April 2017 |
Actual Study Completion Date : | March 2018 |

Arm | Intervention/treatment |
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Experimental: Eteplirsen 30 mg/kg
Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks.
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Drug: Eteplirsen
Eteplirsen solution for IV infusion
Other Names:
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- Incidence of Adverse Events [ Time Frame: Up to 144 Weeks ]
- Number of Participants with Clinical Laboratory Abnormalities (Reported as AEs) [ Time Frame: Up to 144 Weeks ]
- Number of Participants with Abnormalities in Vital Signs and ECG (Reported as AEs) [ Time Frame: Up to 144 Weeks ]
- Mean Change from Baseline in Maximum Inspiratory Pressure (MIP) % Predicted [ Time Frame: Baseline to Week 96 ]
- Mean Change from Baseline in Maximum Expiratory Pressure (MEP) % Predicted [ Time Frame: Baseline to Week 96 ]

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Ages Eligible for Study: | 7 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | Male |
Gender Based Eligibility: | Yes |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male 7 - 21 years of age
- Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
- Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
- Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
- Score of ≤4 on the Brooke Score for Arms and Shoulders.
- Stable cardiac and pulmonary function
- Use of contraceptives for sexually active males throughout the study
- Willing to provide consent and comply with the study
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 treatment with SMT C1100/BMN 195 at any time.
- Previous treatment with drisapersen (PRO051) within the last 6 months.
- Participation in any other DMD interventional clinical study within 12 weeks
- Major change in physiotherapy regimen within the past 3 months
- Major surgery within 3 months
- Presence of other clinically significant illness
- Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
- Forced vital capacity % predicted [FVC % predicted] <40%, or requiring daytime ventilation.
- Require antiarrhythmic and/or antidiuretic therapy for heart failure.
- Have a left ventricular ejection fraction (LVEF) of <40%.
- Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment 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): NCT02286947
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 | |
United States, Iowa | |
University of Iowa Children's Hospital | |
Iowa City, Iowa, United States, 52242 | |
United States, Maryland | |
Kennedy Krieger Institute | |
Baltimore, Maryland, United States, 21205 | |
United States, Massachusetts | |
Massachusetts General Hospital | |
Boston, Massachusetts, United States, 02114 | |
United States, Missouri | |
St. Louis Children's Hospital | |
Saint Louis, Missouri, United States, 63110 | |
United States, New York | |
University of Rochester Medical Center | |
Rochester, New York, United States, 14642 | |
United States, Ohio | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 | |
United States, Washington | |
Seattle Children's Hospital | |
Seattle, Washington, United States, 98105 |
Study Director: | Chris Mix, MD | Sarepta Therapeutics |
Responsible Party: | Sarepta Therapeutics |
ClinicalTrials.gov Identifier: | NCT02286947 History of Changes |
Other Study ID Numbers: |
4658-204 |
First Posted: | November 10, 2014 Key Record Dates |
Last Update Posted: | May 11, 2018 |
Last Verified: | May 2018 |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Sarepta Therapeutics:
DMD exon 51 dystrophin |
dystrophy eteplirsen Duchenne |
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 |