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NCT04729907 · ClinicalTrials.gov registry record · Phase 3
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)
A Phase 3 study of Muscular Atrophy, Spinal, sponsored by Biogen.
- Active
- Registry status
- Phase 3
- Development phase
- 115
- Enrollment target
- 20
- Study locations
NCT04729907 is a Phase 3 study of Muscular Atrophy, Spinal that is active but no longer recruiting, run by Biogen. The registered enrollment target is 115 participants, above the 68-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (69% higher). The trial reports 20 study locations across 14 states.
The verdict
NCT04729907, a Phase 3 study of Muscular Atrophy, Spinal, is active but no longer recruiting, sponsored by Biogen.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 115 participants
- Enrollment target
- 20
- Study locations
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 |
Interested in This Trial?
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, categorized as Phase 3. The registered enrollment target is 115 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 68-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (69% higher). The listed sponsor is Biogen, which has 334 total studies on file at ClinicalTrials.gov.
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.
NCT04729907 reports 20 study locations spanning 14 distinct geographic areas - top geographies include Other, California, Colorado.
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.
What interventions are being tested in trial NCT04729907?
The interventions under investigation include: Nusinersen (DRUG).
Who is sponsoring clinical trial NCT04729907?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
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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