- Trial Name
- ASCEND
- Age:
- 5 – 39 years old
- Aim:
- To evaluate higher dose (HD) Nusinersen in Participants with SMA previously treated with risdiplam
- Status:
- Not yet started
- Locations:
- Trial Name
- DEVOTE
- Age:
- Child, adult, older adult
- Aim:
- To evaluate the safety and efficacy of higher doses of nusinersen
- Status:
- Recruiting
- Locations:
- Estonia, France, Germany, Greece, Hungary, Ireland, Italy, Latvia, Poland, Russian Federation, Spain, UK, Australia, Brazil, Canada, Columbia, Israel, Japan, Korea, Lebanon, Saudi Arabia, Taiwan & USA
- Trial Name
- RESPOND
- Age:
- ≤ 3 years old
- Aim:
- To evaluate higher doses of nusinersen in people previously treated with gene therapy
- Status:
- Recruiting
- Locations:
- Italy, Spain & USA
- Trial Name
- NURTURE
- Age:
- ≤ 6 weeks old
- Aim:
- To assess the efficacy, safety, tolerability, and pharmacokinetics of multiple doses of nusinersen delivered intrathecally to subjects with presymptomatic SMA
- Status:
- Active, not recruiting
- Locations:
- Italy, Germany, Turkey, Australia, Canada Qatar, Taiwan & USA
- Trial Name
- ENDEAR
- Age:
- ≤ 210 days old
- Aim:
- To assess the efficacy and safety of nusinersen in infants with SMA
- Status:
- Terminated
- Locations:
- Trial Name
- CHERISH
- Age:
- 2 to 12 years old
- Aim:
- To assess the efficacy and safety of nusinersen in participants with later-onset SMA
- Status:
- Completed
- Locations:
- Trial Name
- EMBRACE
- Age:
- Child, adult, older adult
- Aim:
- To assess the safety and tolerability of nusinersen in participants with SMA
- Status:
- Terminated
- Locations:
