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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 26
- 试验地点
- 7
- 主要终点
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 1921 ].
研究概览
简要总结
The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol
排除标准
- •Treatment with another investigational therapy or enrollment in another interventional clinical study
- •Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566). Note: Other protocol defined Inclusion/Exclusion criteria may apply.
结局指标
主要结局
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 1921 ].
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 ]..
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 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 Electrocardiogram (ECG) [ Time Frame: Up to Day 1921 ]
Number of Participants With Shifts from Baseline in Vital Signs [ 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 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 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 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 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].
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 ].
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 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 ]
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 ]
次要结局
- 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 ]
研究者
Medical Director
Scientific
Biogen Idec Research Limited