ong-Term Extension Study of Nusinersen in Participants With SMA
Phase 3
- Conditions
- Spinal muscular atrophy
- Registration Number
- JPRN-jRCT2031220035
- Lead Sponsor
- Irzhevsky Victoria
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 172
Inclusion Criteria
Completion of the Day 302 Visit in Study 203 and compliance with the study protocol throughout Study 203.
Exclusion Criteria
- 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 203.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method - Incidence of AEs, including SAE<br><br>- Change in growth parameters<br><br>- Shifts from baseline in clinical laboratory parameters, ECGs, and vital signs<br><br>- Shifts from baseline in coagulation parameters (aPTT, PT, and INR)<br><br>- Change in urine total protein<br><br>- Change from baseline in neurological examination outcomes<br><br>- The proportion of participants with a postbaseline platelet count below the lower limit of normal on at least 2 consecutive measurements<br><br>- The proportion of participants with a postbaseline QTcF of > 500 msec and an increase from baseline to any postbaseline timepoint in QTcF of > 60 msec
- Secondary Outcome Measures
Name Time Method - Total number of new WHO motor milestones<br><br>- Ventilator use<br><br>- Time to death (overall survival)<br><br>- Change from baseline in CHOP INTEND total score<br><br>- Change from baseline in HINE Section 2 motor milestones<br><br>- Proportion of HINE Section 2 motor milestone responders<br><br>- Proportion of time on ventilation<br><br>- Time to death or permanent ventilation (tracheostomyor >= 16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event)<br><br>- Change from baseline in HFMSE score<br><br>- Change from baseline in RULM score