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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)

NCT04729907 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 115

Last updated 2026-02-18

No results posted yet for this 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

  • Muscular Atrophy, Spinal

Interventions

DRUG

Nusinersen

Administered as specified in the treatment arm

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-19
Primary Completion
2026-07-31
Completion
2026-07-31
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Chile
  • China
  • Colombia
  • Estonia
  • Germany
  • Italy
  • Japan
  • Lebanon
  • Mexico
  • Russia
  • Saudi Arabia
  • Spain
  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04729907 on ClinicalTrials.gov