Nusinersen for Spinal Muscular Atrophy

(ONWARD Trial)

Recruiting at 52 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Biogen

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Medical Director

Principal Investigator

Biogen

Eligibility Criteria

This trial is for individuals with spinal muscular atrophy (SMA) who completed the Day 302 visit in a prior study (NCT04089566). They must not have received other investigational therapies or participated in another interventional clinical study, nor started any approved SMA therapy after that specific visit.

Inclusion Criteria

Please provide more details for an accurate summary.

Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol

Exclusion Criteria

Treatment with another investigational therapy or enrollment in another interventional clinical study

You have been treated with approved medication for spinal muscular atrophy after a certain study visit.

Treatment Details

Interventions

• Nusinersen (Antisense Oligonucleotide)

Trial Overview The trial is testing the long-term safety and effectiveness of higher doses of Nusinersen given through spinal injection to those with SMA. Participants are from a previous Nusinersen study and will continue to receive this drug to assess ongoing benefits and risks.

Participant Groups

Group I: BIIB058 50/28 mg (Prior Maintenance Dose 12 mg) Experimental Treatment 1 Intervention

Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.

Group II: BIIB058 28 mg (Prior Maintenance Dose 28 mg) Experimental Treatment 1 Intervention

Participants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada