A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) (NCT05067790)
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
🩺 Plain-English Summary (8th-Grade Level)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on teenagers and adults who are unable to walk on their own and who have previously taken another drug for SMA called risdiplam. The main goal of this study is to learn about the effect of high dose (HD) nusinersen on muscle and movement ability (motor function) in SMA. The main question that researchers want to answer is: \- How do the scores of a movement test called the Revised Upper Limb Module change from the start of treatment? The Revised Upper Limb Module is a test used to measure a participant's ability to do specific tasks that involve their shoulders, arms, wrist, elbows, and hands. It measures the changes in their abilities over time. Researchers will also learn more about the safety of HD nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests. The study will be done as follows: * Participants will be screened to check if they can join the study. * After screening, participants will enter the Core Treatment period. * At the start of the Core Treatment period, they will receive 2 "loading" doses of nusinersen. These are 50 mg doses of nusinersen given 2 weeks apart. * Afterwards, they will continue to receive "maintenance" doses of nusinersen once every 4 months. These doses will be 28 mg. * The Core Treatment period will last about 2 years, with a follow-up visit 4 months after the last dose. * Participants who complete the Core Treatment period will have the option to continue receiving 28 mg of nusinersen in the Long-Term Extension (LTE) period for about 2 years. There will also be a follow-up visit 4 months after the last dose. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * In total, participants will have up to 18 study visits. They will also be called by researchers after each dose of nusinersen. * Participants will stay in the study for about 4.5 years if they complete both the Core Treatment and LTE periods.
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
Age range: 15 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (22 Sites)
- Barrow Neurological Institute — Phoenix, Arizona
- Stanford Neuroscience Health Center — Palo Alto, California
- Rare Disease Research, LLC — Atlanta, Georgia
- Boston Children's Hospital — Boston, Massachusetts
- Memorial Healthcare — Owosso, Michigan
- Columbia University — New York, New York
- Wake Forest University - School of Medicine - Central — Winston-Salem, North Carolina
- University of Pennsylvania — Philadelphia, Pennsylvania
- Neurology Rare Disease Center — Denton, Texas
- The University of Texas Health Science Center at Houston — Houston, Texas
- University of Washington Medical Center — Seattle, Washington
- Universitaetsklinikum Heidelberg — Heidelberg, Baden-Wurttemberg
- Universitaetsklinikum Ulm — Ulm, Baden-Wurttemberg
- Klinikum rechts der Isar der TU Muenchen — Munich, Bavaria
- Universitaetsklinikum Essen — Essen, North Rhine-Westphalia
- Semmelweis Egyetem — Budapest,
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan,
- Fondazione Serena Onlus - Centro Clinico Nemo — Milan,
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma,
- Ospedale S G Battista Molinette — Torino,
- Yokohama City University Hospital — Yokohama, Kanagawa
- NHO Osaka Toneyama Medical Center — Toyonaka-shi, Osaka
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Spinal Muscular Atrophy
- Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — Phoenix, Arizona
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb — Pittsburgh, Pennsylvania
- Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Phoenix, Arizona
- Spinraza in Adult Spinal Muscular Atrophy — Phoenix, Arizona
- Early Check: Expanded Screening in Newborns — Research Triangle Park, North Carolina
Explore by condition: All Spinal Muscular Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.