Higher doses of SMA drug tested for Long-Term safety

NCT ID NCT04729907

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

First seen Jun 25, 2026 · Last updated Aug 12, 2026 · Updated 4 times

Summary

This long-term study is testing higher doses of nusinersen (Spinraza) in people with spinal muscular atrophy who already completed a previous nusinersen study. The main goal is to check safety over several years, including side effects, heart health, and growth. Participants receive the drug via lumbar puncture every 4 months for up to 64 months. The study involves 115 people and is not recruiting new participants.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: nusinersen (Spinraza)
What this could lead to: If this works, it could show that higher doses of nusinersen are safe and help maintain or improve motor function for people with spinal muscular atrophy over the long term.
What could go wrong: This is an extension study with no placebo group, so results may be less clear. Higher doses could also bring new side effects. The study is small (115 people) and focused on safety, not proof of effectiveness.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUSCULAR ATROPHY, SPINAL

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 Las Condes

Santiago, 7550000, Chile
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 Luis Calvo Mackenna

Santiago, 7500539, Chile
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

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