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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.
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

Full criteria on ClinicalTrials.gov

Sites (70)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
DOM Centro de Reumatología
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Buenos AiresArgentinaUnknown status
Royal Children's Hospital Melbourne
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ParkvilleAustraliaUnknown status
Queensland Children's Hospital
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South BrisbaneAustraliaUnknown status
Children's Hospital at Westmead
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WestmeadAustraliaUnknown status
Universitair Ziekenhuis Gent
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GentBelgiumCompleted
UZ Leuven
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LeuvenBelgiumCompleted
Alexandrovska University Hospital
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SofiaBulgariaCompleted
Alberta Childrens Hospital
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CalgaryCanadaUnknown status
London Health Sciences Centre
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LondonCanadaUnknown status
Children'S Hospital Of Eastern Ontario
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OttawaCanadaUnknown status
Children's and Women's Health Centre of British Columbia
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VancouverCanadaUnknown status
Fakultni nemocnice v Motole
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PragueCzechiaCompleted
Charité Universitätsmedizin Berlin
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BerlinGermanyUnknown status
Universitätsklinikum Essen
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EssenGermanyUnknown status
University Hospital Freiburg
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Freiburg im BreisgauGermanyUnknown status
LMU Klinikum der Universität
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MünchenGermanyUnknown status
Rigshospitalet
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Copenhagen OeDenmarkCompleted
Hospital Sant Joan De Deu Barcelona
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Esplugues de LlobregatSpainCompleted
Hospital Universitario Y Politecnico La Fe
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ValenciaSpainCompleted
Reference Centre for Neuromuscular Diseases
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NantesFranceUnknown status
Hôpital Armand-Trousseau
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ParisFranceUnknown status
Hôpital des Enfants
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ToulouseFranceUnknown status
Royal Hospital for Children Glasgow
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GlasgowUnited KingdomUnknown status
Leeds Teaching Hospitals NHS Trust
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LeedsUnited KingdomUnknown status
Alder Hey Childrens Hospital
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LiverpoolUnited KingdomUnknown status
Great Ormond Street Hospital (GOSH)
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LondonUnited KingdomUnknown status
Royal Victoria Infirmary
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Newcastle upon TyneUnited KingdomUnknown status
John Radcliffe Hospital
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OxfordUnited KingdomUnknown status
Iaso Children's Hospital
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MarousiGreeceUnknown status
Ippokratio General Hospital of Thessaloniki
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ThessalonikiGreeceUnknown status
Semmelweis University
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Budapest VIIIHungaryCompleted
Children's Health Ireland
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Dublin 1IrelandCompleted
Schneider Children's Medical Center of Israel
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Petah TikvahIsraelUnknown status
Royal Instituite of Child Neurosciences
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AhmedabadIndiaUnknown status
Deenanth Mangeshkar Hospital
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PuneIndiaUnknown status
IRCCS Istituto Giannina Gaslini
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GenoaItalyCompleted
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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RomeItalyCompleted
Seoul National University Hospital
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SeoulSouth KoreaUnknown status
Pusan National University Yangsan Hospital
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YangsanSouth KoreaUnknown status
Neurociencias Estudios Clinicos S.C.
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CuliacánMexicoUnknown status
Instituto de Investigaciones Clinicas para la Salud A.C
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DurangoMexicoUnknown status
Uniwersyteckie Centrum Kliniczne
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GdanskPolandCompleted
Samodzielny Publiczny Szpital Kliniczny Im. Prof. W. Orlowskiego CMKP
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WarsawPolandCompleted
Clinic for Neurology and Psychiatry for Children and Youth
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BelgradeSerbiaUnknown 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
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MoscowRussiaUnknown status
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
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YekaterinburgRussiaUnknown status
Drottning Silvias barn- och ungdomssjukhus
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GothenburgSwedenUnknown status
Center for Integrative Rare Disease Research (CIRDR)
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AtlantaUnited StatesUnknown status
Boston Children's Hospital
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BostonUnited StatesUnknown status
Ann and Robert H. Lurie Children's Hospital of Chicago
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ChicagoUnited StatesUnknown status
Cincinnati Children's Hospital Medical Center (CCHMC)
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CincinnatiUnited StatesUnknown status
Nationwide Children's Hospital
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ColumbusUnited StatesUnknown status
Children's Medical Center Dallas
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DallasUnited StatesUnknown status
University of Florida
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GainesvilleUnited StatesUnknown status
NW Florida Clinical Research Group, LLC
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Gulf BreezeUnited StatesUnknown status
University of Iowa Children's Hospital
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Iowa CityUnited StatesUnknown status
University of Kansas Medical Center
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Kansas CityUnited StatesUnknown status
Las Vegas Clinic
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Las VegasUnited StatesUnknown status
Children's Hospital Los Angeles
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Los AngelesUnited StatesUnknown status
David Geffen School of Medicine, UCLA
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Los AngelesUnited StatesUnknown status
Children's Hospital of Wisconsin
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MilwaukeeUnited StatesUnknown status
Children's Hospital of The King's Daughters
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NorfolkUnited StatesUnknown status
Neuromuscular Research Center
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PhoenixUnited StatesUnknown status
Children's Hospital of Pittsburgh of UPMC
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PittsburghUnited StatesUnknown status
Shriners Hospital for Children
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PortlandUnited StatesUnknown status
University of Rochester Clinical Research Center
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RochesterUnited StatesUnknown status
University of Utah
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Salt Lake CityUnited StatesUnknown status
Rady Children's Hospital San Diego/ UCSD
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San DiegoUnited StatesUnknown status
Stanford University School of Medicine/Medical Center
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StanfordUnited StatesUnknown status
St. Louis Children's Hospital
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St LouisUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.