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

Locations

  • A.O.U Policlinico G. Martino

    Messina, 98125, Italy

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, 20162, Italy

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

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHU Lille - Hôpital Jeanne de Flandre

    Lille, 59037, France

  • CHU Toulouse - Hopital des Enfants

    Toulouse, 31059, France

  • Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore

    Roma, 00168, Italy

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, 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 of Alabama

    Birmingham, Alabama, 35233, United States

  • Childrens Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Chr de La Citadelle

    Liège, 4000, Belgium

  • Columbia University, SMA Clinical Research Center

    New York, New York, 10032, United States

  • Dr. von Haunersches Kinderspital

    Munich, 80337, Germany

  • Foundation I.R.C.C.S. Carlo Besta Neurological Institute

    Milan, 20133, Italy

  • Gillette Children's Specialty Healthcare

    Saint Paul, Minnesota, 55101, United States

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, United Kingdom

  • Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Hospital Universitario y Politécnico La Fe

    Valencia, Spain

  • Hôpital Armand Trousseau, I-Motion

    Paris, 75012, France

  • IRCCS Istituto Giannina Gaslini

    Genoa, Italy

  • Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa

    Warsaw, 04-736, Poland

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nemours Children's Hospital

    Orlando, Florida, 32827, United States

  • Oregon Health & Sciences University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rady's Children's Hospital/UCSD

    San Diego, California, 92123, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • UZ Gent

    Ghent, 9000, Belgium

  • UZ Leuven

    Leuven, 3000, Belgium

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584, Netherlands

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Oxford

    Oxford, OX3 0ER, United Kingdom

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • University of Wisconsin School of Medicine and Public Health

    Madison, Wisconsin, 53705, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne w Gdańsku

    Gdansk, 80-001, Poland

  • Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu

    Poznan, 61-701, Poland

  • Wake Forest Baptist Medical Center

    Winston-Salem, North Carolina, 27157, United States

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.