Higher doses of SMA drug tested for Long-Term safety
NCT ID NCT04729907
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-260, United States
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Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
Guadalajara, 44280, Mexico
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Medical Center
Plano, Texas, 75024, United States
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Clinica MEDS La Dehesa
Santiago, 7691236, Chile
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168, Italy
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Guangzhou Woman and Children's Medical Center
Guangzhou, Guangdong, 510623, China
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Hospital Infantil de Mexico Federico Gomez
Mexico City, Mexico City, 6720, Mexico
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Hospital Sant Joan de Deu
Esplugues Del Llobregat, Barcelona, 8950, Spain
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Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
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Hospital Universitario La Paz
Madrid, 28046, Spain
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Hospital Universitario San Ignacio
Bogotá, 110231, Colombia
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Hospital das Clinicas - FMUSP
São Paulo, 5403900, Brazil
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Hospital de Clínicas de Porto Alegre
Porto Alegre, 90035-903, Brazil
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Hyogo Medical University Hospital
Nishinomiya-shi, Hyōgo, 663-8501, Japan
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Instituto Nacional de Pediatria
Mexico City, Mexico City, 4530, Mexico
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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King Fahad Specialist Hospital
Dammam, 31444, Saudi Arabia
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King Faisal Specialist Hospital & Research Center
Riyadh, 11211, Saudi Arabia
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Kurume University Hospital
Kurume-shi, Fukuoka, 830-0011, Japan
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London Health Sciences Centre (LHSC) - Children's Hospital
London, Ontario, N6A 5W9, Canada
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National Guard Health Affairs: King Abdulaziz Medical City
Jeddah, 21423, Saudi Arabia
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Peking University First Hospital
Beijing, Beijing Municipality, 100034, China
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Regional Pediatric Clinical Hospital #1
Yekaterinburg, 620149, Russia
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Russian Children Neuromuscular Center of Veltischev
Moskva, 125412, Russia
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Saint George University Hospital Medical Center
Beirut, 11 00 2807, Lebanon
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Stanford University Medical Center
Sacramento, California, 94304, United States
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Tallinn Children's Hospital
Tallinn, 13419, Estonia
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The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052, China
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The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
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Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo-To, 162-8666, Japan
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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Universitaetsklinikum Giessen und Marburg GmbH
Giessen, Hesse, 35392, Germany