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Higher doses of SMA drug tested for Long-Term safety

NCT ID NCT04729907

Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

115 people

The number who actually took part.

Started

Apr 2021

Finished

Jul 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol Key Exclusion Criteria: * Treatment with another investigational therapy or enrollment in another interventional clinical study * Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566) NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

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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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