New study tests higher dose of SMA drug over 6 years

NCT ID NCT04729907

Ongoing Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

First seen Feb 22, 2026 · Last updated May 24, 2026 · Updated 14 times

Summary

This study looks at the long-term safety of a higher dose of nusinersen (a drug for spinal muscular atrophy) in about 115 people who already completed a previous nusinersen study. Participants will receive the drug every 4 months via a lumbar puncture for up to 64 months, with follow-up visits over nearly 6 years. Researchers will track side effects, heart health, growth, and movement abilities.

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Contacts and locations

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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.

MUSCULAR ATROPHY, SPINAL