--- title: Study of Eteplirsen in DMD Patients nct_id: NCT02255552 overall_status: COMPLETED phase: PHASE3 sponsor: Sarepta Therapeutics, Inc. study_type: INTERVENTIONAL primary_condition: Duchenne Muscular Dystrophy (DMD) countries: United States canonical_url: "https://parkinsonspathways.com/agent/trials/NCT02255552.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT02255552" ct_last_update_post_date: 2021-01-25 last_seen_at: "2026-05-12T06:42:51.485Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # Study of Eteplirsen in DMD Patients **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 **NCT ID:** [NCT02255552](https://clinicaltrials.gov/study/NCT02255552) ## Key Facts - **Status:** COMPLETED - **Phase:** PHASE3 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 109 - **Lead Sponsor:** Sarepta Therapeutics, Inc. - **Conditions:** Duchenne Muscular Dystrophy (DMD) - **Start Date:** 2014-11-17 - **Completion Date:** 2019-06-14 - **CT.gov Last Update:** 2021-01-25 ## Brief Summary 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). ## Detailed Description This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Following primary efficacy endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen. Patients in the treated group 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). Patients in the untreated group will not receive treatment. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy. Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients. ## Eligibility - **Minimum age:** 7 Years - **Maximum age:** 16 Years - **Sex:** MALE - **Healthy Volunteers:** No ``` Inclusion Criteria: * Male 7-16 years old * Diagnosed with DMD, genotypically confirmed * Stable dose of corticosteroids for at least 24 weeks * Have intact right and left alternative upper muscle groups * Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters) * Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50% Exclusion Criteria: * Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months * Participation in any other DMD interventional clinical study within 12 weeks * Major surgery within 3 months * Presence of other clinically significant illness * Major change in the physical therapy regime within 3 months Other inclusion/exclusion criteria apply. ``` ## Arms - **Treated Group** (EXPERIMENTAL) — Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping will receive 30 mg/kg of eteplirsen weekly for 96 weeks, followed by a safety extension (not to exceed 48 weeks). - **Untreated Group** (NO_INTERVENTION) — Approximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen. ## Interventions - **eteplirsen** (DRUG) — Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks). ## Primary Outcomes - **Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96** _(time frame: Baseline, Week 96)_ — 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported. ## Secondary Outcomes - **Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96** _(time frame: Baseline, Week 96)_ - **Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96** _(time frame: Week 96)_ - **Number of Participants Who Lost Ambulation (LOA) by Week 96** _(time frame: Up to Week 96)_ - **Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96** _(time frame: Baseline, Week 96)_ - **Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96** _(time frame: Baseline, Week 96)_ - **Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96** _(time frame: Baseline, Week 96)_ ## Locations (37) - Neuromuscular Research Center, Phoenix, Arizona, United States - David Geffen School of Medicine at UCLA, Los Angeles, California, United States - University of California, Davis Medical Center, Sacramento, California, United States - Rady Children's Hospital, U.C. San Diego, San Diego, California, United States - Stanford University School of Medicine/Medical Center, Stanford, California, United States - Children's Hospital Colorado, Aurora, Colorado, United States - Connecticut Children's Medical Center, Hartford, Connecticut, United States - Children's National Health System, Washington D.C., District of Columbia, United States - The University of Florida, Powell Gene Therapy Center, Gainesville, Florida, United States - NW FL Clinical Research Group, LLC, Gulf Breeze, Florida, United States - Nemours Children's Hospital, Orlando, Florida, United States - Rare Disease Research Center, Atlanta, Georgia, United States - Emory University, Atlanta, Georgia, United States - Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States - University of Iowa Children's Hospital, Iowa City, Iowa, United States - University of Kansas Medical Center, Kansas City, Kansas, United States - Kennedy Krieger Institute, Baltimore, Maryland, United States - Massachusetts General Hospital, Boston, Massachusetts, United States - Children's Hospital Boston, Boston, Massachusetts, United States - Children's Hospital of Michigan, Detroit, Michigan, United States - University of Minnesota, Minneapolis, Minnesota, United States - St. Louis Children's Hospital, St Louis, Missouri, United States - Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire, United States - Columbia University Medical Center, New York, New York, United States - University of Rochester Clinical Research Center, Rochester, New York, United States - Levine Childrens Hospital, Carolinas Medical Center, Charlotte, North Carolina, United States - Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, Ohio, United States - Nationwide Children's Hospital, Columbus, Ohio, United States - Shriners Hospital for Children, Portland, Oregon, United States - Penn State Hershey Medical Center, Hershey, Pennsylvania, United States - Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States - Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States - Vanderbilt University Medical Center, Nashville, Tennessee, United States - The University of Texas Southwestern Medical Center, Dallas, Texas, United States - Texas Children's Hospital, Houston, Texas, United States - University of Utah, Salt Lake City, Utah, United States - Seattle Children's Hospital, Seattle, Washington, United States ## Recent Field Changes (last 30 days) - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.maxAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.neuromuscular research center|phoenix|arizona|united states` — added _(2026-05-12)_ - `locations.david geffen school of medicine at ucla|los angeles|california|united states` — added _(2026-05-12)_ - `locations.university of california, davis medical center|sacramento|california|united states` — added _(2026-05-12)_ - `locations.rady children's hospital, u.c. san diego|san diego|california|united states` — added _(2026-05-12)_ - `locations.stanford university school of medicine/medical center|stanford|california|united states` — added _(2026-05-12)_ - `locations.children's hospital colorado|aurora|colorado|united states` — added _(2026-05-12)_ - `locations.connecticut children's medical center|hartford|connecticut|united states` — added _(2026-05-12)_ - `locations.children's national health system|washington d.c.|district of columbia|united states` — added _(2026-05-12)_ - `locations.the university of florida, powell gene therapy center|gainesville|florida|united states` — added _(2026-05-12)_ - `locations.nw fl clinical research group, llc|gulf breeze|florida|united states` — added _(2026-05-12)_ - `locations.nemours children's hospital|orlando|florida|united states` — added _(2026-05-12)_ - `locations.rare disease research center|atlanta|georgia|united states` — added _(2026-05-12)_ - `locations.emory university|atlanta|georgia|united states` — added _(2026-05-12)_ - `locations.ann and robert h. lurie children's hospital of chicago|chicago|illinois|united states` — added _(2026-05-12)_ - `locations.university of iowa children's hospital|iowa city|iowa|united states` — added _(2026-05-12)_ - `locations.university of kansas medical center|kansas city|kansas|united states` — added _(2026-05-12)_ - `locations.kennedy krieger institute|baltimore|maryland|united states` — added _(2026-05-12)_ - `locations.massachusetts general hospital|boston|massachusetts|united states` — added _(2026-05-12)_ - `locations.children's hospital boston|boston|massachusetts|united states` — added _(2026-05-12)_ - `locations.children's hospital of michigan|detroit|michigan|united states` — added _(2026-05-12)_ - `locations.university of minnesota|minneapolis|minnesota|united states` — added _(2026-05-12)_ - `locations.st. louis children's hospital|st louis|missouri|united states` — added _(2026-05-12)_ - `locations.dartmouth-hitchcock medical center|lebanon|new hampshire|united states` — added _(2026-05-12)_ - `locations.columbia university medical center|new york|new york|united states` — added _(2026-05-12)_ - `locations.university of rochester clinical research center|rochester|new york|united states` — added _(2026-05-12)_ - `locations.levine childrens hospital, carolinas medical center|charlotte|north carolina|united states` — added _(2026-05-12)_ - `locations.cincinnati children's hospital medical center (cchmc)|cincinnati|ohio|united states` — added _(2026-05-12)_ - `locations.nationwide children's hospital|columbus|ohio|united states` — added _(2026-05-12)_ - `locations.shriners hospital for children|portland|oregon|united states` — added _(2026-05-12)_ - `locations.penn state hershey medical center|hershey|pennsylvania|united states` — added _(2026-05-12)_ - `locations.children's hospital of philadelphia|philadelphia|pennsylvania|united states` — added _(2026-05-12)_ - `locations.children's hospital of pittsburgh of upmc|pittsburgh|pennsylvania|united states` — added _(2026-05-12)_ - `locations.vanderbilt university medical center|nashville|tennessee|united states` — added _(2026-05-12)_ - `locations.the university of texas southwestern medical center|dallas|texas|united states` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT02255552.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT02255552* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*