Six-Year spinal injection study aims to confirm safety for rare muscle disease

NCT ID NCT04729907

Summary

This study follows 115 people with spinal muscular atrophy (SMA) for nearly six years to learn about the long-term safety of a higher dose of the drug nusinersen. Participants, who previously took part in a related study, receive the drug via a spinal injection every four months. Researchers are primarily checking for side effects and also monitoring changes in movement, breathing, and growth over time.

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Contacts and locations

Locations

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

    Chicago, Illinois, 60611-260, United States

  • Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde

    Guadalajara, 44280, Mexico

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Medical Center

    Plano, Texas, 75024, United States

  • Clinica MEDS La Dehesa

    Santiago, 7691236, Chile

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Fondazione Serena Onlus - Centro Clinico Nemo

    Milan, 20162, Italy

  • Guangzhou Woman and Children's Medical Center

    Guangzhou, Guangdong, 510623, China

  • Hospital Infantil de Mexico Federico Gomez

    Mexico City, Mexico City, 6720, Mexico

  • Hospital Sant Joan de Deu

    Esplugues Del Llobregat, Barcelona, 8950, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario San Ignacio

    Bogotá, 110231, Colombia

  • Hospital das Clinicas - FMUSP

    São Paulo, 5403900, Brazil

  • Hospital de Clínicas de Porto Alegre

    Porto Alegre, 90035-903, Brazil

  • Hyogo Medical University Hospital

    Nishinomiya-shi, Hyōgo, 663-8501, Japan

  • Instituto Nacional de Pediatria

    Mexico City, Mexico City, 4530, Mexico

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • King Fahad Specialist Hospital

    Dammam, 31444, Saudi Arabia

  • King Faisal Specialist Hospital & Research Center

    Riyadh, 11211, Saudi Arabia

  • Kurume University Hospital

    Kurume-shi, Fukuoka, 830-0011, Japan

  • London Health Sciences Centre (LHSC) - Children's Hospital

    London, Ontario, N6A 5W9, Canada

  • National Guard Health Affairs: King Abdulaziz Medical City

    Jeddah, 21423, Saudi Arabia

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Peking University First Hospital

    Beijing, Beijing Municipality, 100034, China

  • Regional Pediatric Clinical Hospital #1

    Yekaterinburg, 620149, Russia

  • Russian Children Neuromuscular Center of Veltischev

    Moskva, 125412, Russia

  • Saint George University Hospital Medical Center

    Beirut, 11 00 2807, Lebanon

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Stanford University Medical Center

    Sacramento, California, 94304, United States

  • Tallinn Children's Hospital

    Tallinn, 13419, Estonia

  • The Children's Hospital of Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310052, China

  • The Johns Hopkins Hospital

    Baltimore, Maryland, 21205, United States

  • Tokyo Women's Medical University Hospital

    Shinjuku-ku, Tokyo-To, 162-8666, Japan

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Universitaetsklinikum Giessen und Marburg GmbH

    Giessen, Hesse, 35392, Germany

Conditions

Explore the condition pages connected to this study.