Muscle-Boosting drug shows promise for children with rare neuromuscular disease
NCT ID NCT05156320
Summary
This completed Phase 3 trial tested whether adding the experimental drug apitegromab to standard SMA treatments could improve muscle function in 188 children and young adults (ages 2-21) with later-onset spinal muscular atrophy. All participants were already receiving approved SMA medications (nusinersen or risdiplam) and could not walk independently. The study measured changes in motor skills, upper limb function, and safety over 12 months.
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Contacts and locations
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Locations
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A.O.U Policlinico G. Martino
Messina, 98125, Italy
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ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHU Lille - Hôpital Jeanne de Flandre
Lille, 59037, France
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CHU Toulouse - Hopital des Enfants
Toulouse, 31059, France
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Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore
Roma, 00168, Italy
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, 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 of Alabama
Birmingham, Alabama, 35233, United States
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Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Chr de La Citadelle
Liège, 4000, Belgium
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Columbia University, SMA Clinical Research Center
New York, New York, 10032, United States
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Dr. von Haunersches Kinderspital
Munich, 80337, Germany
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Foundation I.R.C.C.S. Carlo Besta Neurological Institute
Milan, 20133, Italy
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Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Hospital Sant Joan de Déu
Barcelona, Spain
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Hospital Universitario y Politécnico La Fe
Valencia, Spain
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Hôpital Armand Trousseau, I-Motion
Paris, 75012, France
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IRCCS Istituto Giannina Gaslini
Genoa, Italy
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Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa
Warsaw, 04-736, Poland
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Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX, United Kingdom
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Oregon Health & Sciences University
Portland, Oregon, 97239, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Rady's Children's Hospital/UCSD
San Diego, California, 92123, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Stanford University Medical Center
Palo Alto, California, 94304, United States
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The Johns Hopkins University
Baltimore, Maryland, 21287, United States
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UZ Gent
Ghent, 9000, Belgium
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UZ Leuven
Leuven, 3000, Belgium
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
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Universitair Medisch Centrum Utrecht
Utrecht, 3584, Netherlands
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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 Oxford
Oxford, OX3 0ER, United Kingdom
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University of Utah
Salt Lake City, Utah, 84112, United States
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University of Wisconsin School of Medicine and Public Health
Madison, Wisconsin, 53705, United States
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Universitätsklinikum Bonn
Bonn, 53127, Germany
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Universitätsklinikum Essen
Essen, 45147, Germany
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Uniwersyteckie Centrum Kliniczne w Gdańsku
Gdansk, 80-001, Poland
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Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu
Poznan, 61-701, Poland
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Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
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Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.