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)
Public ClinicalTrials.gov record NCT05067790. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05067790
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 45 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 15 Years to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 20, 2022
- Primary completion
- Sep 23, 2027
- Completion
- Sep 23, 2027
- Last update posted
- May 21, 2026
2022 – 2027
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| Stanford Neuroscience Health Center | Palo Alto | California | 94304 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115-5724 | — |
| Memorial Healthcare | Owosso | Michigan | 48867 | — |
| Columbia University | New York | New York | 10032 | — |
| Wake Forest University - School of Medicine - Central | Winston-Salem | North Carolina | 27157 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| Neurology Rare Disease Center | Denton | Texas | 76208 | — |
| The University of Texas Health Science Center at Houston | Houston | Texas | 77030 | — |
| University of Washington Medical Center | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 11 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05067790, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 21, 2026 · Synced Sep 1, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05067790 live on ClinicalTrials.gov.