Overview
A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
Status:
Recruiting
Recruiting
Trial end date:
2024-09-04
2024-09-04
Target enrollment:
0
0
Participant gender:
All
All
Summary
The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec. The secondary objectives of this study are to evaluate the safety and tolerability; and clinical outcomes following treatment with nusinersen in participants with SMA who previously received onasemnogene abeparvovec.Phase:
Phase 4Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Biogen
Criteria
Key Inclusion Criteria:- Genetic documentation of 5q SMA homozygous gene SMN1 deletion or mutation, or compound
heterozygous mutation
- Must have previously received onasemnogene abeparvovec
- Participants with suboptimal clinical status per the Investigator
Key Exclusion Criteria:
- Prior exposure to nusinersen
- Ongoing severe or serious AEs related to onasemnogene abeparvovec
Note: Other protocol defined Inclusion/Exclusion criteria may apply.