Study of Nusinersen Among Patients with Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
Phase 4
- Conditions
- Spinal muscular atrophy (SMA)
- Registration Number
- JPRN-jRCT2051210186
- Lead Sponsor
- Toda Yasuo
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
Inclusion Criteria
Genetic documentation of 5q SMA homozygous SMN1 gene deletion or mutation or compound heterozygous mutation.
- Must have received onasemnogene abeparvovec.
- Suboptimal clinical status as determined by the Investigator.
Exclusion Criteria
- Severe or serious AEs related to onasemnogene abeparvovec therapy that were ongoing.
- Prior exposure to nusinersen.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method - Total HINE Section 2 motor milestones score<br>Section 2 of the HINE is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.
- Secondary Outcome Measures
Name Time Method