Two-Drug combo aims to build muscle, boost movement in SMA
NCT ID NCT05115110
Summary
This study is testing whether adding a new investigational drug called RO7204239 to the approved SMA drug risdiplam is safe and more effective. RO7204239 is designed to block a protein that limits muscle growth, potentially helping muscles get bigger and stronger. The trial will enroll about 259 children and young adults (ages 2-25) with SMA who can walk, to see if the combination improves their motor skills and muscle mass compared to risdiplam alone.
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Contacts and locations
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Locations
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Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162, Italy
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Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
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Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1, Canada
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CHULC, E.P.E. - Hospital Dona Estefania
Lisbon, 1169-045, Portugal
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Chr de La Citadelle
Liège, 4000, Belgium
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Clinical Hospital Centre Zagreb
Zagreb, 10000, Croatia
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Columbia University Medical Center
New York, New York, 10032, United States
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Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, 20133, Italy
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Great Ormond Street Hospital For Children
London, WC1N 3JH, United Kingdom
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Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
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Hospital Universitario La Paz
Madrid, 28046, Spain
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Hospital Universitario la Fe
Valencia, 46026, Spain
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Hospital Vall d'Hebron
Barcelona, 08035, Spain
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Hospital de Santa Maria
Lisbon, 1649-035, Portugal
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IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147, Italy
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Instytut Centrum Zdrowia Matki Polki
?ód?, 93-338, Poland
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Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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Kagoshima University Hospital
Kagoshima, 890-8520, Japan
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Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
Warsaw, 02-097, Poland
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Kobe University Hospital
Hyōgo, 650-0017, Japan
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McGill University Health Centre - Glen Site
Montreal, Quebec, H4A 3J1, Canada
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National Center for Global Health and Medicine
Tokyo, 162-0052, Japan
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Neurology & Neuromuscular Care Center
Flower Mound, Texas, 75028, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
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Ospedali Riuniti Torrette di Ancona
Ancona, The Marches, 60126, Italy
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Policlinico Agostino Gemelli
Rome, Lazio, 00168, Italy
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Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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UZ Gent
Ghent, 9000, Belgium
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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Uniwersytecki Szpital Kliniczny w Poznaniu
Późna, 60-355, Poland
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Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-952, Poland
Conditions
Explore the condition pages connected to this study.