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A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
NCT04729907 · View on ClinicalTrials.gov ↗
Study Summary
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203. The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are: * How many participants have adverse events and serious adverse events during the study? * How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment? * How many participants have a low platelet count after treatment? * How many participants had a change in the time it took for their heart to recharge between beats after treatment? * How does each participant's height and other measures of growth change after treatment? * How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment? Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing. The 232SM302 study will be done as follows: * Participants will be screened to check if they can join the study. * Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study. * Each participant will receive nusinersen once every 4 months during the treatment period. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * The treatment period will last for up to 64 months (1921 days). * There will be a follow-up safety period that lasts from 4 to 8 weeks. * In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
Conditions Studied
Interventions
- DRUG Nusinersen
Study Locations (20)
Other
- Hospital de Clínicas de Porto Alegre — Porto Alegre
- Hospital das Clinicas - FMUSP — São Paulo
- Clinica MEDS La Dehesa — Santiago
- Hospital Universitario San Ignacio — Bogotá
- Tallinn Children's Hospital — Tallinn
- Fondazione Serena Onlus - Centro Clinico Nemo — Milan
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma
California
- Stanford University Medical Center — Sacramento
Colorado
- Children's Hospital Colorado — Aurora
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Maryland
- The Johns Hopkins Hospital — Baltimore
Massachusetts
- Boston Children's Hospital — Boston
Tennessee
- St. Jude Children's Research Hospital — Memphis
Texas
- Children's Medical Center — Plano
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 115 participants |
| Start Date | 2021-04-19 |
| Est. Completion | 2026-07-31 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04729907
The ClinicalTrials.gov registry entry for NCT04729907 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 115 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biogen, which has 74 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 1 intervention — of which Nusinersen is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04729907 reports 20 study locations spanning 14 distinct geographic areas — top geographies include Other, California, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04729907 about?
NCT04729907 is a clinical study titled "A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)". In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203. The main goal of the study is to ...
What is the current status of trial NCT04729907?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 115 participants. The study started on 2021-04-19. Estimated completion is 2026-07-31.
What conditions does trial NCT04729907 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04729907?
The interventions under investigation include: Nusinersen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04729907?
This trial is sponsored by Biogen, which has 74 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04729907 being conducted?
This trial has 20 study locations across California, Colorado, Illinois, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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