NCT02500381CompletedPhase 3
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Source: ClinicalTrials.gov · Last updated 18 Nov 2025
Brief summary (registry, in English)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 228 (actual)
- First submitted
- 14 Jul 2015
- Start date
- 28 Sept 2016
- Primary completion date
- 12 Nov 2024
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT02500381
- EU CT number
- 2024-514698-23-00
- EudraCT number
- 2015-002069-52
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 228
- From 28 Sept 2016 to the primary completion date, 12 Nov 2024
- 70 sites in 24 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment with gene therapy at any time
- Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1
- Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1
- Major surgery within 3 months prior to Week 1
Other illness
- Presence of other clinically significant illness
Other
- Other inclusion/exclusion criteria may apply.
Who the study is looking for
- Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
- Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- Intact right and left biceps brachii muscles or 2 alternative upper arm muscle groups
- Mean 6MWT ≥300 meters and ≤450 meters
- Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted
Age in the registry: 6 years to 13 years · Sex: Male · Does not accept healthy volunteers
Sites (70)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| DOM Centro de Reumatología Coordinator of this site? Confirm its status | Buenos Aires | Argentina | Unknown status |
| Royal Children's Hospital Melbourne Coordinator of this site? Confirm its status | Parkville | Australia | Unknown status |
| Queensland Children's Hospital Coordinator of this site? Confirm its status | South Brisbane | Australia | Unknown status |
| Children's Hospital at Westmead Coordinator of this site? Confirm its status | Westmead | Australia | Unknown status |
| Universitair Ziekenhuis Gent Coordinator of this site? Confirm its status | Gent | Belgium | Completed |
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Completed |
| Alexandrovska University Hospital Coordinator of this site? Confirm its status | Sofia | Bulgaria | Completed |
| Alberta Childrens Hospital Coordinator of this site? Confirm its status | Calgary | Canada | Unknown status |
| London Health Sciences Centre Coordinator of this site? Confirm its status | London | Canada | Unknown status |
| Children'S Hospital Of Eastern Ontario Coordinator of this site? Confirm its status | Ottawa | Canada | Unknown status |
| Children's and Women's Health Centre of British Columbia Coordinator of this site? Confirm its status | Vancouver | Canada | Unknown status |
| Fakultni nemocnice v Motole Coordinator of this site? Confirm its status | Prague | Czechia | Completed |
| Charité Universitätsmedizin Berlin Coordinator of this site? Confirm its status | Berlin | Germany | Unknown status |
| Universitätsklinikum Essen Coordinator of this site? Confirm its status | Essen | Germany | Unknown status |
| University Hospital Freiburg Coordinator of this site? Confirm its status | Freiburg im Breisgau | Germany | Unknown status |
| LMU Klinikum der Universität Coordinator of this site? Confirm its status | München | Germany | Unknown status |
| Rigshospitalet Coordinator of this site? Confirm its status | Copenhagen Oe | Denmark | Completed |
| Hospital Sant Joan De Deu Barcelona Coordinator of this site? Confirm its status | Esplugues de Llobregat | Spain | Completed |
| Hospital Universitario Y Politecnico La Fe Coordinator of this site? Confirm its status | Valencia | Spain | Completed |
| Reference Centre for Neuromuscular Diseases Coordinator of this site? Confirm its status | Nantes | France | Unknown status |
| Hôpital Armand-Trousseau Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| Hôpital des Enfants Coordinator of this site? Confirm its status | Toulouse | France | Unknown status |
| Royal Hospital for Children Glasgow Coordinator of this site? Confirm its status | Glasgow | United Kingdom | Unknown status |
| Leeds Teaching Hospitals NHS Trust Coordinator of this site? Confirm its status | Leeds | United Kingdom | Unknown status |
| Alder Hey Childrens Hospital Coordinator of this site? Confirm its status | Liverpool | United Kingdom | Unknown status |
| Great Ormond Street Hospital (GOSH) Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Royal Victoria Infirmary Coordinator of this site? Confirm its status | Newcastle upon Tyne | United Kingdom | Unknown status |
| John Radcliffe Hospital Coordinator of this site? Confirm its status | Oxford | United Kingdom | Unknown status |
| Iaso Children's Hospital Coordinator of this site? Confirm its status | Marousi | Greece | Unknown status |
| Ippokratio General Hospital of Thessaloniki Coordinator of this site? Confirm its status | Thessaloniki | Greece | Unknown status |
| Semmelweis University Coordinator of this site? Confirm its status | Budapest VIII | Hungary | Completed |
| Children's Health Ireland Coordinator of this site? Confirm its status | Dublin 1 | Ireland | Completed |
| Schneider Children's Medical Center of Israel Coordinator of this site? Confirm its status | Petah Tikvah | Israel | Unknown status |
| Royal Instituite of Child Neurosciences Coordinator of this site? Confirm its status | Ahmedabad | India | Unknown status |
| Deenanth Mangeshkar Hospital Coordinator of this site? Confirm its status | Pune | India | Unknown status |
| IRCCS Istituto Giannina Gaslini Coordinator of this site? Confirm its status | Genoa | Italy | Completed |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS Coordinator of this site? Confirm its status | Rome | Italy | Completed |
| Seoul National University Hospital Coordinator of this site? Confirm its status | Seoul | South Korea | Unknown status |
| Pusan National University Yangsan Hospital Coordinator of this site? Confirm its status | Yangsan | South Korea | Unknown status |
| Neurociencias Estudios Clinicos S.C. Coordinator of this site? Confirm its status | Culiacán | Mexico | Unknown status |
| Instituto de Investigaciones Clinicas para la Salud A.C Coordinator of this site? Confirm its status | Durango | Mexico | Unknown status |
| Uniwersyteckie Centrum Kliniczne Coordinator of this site? Confirm its status | Gdansk | Poland | Completed |
| Samodzielny Publiczny Szpital Kliniczny Im. Prof. W. Orlowskiego CMKP Coordinator of this site? Confirm its status | Warsaw | Poland | Completed |
| Clinic for Neurology and Psychiatry for Children and Youth Coordinator of this site? Confirm its status | Belgrade | Serbia | Unknown status |
| Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation Coordinator of this site? Confirm its status | Moscow | Russia | Unknown status |
| State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg Coordinator of this site? Confirm its status | Yekaterinburg | Russia | Unknown status |
| Drottning Silvias barn- och ungdomssjukhus Coordinator of this site? Confirm its status | Gothenburg | Sweden | Unknown status |
| Center for Integrative Rare Disease Research (CIRDR) Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Ann and Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center (CCHMC) Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
| Children's Medical Center Dallas Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| University of Florida Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| NW Florida Clinical Research Group, LLC Coordinator of this site? Confirm its status | Gulf Breeze | United States | Unknown status |
| University of Iowa Children's Hospital Coordinator of this site? Confirm its status | Iowa City | United States | Unknown status |
| University of Kansas Medical Center Coordinator of this site? Confirm its status | Kansas City | United States | Unknown status |
| Las Vegas Clinic Coordinator of this site? Confirm its status | Las Vegas | United States | Unknown status |
| Children's Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| David Geffen School of Medicine, UCLA Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Children's Hospital of Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| Children's Hospital of The King's Daughters Coordinator of this site? Confirm its status | Norfolk | United States | Unknown status |
| Neuromuscular Research Center Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| Children's Hospital of Pittsburgh of UPMC Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Shriners Hospital for Children Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| University of Rochester Clinical Research Center Coordinator of this site? Confirm its status | Rochester | United States | Unknown status |
| University of Utah Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| Rady Children's Hospital San Diego/ UCSD Coordinator of this site? Confirm its status | San Diego | United States | Unknown status |
| Stanford University School of Medicine/Medical Center Coordinator of this site? Confirm its status | Stanford | United States | Unknown status |
| St. Louis Children's Hospital Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.