Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT05067790 · ClinicalTrials.gov registry record · Phase 3
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)
A Phase 3 study of Spinal Muscular Atrophy, sponsored by Biogen.
- Active
- Registry status
- Phase 3
- Development phase
- 45
- Enrollment target
- 20
- Study locations
NCT05067790: Active Phase 3 study of Spinal Muscular Atrophy, sponsored by Biogen.
NCT05067790 is a Phase 3 study of Spinal Muscular Atrophy that is active but no longer recruiting, run by Biogen. The registered enrollment target is 45 participants, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (58% lower). The trial reports 20 study locations across 18 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05067790, a Phase 3 study of Spinal Muscular Atrophy, is active but no longer recruiting, sponsored by Biogen.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 45 participants
- Enrollment target
- 20
- Study locations
Study Summary
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 tota
Primary Outcome
The RULM is being utilized to assess upper limb functional abilities of participants with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
Conditions Studied
Interventions
- DRUG Nusinersen
Study Locations (20)
California
- Loma Linda University Children's Hospital - Loma Linda
- Stanford Neuroscience Health Center - Palo Alto
Texas
- Neurology Rare Disease Center - Denton
- The University of Texas Health Science Center at Houston - Houston
Arizona
- Barrow Neurological Institute - Phoenix
Arkansas
- Arkansas Children's Hospital - Little Rock
District of Columbia
- Georgetown University - Washington D.C.
Georgia
- Rare Disease Research, LLC - Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Iowa
- University of Iowa Stead Family Children's Hospital - Iowa City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2022-01-21 |
| Est. Completion | 2027-06-14 |
| Phase | Phase 3 |
What the registry record for NCT05067790 still lists
NCT05067790 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 45 participants, a relatively small participant target, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (58% lower).
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention - of which Nusinersen is the first listed.
NCT05067790 names 20 study sites across 18 states, led by California, Texas, Arizona.
Frequently Asked Questions
What is clinical trial NCT05067790 about?
NCT05067790 is a clinical study titled "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)". 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....
What is the current status of trial NCT05067790?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 45 participants. The study started on 2022-01-21. Estimated completion is 2027-06-14.
What conditions does trial NCT05067790 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy.
What interventions are being tested in trial NCT05067790?
The interventions under investigation include: Nusinersen (DRUG).
Who is sponsoring clinical trial NCT05067790?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05067790 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, District of Columbia, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05067790's enrollment target sits among peer trials
45 15th of 22 higher than 7 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
ACTIVE NOT RECRUITING · Phase 3
-
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
ACTIVE NOT RECRUITING · Phase 3
-
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
COMPLETED · Phase 3
-
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RECRUITING
-
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
RECRUITING · NA
-
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
RECRUITING · Phase 2
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00804154 · 45 participants · Phase 1
Resiniferatoxin to Treat Severe Pain Associated With Advanced Cancer
- NCT01851694 · 45 participants · NA
Beta-cell Response to Incretin Hormones in Cystic Fibrosis
- NCT02896335 · 45 participants · Phase 2
Palbociclib and Pembrolizumab In Central Nervous System Metastases
- NCT03454035 · 45 participants · Phase 1
Ulixertinib/Palbociclib in Patients With Advanced Pancreatic and Other Solid Tumors
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI