Table 1.
Completed OA IV clinical trials.
| Study number phase country(ies) | Study design/route of administration/duration of treatment | Number of subjects | Diagnosis of patients | SMA type (SMN2 Copy No.) | NCT number |
|---|---|---|---|---|---|
|
START Phase 1 United States |
Open-label, single-center IV Single dose |
15 | Patients with SMA Type 1 with 2 copies of SMN2 < 6 months of age at the time of gene replacement therapy. | 1 (2) | NCT02122952 |
|
STR1VE Phase 3 United States |
Open-label, single-arm, multicenter IV Single dose |
22 | Patients with SMA Type 1 with 1 or 2 copies of SMN2 < 6 months of age at the time of gene replacement therapy | 1 (1,2) | NCT03306277 |
|
SPR1NT Phase 3 Global (multi-countries) |
Open-label, single-arm, multicenter IV Single dose |
29 | Pre-symptomatic patients with genetically confirmed SMA Type 1 or 2 with 2 or 3 copies of SMN2, ≤ 6 weeks of age at the time of gene replacement therapy |
1, 2 (2, 3, 4) |
NCT03505099 |
|
STR1VE-EU Phase 3 Europe |
Open-label, single-arm, multicenter IV Single dose |
33 | Patients with SMA Type 1 with 1 or 2 copies of SMN2 < 6 months of age at the time of gene replacement therapy | 1 (1,2) | NCT03461289 |
|
STR1VE-AP Phase 3 Asia |
Open-label, single-arm, multicenter IV Single dose |
2 |
Patients with SMA Type 1 with 1 or 2 copies of SMN2 < 6 months of age at the time of gene replacement therapy |
1 (1, 2) | NCT03837184 |