Spinal Muscular Atrophy
Conditions
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
Eligibility
Design outcomes
Primary
| Measure | Time 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
| Measure | Time 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