New hope for boys with muscular dystrophy as major drug trial completes
NCT ID NCT02500381
Summary
This large, completed study tested whether two drugs, casimersen and golodirsen, could help boys with a specific genetic form of Duchenne muscular dystrophy. For the first 96 weeks, some participants received the drug while others received a placebo, followed by a period where everyone received the active drug. The main goal was to see if the drugs could slow the decline in muscle function, measured by tests like walking speed and distance.
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Contacts and locations
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Locations
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Alberta Childrens Hospital
Calgary, Alberta, T3B 6A8, Canada
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Alder Hey Childrens Hospital
Liverpool, L12 2AP, United Kingdom
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Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Az Ospedaliera Universitaria Policlinico G Martino
Messina, 98125, Italy
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Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Ferrara, 44124, Italy
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHR de la Citadelle
Liège, 4000, Belgium
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Center for Integrative Rare Disease Research (CIRDR)
Atlanta, Georgia, 30318, United States
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Charité - Universitätsmedizin Berlin
Berlin, 13353, Germany
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital at Westmead
Westmead, 2145, Australia
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Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
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Children's Medical Center Dallas
Dallas, Texas, 75235, United States
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Children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, V6H 3V4, Canada
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Cincinnati Children's Hospital Medical Center (CCHMC)
Cincinnati, Ohio, 45229, United States
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Clinic for Neurology and Psychiatry for Children and Youth
Belgrade, 11000, Serbia
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DOM Centro de Reumatologia
Buenos Aires, 1111, Argentina
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David Geffen School of Medicine, UCLA
Los Angeles, California, 90095, United States
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Deenanth Mangeshkar Hospital
Pune, Maharashtra, 411004, India
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Drottning Silvias Barn Och Ungdomssjukhus
Gothenburg, SE-41685, Sweden
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Fakultni nemocnice v Motole
Prague, 15008, Czechia
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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, 125412, Russia
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
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Great Ormond Street Hospital (GOSH)
London, WC1N 1EH, United Kingdom
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Hospital Sant Joan de Deu
Barcelona, 08950, Spain
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Hospital Universitari i Politecnic La Fe de Valencia
Valencia, Spain
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Hospital de La Santa Creu i Sant Pau
Barcelona, 08041, Spain
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Hôpital Armand Trousseau
Paris, 75012, France
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Hôpital Des Enfants
Toulouse, Haute-Garonne, 31059, France
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IASO Children's Hospital
Marousi, 15123, Greece
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Instituto de Investigaciones Clínicas para la Salud A.C
Durango, 34000, Mexico
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Ippokratio General Hospital of Thessaloniki
Thessaloniki, 54642, Greece
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Istituto Giannina Gaslini
Genoa, 16147, Italy
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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LMU Klinikum der Universität
München, Bavaria, 80337, Germany
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Las Vegas Clinic
Las Vegas, Nevada, 89145, United States
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Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX, United Kingdom
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London Health Sciences Centre
London, Ontario, N6A 5W9, Canada
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NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Neurociencias Estudios Clínicos S.C
Culiacán, 80020, Mexico
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Neuromuscular Research Center
Phoenix, Arizona, 85028, United States
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Policlinico Universitario A Gemelli
Rome, 00168, Italy
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Pusan National University Yangsan Hospital
Yangsan, 50612, South Korea
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Queensland Children's Hospital
South Brisbane, 4101, Australia
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Rady Children's Hospital San Diego/ UCSD
San Diego, California, 92123, United States
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Reference Centre for Neuromuscular Diseases
Nantes, 44093, France
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Rigshospitalet Copenhagen University Hospital
København Ø, 2100, Denmark
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Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
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Royal Hospital for Children (Glasgow)
Glasgow, G51 4TF, United Kingdom
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Royal Instituite of Child Neurosciences
Ahmedabad, Gujarat, 380054, India
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Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP, United Kingdom
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Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw, Masovian Voivodeship, 02-097, Poland
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Schneider Children's Medical Center of Israel
Petah Tikvah, 49102, Israel
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Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
Budapest, 1083, Hungary
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Seoul National University Hospital
Seoul, 03080, South Korea
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Shriners Hospital for Children
Portland, Oregon, 97239, United States
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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Stanford University School of Medicine/Medical Center
Stanford, California, 94305, United States
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State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
Yekaterinburg, Russia
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The Children's University Hospital
Dublin, D1, Ireland
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Universitair Ziekenhuis Gent
Ghent, 9000, Belgium
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Universitair Ziekenhuis Leuven
Leuven, 3000, Belgium
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University Hospital Brno
Brno, 61300, Czechia
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University Hospital Freiburg
Freiburg im Breisgau, 79106, Germany
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University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Sofia-Grad, 1431, Bulgaria
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
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University of Kansas, Medical Center
Kansas City, Kansas, 66160, United States
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University of Rochester Clinical Research Center
Rochester, New York, 14642, United States
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University of Utah
Salt Lake City, Utah, 84132, United States
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Universitätsklinikum Essen
Essen, 45122, Germany
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Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952, Poland
Conditions
Explore the condition pages connected to this study.