- Trial Name
- SMART
- Age:
- Up to 17 years old
- Aim:
- To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 in people with SMA with bi-allelic mutations in the SMN1 gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
- Status:
- Recruiting
- Locations:
- Belgium, France, Portugal, Australia & Canada.
- Identifier:
- NCT04089566
- Trial Name
- START
- Age:
- Child, Adult, Older Adult
- Aim:
- A long term follow-up safety study of people in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering AVXS-101
- Status:
- Active, not recruiting
- Locations:
- USA
- Identifier:
- NCT03421977
- Trial Name
- STRONG
- Age:
- Children6 to 60 months old
- Aim:
- To evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with SMA with 3 copies of SMN2 and deletion of SMN1.
- Status:
- Suspended
- Locations:
- USA
- Identifier:
- NCT03381729
- Trial Name
- STR1VE
- Age:
- Up to 180 days old
- Aim:
- Phase 3, open-label, single-arm, single-dose gene replacement therapy clinical trial for infants with SMA Type 1 with one or two SMN2 copies delivering AVXS-101 by intravenous infusion
- Status:
- Completed
- Locations:
- USA
- Identifier:
- NCT03306277
- Trial Name
- SPRINT
- Age:
- Up to 42 days
- Aim:
- A global study of a single, one-time dose of AVXS-101 delivered to infants with genetically diagnosed and pre-symptomatic SMA with multiple copies of SMN2
- Status:
- Completed
- Locations:
- Belgium, Germany,Italy, Spain, UK, Israel, Australia, Canada, Japan, Korea, Taiwan, USA
- Identifier:
- NCT03505099