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)

Study type

Interventional

Primary purpose

Treatment

Allocation

Non randomized

Intervention model

Parallel

Masking

Quadruple

Arms

2

Interventions

• Nusinersen Drug

• Muscular Atrophy, Spinal

Primary outcome

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (Up to Day 1921); Change from Baseline in Growth Parameters (Up to Day 1921); Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters (Up to Day 1921); Number of Participants With Shifts from Baseline in Electrocardiogram (ECG) (Up to Day 1921); Number of Participants With Shifts from Baseline in Vital Signs (Up to Day 1921); Change from Baseline in Activated Partial Thromboplastin Time (aPTT) (Up to Day 1921); Change from Baseline in Prothrombin Time (PT) (Up to Day 1921); Change from Baseline in International Normalized Ratio (INR) (Up to Day 1921); Change from Baseline in Urine Total Protein (Up to Day 1921); Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age (Up to Day 1921); Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age (Up to Day 1921); Percentage of Participa…

• Nusinersen

• NCT04729907 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read 25 Oct 2026

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.