A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - RESPOND

Conditions

Spinal Muscular Atrophy

Brief summary

Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score [ Time Frame: Up to Day 778 ]

Detailed description

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Clinical Laboratory Parameters [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Electrocardiograms (ECGs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Vital Signs [ Time Frame: Up to Day 778 ], Number of Participants who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria [ Time Frame: Up to Day 778 ], Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Revised Upper Limb Module (RULM) Score [ Time Frame: Up to Day 778 ], Time to Death or Permanent Ventilation [ Time Frame: Up to Day 778 ], Change From Baseline in Cerebrospinal Fluid (CSF) Levels of Neurofilament Light Subunit (NF-L) [ Time Frame: Up to Day 659 ], Change From Baseline in Plasma Levels of NF-L [ Time Frame: Up to Day 778 ]

Related papers

No related papers found yet.

Interventions

DRUGSpinraza 12 mg solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score [ Time Frame: Up to Day 778 ]	—

Secondary

Measure	Time frame
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Clinical Laboratory Parameters [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Electrocardiograms (ECGs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Vital Signs [ Time Frame: Up to Day 778 ], Number of Participants who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria [ Time Frame: Up to Day 778 ], Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Revised Upper Limb Module (RULM) Score [ Time Frame: Up to Day 778 ], Time to Death or Permanent Ventilation [ Time Frame: Up to Day 778 ],	—

Measure

Time frame

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Clinical Laboratory Parameters [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Electrocardiograms (ECGs) [ Time Frame: Up to Day 778 ], Number of Participants with Change from Baseline in Vital Signs [ Time Frame: Up to Day 778 ], Number of Participants who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria [ Time Frame: Up to Day 778 ], Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score [ Time Frame: Up to Day 778 ], Change from Baseline in Revised Upper Limb Module (RULM) Score [ Time Frame: Up to Day 778 ], Time to Death or Permanent Ventilation [ Time Frame: Up to Day 778 ],

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Countries

Germany, Italy, Spain

Outcome results

None listed