Study of Eteplirsen in DMD Patients
Public ClinicalTrials.gov record NCT02255552. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Study identification
- NCT ID
- NCT02255552
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 109 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 7 Years to 16 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 17, 2014 (Actual)
- Primary completion
- Jun 14, 2019 (Actual)
- Completion
- Jun 14, 2019 (Actual)
- Last update posted
- Jan 25, 2021
2014 – 2019
United States locations
- U.S. sites
- 37
- U.S. states
- 25
- U.S. cities
- 35
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Neuromuscular Research Center | Phoenix | Arizona | 85028 | — |
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90095 | — |
| University of California, Davis Medical Center | Sacramento | California | 95817 | — |
| Rady Children's Hospital, U.C. San Diego | San Diego | California | 92130 | — |
| Stanford University School of Medicine/Medical Center | Stanford | California | 94305 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | — |
| Children's National Health System | Washington D.C. | District of Columbia | 20010 | — |
| The University of Florida, Powell Gene Therapy Center | Gainesville | Florida | 32610 | — |
| NW FL Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Rare Disease Research Center | Atlanta | Georgia | 30318 | — |
| Emory University | Atlanta | Georgia | 30324 | — |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Dartmouth-Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| University of Rochester Clinical Research Center | Rochester | New York | 14642 | — |
| Levine Childrens Hospital, Carolinas Medical Center | Charlotte | North Carolina | 28207 | — |
| Cincinnati Children's Hospital Medical Center (CCHMC) | Cincinnati | Ohio | 45229 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Shriners Hospital for Children | Portland | Oregon | 97239 | — |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02255552, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 25, 2021 · Synced Oct 24, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02255552 live on ClinicalTrials.gov.