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.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Alberta Childrens Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • Alder Hey Childrens Hospital

    Liverpool, L12 2AP, United Kingdom

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Az Ospedaliera Universitaria Policlinico G Martino

    Messina, 98125, Italy

  • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna

    Ferrara, 44124, Italy

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHR de la Citadelle

    Liège, 4000, Belgium

  • Center for Integrative Rare Disease Research (CIRDR)

    Atlanta, Georgia, 30318, United States

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353, Germany

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital at Westmead

    Westmead, 2145, Australia

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Children's and Women's Health Centre of British Columbia

    Vancouver, British Columbia, V6H 3V4, Canada

  • Cincinnati Children's Hospital Medical Center (CCHMC)

    Cincinnati, Ohio, 45229, United States

  • Clinic for Neurology and Psychiatry for Children and Youth

    Belgrade, 11000, Serbia

  • DOM Centro de Reumatologia

    Buenos Aires, 1111, Argentina

  • David Geffen School of Medicine, UCLA

    Los Angeles, California, 90095, United States

  • Deenanth Mangeshkar Hospital

    Pune, Maharashtra, 411004, India

  • Drottning Silvias Barn Och Ungdomssjukhus

    Gothenburg, SE-41685, Sweden

  • Fakultni nemocnice v Motole

    Prague, 15008, Czechia

  • 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

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Great Ormond Street Hospital (GOSH)

    London, WC1N 1EH, United Kingdom

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, Spain

  • Hospital de La Santa Creu i Sant Pau

    Barcelona, 08041, Spain

  • Hôpital Armand Trousseau

    Paris, 75012, France

  • Hôpital Des Enfants

    Toulouse, Haute-Garonne, 31059, France

  • IASO Children's Hospital

    Marousi, 15123, Greece

  • Instituto de Investigaciones Clínicas para la Salud A.C

    Durango, 34000, Mexico

  • Ippokratio General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • Istituto Giannina Gaslini

    Genoa, 16147, Italy

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • LMU Klinikum der Universität

    München, Bavaria, 80337, Germany

  • Las Vegas Clinic

    Las Vegas, Nevada, 89145, United States

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • London Health Sciences Centre

    London, Ontario, N6A 5W9, Canada

  • NW Florida Clinical Research Group, LLC

    Gulf Breeze, Florida, 32561, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Neurociencias Estudios Clínicos S.C

    Culiacán, 80020, Mexico

  • Neuromuscular Research Center

    Phoenix, Arizona, 85028, United States

  • Policlinico Universitario A Gemelli

    Rome, 00168, Italy

  • Pusan National University Yangsan Hospital

    Yangsan, 50612, South Korea

  • Queensland Children's Hospital

    South Brisbane, 4101, Australia

  • Rady Children's Hospital San Diego/ UCSD

    San Diego, California, 92123, United States

  • Reference Centre for Neuromuscular Diseases

    Nantes, 44093, France

  • Rigshospitalet Copenhagen University Hospital

    København Ø, 2100, Denmark

  • Royal Children's Hospital Melbourne

    Parkville, Victoria, 3052, Australia

  • Royal Hospital for Children (Glasgow)

    Glasgow, G51 4TF, United Kingdom

  • Royal Instituite of Child Neurosciences

    Ahmedabad, Gujarat, 380054, India

  • Royal Victoria Infirmary

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • Samodzielny Publiczny Centralny Szpital Kliniczny

    Warsaw, Masovian Voivodeship, 02-097, Poland

  • Schneider Children's Medical Center of Israel

    Petah Tikvah, 49102, Israel

  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

    Budapest, 1083, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Shriners Hospital for Children

    Portland, Oregon, 97239, United States

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Stanford University School of Medicine/Medical Center

    Stanford, California, 94305, United States

  • State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg

    Yekaterinburg, Russia

  • The Children's University Hospital

    Dublin, D1, Ireland

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • Universitair Ziekenhuis Leuven

    Leuven, 3000, Belgium

  • University Hospital Brno

    Brno, 61300, Czechia

  • University Hospital Freiburg

    Freiburg im Breisgau, 79106, Germany

  • University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

    Sofia, Sofia-Grad, 1431, Bulgaria

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa Children's Hospital

    Iowa City, Iowa, 52242, United States

  • University of Kansas, Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Rochester Clinical Research Center

    Rochester, New York, 14642, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Essen

    Essen, 45122, Germany

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952, Poland

Conditions

Explore the condition pages connected to this study.