New drug trial aims to slow muscle decline in genetic disorder
NCT ID NCT05291091
Summary
This study is testing whether an investigational drug called sevasemten (EDG-5506) is safe and can help control the progression of Becker muscular dystrophy. It involves about 244 adults and adolescents with the condition, who will receive either the drug or a placebo for 12 to 18 months. Researchers will measure changes in muscle function, strength, and blood markers to see if the treatment is effective.
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
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Locations
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AP-HP Hopital Pitie-Salpetriere
Paris, 75013, France
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Azienda Ospedale - Università Padova
Padua, 35128, Italy
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CHU de Nantes
Nantes, 44093, France
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CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, 06001, France
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Centre Hospitalier Régional de la Citadelle
Liège, 4000, Belgium
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Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, 13005, France
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Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122, Italy
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Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, 00168, Italy
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Hadassah University Hospital
Jerusalem, 91240, Israel
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Hospital Universitari de Bellvitge
Barcelona, 08907, Spain
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Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
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Hospital Universitario Donostia
Donostia / San Sebastian, 20014, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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Indiana University School of Medicine
Indianapolis, Indiana, 46202, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, 80336, Germany
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Leids Universitair Medisch Centrum
Leiden, 2333 ZA, Netherlands
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National Neuromuscular Research Institute
Austin, Texas, 78759, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Neurology Rare Disease Center
Denton, Texas, 76208, United States
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Newcastle Freeman Hospital
Newcastle, NE7 7DN, United Kingdom
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Northwestern University
Chicago, Illinois, 60611, United States
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Optimal Clinical Trials
Auckland, 1010, New Zealand
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Rare Disease Research, LLC NC
Hillsborough, North Carolina, 27278, United States
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Rigshospitalet
Copenhagen, Denmark
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Salford Royal Hospital
Salford, M68HD, United Kingdom
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Schneider Children's Hospital of Israel
Petah Tikva, 49202, Israel
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St Vincent's Hospital Melbourne
Fitzroy, VIC, 3065, Australia
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St. George's University Hospitals NHS Foundation Trust
London, SW17 0QT, United Kingdom
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Stanford Neuroscience Health Center
Palo Alto, California, 94304, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UC Denver
Aurora, Colorado, 80045, United States
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UC Irvine Medical Center
Orange, California, 92868, United States
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UC San Diego
La Jolla, California, 92037, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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Universitaire Ziekenhuizen Leuven
Leuven, Belgium
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University College London Hospital
London, NW1 2PG, United Kingdom
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University Hospital Gent
Ghent, 9000, Belgium
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University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, 45219, United States
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University of Florida
Gainesville, Florida, 32611, United States
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University of Iowa
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 Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01605, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University Health
Richmond, Virginia, 23298, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.