Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT02500381
Last Updated: 2025-11-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
228 participants
INTERVENTIONAL
2016-09-28
2025-10-16
Brief Summary
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Detailed Description
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The study will enroll approximately 222 participants. Twice as many participants will be randomized to receive active treatment as will receive placebo (2:1 randomization).
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests, such as the 6-minute walk test (6MWT). All participants will undergo a muscle biopsy at baseline and a second muscle biopsy either at Week 48 or Week 96.
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of both drugs.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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SRP-4045
Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
SRP-4045
SRP-4045 solution for IV infusion
SRP-4053
Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
SRP-4053
SRP-4053 solution for IV infusion
Placebo followed by SRP-4045 or SRP-4053
Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
SRP-4045
SRP-4045 solution for IV infusion
SRP-4053
SRP-4053 solution for IV infusion
Placebo
SRP-4045 or SRP-4053 placebo-matching solution for IV infusion
Interventions
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SRP-4045
SRP-4045 solution for IV infusion
SRP-4053
SRP-4053 solution for IV infusion
Placebo
SRP-4045 or SRP-4053 placebo-matching solution for IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
* Presence of other clinically significant illness
6 Years
13 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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Neuromuscular Research Center
Phoenix, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
David Geffen School of Medicine, UCLA
Los Angeles, California, United States
Rady Children's Hospital San Diego/ UCSD
San Diego, California, United States
Stanford University School of Medicine/Medical Center
Stanford, California, United States
University of Florida
Gainesville, Florida, United States
NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Center for Integrative Rare Disease Research (CIRDR)
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
Boston Children's Hospital
Boston, Massachusetts, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Las Vegas Clinic
Las Vegas, Nevada, United States
University of Rochester Clinical Research Center
Rochester, New York, 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
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Children's Medical Center Dallas
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
DOM Centro de Reumatologia
Buenos Aires, , Argentina
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
Queensland Children's Hospital
South Brisbane, , Australia
Children's Hospital at Westmead
Westmead, , Australia
Universitair Ziekenhuis Gent
Ghent, , Belgium
Universitair Ziekenhuis Leuven
Leuven, , Belgium
CHR de la Citadelle
Liège, , Belgium
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Sofia-Grad, Bulgaria
Alberta Childrens Hospital
Calgary, Alberta, Canada
Children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, Canada
London Health Sciences Centre
London, Ontario, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
University Hospital Brno
Brno, , Czechia
Fakultni nemocnice v Motole
Prague, , Czechia
Rigshospitalet Copenhagen University Hospital
København Ø, , Denmark
Hôpital Des Enfants
Toulouse, Haute-Garonne, France
Reference Centre for Neuromuscular Diseases
Nantes, , France
Hôpital Armand Trousseau
Paris, , France
LMU Klinikum der Universität
München, Bavaria, Germany
Charité - Universitätsmedizin Berlin
Berlin, , Germany
Universitätsklinikum Essen
Essen, , Germany
University Hospital Freiburg
Freiburg im Breisgau, , Germany
IASO Children's Hospital
Marousi, , Greece
Ippokratio General Hospital of Thessaloniki
Thessaloniki, , Greece
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
Budapest, , Hungary
Royal Instituite of Child Neurosciences
Ahmedabad, Gujarat, India
Deenanth Mangeshkar Hospital
Pune, Maharashtra, India
The Children's University Hospital
Dublin, , Ireland
Schneider Children's Medical Center of Israel
Petah Tikvah, , Israel
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Ferrara, , Italy
Istituto Giannina Gaslini
Genoa, , Italy
Az Ospedaliera Universitaria Policlinico G Martino
Messina, , Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Policlinico Universitario A Gemelli
Rome, , Italy
Neurociencias Estudios Clínicos S.C
Culiacán, , Mexico
Instituto de Investigaciones Clínicas para la Salud A.C
Durango, , Mexico
Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw, Masovian Voivodeship, Poland
Uniwersyteckie Centrum Kliniczne
Gdansk, , Poland
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
Moscow, , Russia
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
Yekaterinburg, , Russia
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade, , Serbia
Seoul National University Hospital
Seoul, , South Korea
Pusan National University Yangsan Hospital
Yangsan, , South Korea
Hospital de La Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, , Spain
Drottning Silvias Barn Och Ungdomssjukhus
Gothenburg, , Sweden
Royal Hospital for Children (Glasgow)
Glasgow, , United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, , United Kingdom
Alder Hey Childrens Hospital
Liverpool, , United Kingdom
Great Ormond Street Hospital (GOSH)
London, , United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, , United Kingdom
John Radcliffe Hospital
Oxford, , United Kingdom
Countries
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References
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Vandekerckhove I, Hanssen B, Peeters N, Dewit T, De Beukelaer N, Van den Hauwe M, De Waele L, Van Campenhout A, De Groote F, Desloovere K. Anthropometric-related percentile curves for muscle size and strength of lower limb muscles of typically developing children. J Anat. 2025 Aug;247(2):348-362. doi: 10.1111/joa.14241. Epub 2025 Mar 17.
Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.
Other Identifiers
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4045-301
Identifier Type: -
Identifier Source: org_study_id
2015-002069-52
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-514698-23-00
Identifier Type: OTHER
Identifier Source: secondary_id
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