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
Who is this study for? Child male patients with Duchenne Muscular Dystrophy
Status: Active_not_recruiting
Location: See all (73) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 6
Maximum Age: 13
Healthy Volunteers: f
View:
• 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
Locations
United States
Arizona
Neuromuscular Research Center
Phoenix
California
Children's Hospital Los Angeles
Los Angeles
David Geffen School of Medicine, UCLA
Los Angeles
Rady Children's Hospital San Diego/ UCSD
San Diego
Stanford University School of Medicine/Medical Center
Stanford
Florida
University of Florida
Gainesville
NW Florida Clinical Research Group, LLC
Gulf Breeze
Georgia
Center for Integrative Rare Disease Research (CIRDR)
Atlanta
Iowa
University of Iowa Children's Hospital
Iowa City
Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago
Kansas
University of Kansas, Medical Center
Kansas City
Massachusetts
Boston Children's Hospital
Boston
Missouri
St. Louis Children's Hospital
Saint Louis
Nevada
Las Vegas Clinic
Las Vegas
New York
University of Rochester Clinical Research Center
Rochester
Ohio
Cincinnati Children's Hospital Medical Center (CCHMC)
Cincinnati
Nationwide Children's Hospital
Columbus
Oregon
Shriners Hospital for Children
Portland
Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh
Texas
Children's Medical Center Dallas
Dallas
Utah
University of Utah
Salt Lake City
Virginia
Children's Hospital of the King's Daughters
Norfolk
Wisconsin
Children's Hospital of Wisconsin
Milwaukee
Other Locations
Argentina
DOM Centro de Reumatologia
Ciudad Autonoma De Buenos Aires
Australia
Royal Children's Hospital Melbourne
Parkville
Queensland Children's Hospital
South Brisbane
Children's Hospital at Westmead
Westmead
Belgium
Universitair Ziekenhuis Gent
Ghent
Universitair Ziekenhuis Leuven
Leuven
CHR de la Citadelle
Liège
Bulgaria
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia
Canada
Alberta Childrens Hospital
Calgary
London Health Sciences Centre
London
Children's Hospital of Eastern Ontario
Ottawa
Children's and Women's Health Centre of British Columbia
Vancouver
Denmark
Rigshospitalet Copenhagen University Hospital
København Ø
France
Reference Centre for Neuromuscular Diseases
Nantes
Hôpital Armand Trousseau
Paris
Hôpital Des Enfants
Toulouse
Germany
Charité - Universitätsmedizin Berlin
Berlin
Universitätsklinikum Essen
Essen
University Hospital Freiburg
Freiburg
Greece
IASO Children's Hospital
Maroussi
Ippokratio General Hospital of Thessaloniki
Thessaloniki
Hungary
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
Budapest
India
Royal Instituite of Child Neurosciences
Ahmedabad
Deenanth Mangeshkar Hospital
Pune
Ireland
The Children's University Hospital
Dublin
Israel
Schneider Children's Medical Center of Israel
Petah Tikvah
Italy
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Ferrara
Istituto Giannina Gaslini
Genoa
Az Ospedaliera Universitaria Policlinico G Martino
Messina
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano
Policlinico Universitario A Gemelli
Rome
Mexico
Neurociencias Estudios Clínicos S.C
Culiacán
Instituto de Investigaciones Clínicas para la Salud A.C
Durango
Poland
Uniwersyteckie Centrum Kliniczne
Gdansk
Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw
Republic of Korea
Seoul National University Hospital
Seoul
Pusan National University Yangsan Hospital
Yangsan
Russian Federation
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
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
Yekaterinburg
Serbia
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade
Spain
Hospital de La Santa Creu i Sant Pau
Barcelona
Hospital Sant Joan de Deu
Barcelona
Hospital Universitari i Politecnic La Fe de Valencia
Valencia
Sweden
Drottning Silvias Barn Och Ungdomssjukhus
Göteborg
United Kingdom
Royal Hospital for Children (Glasgow)
Glasgow
Leeds Teaching Hospitals NHS Trust
Leeds
Alder Hey Childrens Hospital
Liverpool
Great Ormond Street Hospital (GOSH)
London
Royal Victoria Infirmary
Newcastle Upon Tyne
John Radcliffe Hospital
Oxford
Time Frame
Start Date: 2016-09-28
Completion Date: 2025-10-17
Participants
Target number of participants: 228
Treatments
Experimental: 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).
Experimental: 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).
Placebo_comparator: 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).
Authors
Ann M Connolly, Katherine Mathews, Hoda Z Abdel-Hamid, Kumaraswamy Sivakumar, Jonathan McKinnon, Susan Iannaccone, Barry Byrne, John Day, Leigh Maria Ramos-Platt, Russell Butterfield, Emma Ciafaloni, Jerry R Mendell, Basil Darras, Carla Grosmann, Gihan Tennekoon, Erika Finanger, Gyula Acsadi, Perry Shieh, Chet Villa, Crystal Proud, Matthew Harmelink, Han Phan, Ulrike Schara, Laurent Servais, Janbernd Kirschner, Yann Péréon, Volker Straub, Wolfgang Müller-Felber, Claude Cances, Claudio Bruno, Craig McDonald, David Schorling, Giuseppe Vita, Lenka Jurikova, Yoram Nevo, Antigone Papavasiliou, Jana Haberlová, Dimitrios Zafeiriou, Anna Kostera-Pruszczyk, Vedrana Milic Rasic, Anita Cairns, Ian Woodcock, Riccardo Masson, Jean Mah, Mar Tulinius, Nathalie Goemans, Craig Campbell, Michelle Lorentzos, Christina Hoi-Hansen, Maria Judith Molnar, Nuria Muelas, Sithara Ramdas, Andres Nascimento, Hanns Lochmuller, Dmitry Vlodavets, Francesco Muntoni, Maria Mazurkiewicz-Beldzinska, Nicolas Deconinck, Stefan Spinty, Monique Ryan, Liesbeth De Waele, Iain Horrocks, Eugenio Mercuri, Ulrike Schara-Schmidt, Kathryn Selby
Related Therapeutic Areas
Sponsors
Leads: Sarepta Therapeutics, Inc.