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)
Running, not enrolling · Phase 3
Conditions studied: Muscular Atrophy, Spinal
In brief
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.
Key facts
- Study ID
- NCT04729907
- Run by
- Biogen
- People needed
- 115
- Starts
- 2021-04-19
- Expected to finish
- 2026-07-31
- Last updated by the study team
- 2026-02-18
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Stanford University Medical Center — Sacramento, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- The Johns Hopkins Hospital — Baltimore, Maryland, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States
- Children's Medical Center — Plano, Texas, United States
- Hospital de Clínicas de Porto Alegre — Porto Alegre, Brazil
- Hospital das Clinicas - FMUSP — São Paulo, Brazil
- London Health Sciences Centre (LHSC) - Children's Hospital — London, Ontario, Canada
- Clinica MEDS La Dehesa — Santiago, Chile
- Peking University First Hospital — Beijing, Beijing Municipality, China
- Guangzhou Woman and Children's Medical Center — Guangzhou, Guangdong, China
- The Children's Hospital of Zhejiang University School of Medicine — Hangzhou, Zhejiang, China
- Hospital Universitario San Ignacio — Bogotá, Colombia
- Tallinn Children's Hospital — Tallinn, Estonia
- Universitaetsklinikum Freiburg — Freiburg im Breisgau, Baden-Wurttemberg, Germany
- Universitaetsklinikum Giessen und Marburg GmbH — Giessen, Hesse, Germany
- Fondazione Serena Onlus - Centro Clinico Nemo — Milan, Italy
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma, Italy
- Kurume University Hospital — Kurume-shi, Fukuoka, Japan
- Hyogo Medical University Hospital — Nishinomiya-shi, Hyōgo, Japan
- Tokyo Women's Medical University Hospital — Shinjuku-ku, Tokyo-To, Japan
- Saint George University Hospital Medical Center — Beirut, Lebanon
- Instituto Nacional de Pediatria — Mexico City, Mexico City, Mexico
Full record on ClinicalTrials.gov
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