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A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD

Status
Active, not recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-505637-27-00
Acronym
232SM302
Enrollment
26
Registered
2024-06-27
Start date
2021-07-12
Completion date
Unknown
Last updated
2026-01-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal Muscular Atrophy

Brief summary

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 1921 ]., Change from Baseline in Growth Parameters [ Time Frame: Up to Day 1921 ].., Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters [ Time Frame: Up to Day 1921 ], Number of Participants With Shifts from Baseline in Electrocardiogram (ECG) [ Time Frame: Up to Day 1921 ], Number of Participants With Shifts from Baseline in Vital Signs [ Time Frame: Up to Day 1921 ], Change from Baseline in Activated Partial Thromboplastin Time (aPTT) [ Time Frame: Up to Day 1921 ], Change from Baseline in Prothrombin Time (PT) [ Time Frame: Up to Day 1921 ], Change from Baseline in International Normalized Ratio (INR) [ Time Frame: Up to Day 1921 ], Change from Baseline in Urine Total Protein [ Time Frame: Up to Day 1921 ], Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age [ Time Frame: Up to Day 1921]., Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age [ Time Frame: Up to Day 1921 ]., Percentage of Participants With A Postbaseline Platelet Count Below the Lower Limit of Normal on at least 2 Consecutive Measurements [ Time Frame: Up to Day 1921 ], Percentage of Participants With a Postbaseline Corrected QT Interval Using Fridericia's Formula (QTcF) of >500 millisecond (msec) and an Increase from Baseline to Any Postbaseline Timepoint in QTcF of >60 msec [ Time Frame: Up toDay 1921 ]

Detailed description

Total Number of New World Health Organization (WHO) Motor Milestones [ Time Frame: Up to Day 1921], Number of Participants Who Used Respiratory Support, by Type [ Time Frame: Up to Day 1921 ], Number of Hours Per Day of Respiratory Support [ Time Frame: Up to Day 1921 ], Number of Days That Respiratory Support Is Used [ Time Frame: Up to Day 1921 ], Time to Death (Overall Survival) [ Time Frame: Up to Day 1921 ], Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score [ Time Frame: Up to Day 1921 ]., Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones [ Time Frame: Up to Day 1921 ]., Percentage of HINE Section 2 Motor Milestone Responders [ Time Frame: Up to Day 1921 ]., Percentage of Time Spent on Ventilation [ Time Frame: Up to Day 1921 ]., Time to Death or Permanent Ventilation [ Time Frame: Up to Day 1921 ]., Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score [ Time Frame: Up to Day 1921 ]., Change from Baseline in Revised Upper Limb Module (RULM) Score [ Time Frame: Up to Day 1921 ].., Change From Baseline in Plasma Levels of Neurofilament (NF) [ Time Frame: Up to Day 1921 ]

Interventions

Sponsors

Biogen Idec Research Limited
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 1921 ]., Change from Baseline in Growth Parameters [ Time Frame: Up to Day 1921 ].., Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters [ Time Frame: Up to Day 1921 ], Number of Participants With Shifts from Baseline in Electrocardiogram (ECG) [ Time Frame: Up to Day 1921 ], Number of Participants With Shifts from Baseline in Vital Signs [ Time Frame: Up to Day 1921 ], Change from Baseline in Activated Partial Thromboplastin Time (aPTT) [ Time Frame: Up to Day 1921 ], Change from Baseline in Prothrombin Time (PT) [ Time Frame: Up to Day 1921 ], Change from Baseline in International Normalized Ratio (INR) [ Time Frame: Up to Day 1921 ], Change from Baseline in Urine Total Protein [ Time Frame: Up to Day 1921 ], Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age [ Time Frame: Up to Day 1921]., Number of Particip

Secondary

MeasureTime frame
Total Number of New World Health Organization (WHO) Motor Milestones [ Time Frame: Up to Day 1921], Number of Participants Who Used Respiratory Support, by Type [ Time Frame: Up to Day 1921 ], Number of Hours Per Day of Respiratory Support [ Time Frame: Up to Day 1921 ], Number of Days That Respiratory Support Is Used [ Time Frame: Up to Day 1921 ], Time to Death (Overall Survival) [ Time Frame: Up to Day 1921 ], Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score [ Time Frame: Up to Day 1921 ]., Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones [ Time Frame: Up to Day 1921 ]., Percentage of HINE Section 2 Motor Milestone Responders [ Time Frame: Up to Day 1921 ]., Percentage of Time Spent on Ventilation [ Time Frame: Up to Day 1921 ]., Time to Death or Permanent Ventilation [ Time Frame: Up to Day 1921 ]., Change from Baseline in Hammersmith Functional Motor Sca

Countries

Germany, Italy, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026