| AVXS-101-CL-101/ NCT02122952/ 2014 |
Phase 1, Open-Label, two experimental arms (low and high dose) of IV single-dose AVXS-101 |
SMA type 1 with two copies of SMN2 up to six or nine months age |
N=15 (3 in low dose cohort and 12 in high dose cohort) |
Safety and tolerability/ All patients in high dose (N=12) were event-free* and 10/12 sitting |
| START/ NCT03421977/2017 |
Observational, long-term follow-up |
Patients from AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 |
N=13 (10 patients from previous high cohort) |
Long term safety data / 6/10 pt sitting, 2/10 pt standing, 2/10 pt walking in 5 years follow-up- ONGOING |
| AVXS-101-CL-303 or STR1VE-US/ NCT03306277/ 2017 |
Phase 3, Open-Label, Single-Arm, of IV single-dose AVXS-101 |
SMA type 1 with one or two copies of SMN2 up to 6 months of age |
N=22 |
13 pt (59%) sitting for 30 sec up to 18m and 90.9% of event-free* survival |
| STR1VE-EU/ NCT03461289/ 2018 |
Phase 3, Open-Label, Single-Arm, of IV single-dose AVXS-101 |
SMA type 1 with one or two copies of SMN2 up to 6 months of age |
N=33 |
14 pt (44%) sitting for 10 sec up to 18m / 31 pt (97%) are event-free* at 14m of age |
| SPR1NT/ NCT03505099/ 2018 |
Phase 3, Open-Label, Single-Arm, of IV single-dose AVXS-101 |
Pre-symptomatic patients with two or three copies of SMN2 up to 42 days (6 weeks) of live at dosing date |
N=30 (cohort 1: two copies of SMN2 and cohort 2: three copies of SMN2) |
All pt event-free* at 14m of age and 11/15 (79%) sitting for 30 Sec (cohort 1)/ 8/15 pt standing for 3 sec and 6/15 walking (cohort 2) |
| STRONG/ NCT03381729/2017 |
Phase 1, Open-Label, dose-compared study of intrathecal single-dose AVXS-101 |
SMA type 2, with three copies of SMN2, older than 6m and up to 5 years old |
N=33 (3 different experimental cohorts: dose A, B and C) |
ONGOING - determine optimal dose, standing ability and change in HFMSE/ walking ability and change in ventilation support |
| OFELIA/ NCT05073133/ 2021 |
Phase 4, Open-label, single-arm of IV single-dose AVXS-101 |
Symptomatic SMA (any type) with any copy of SMN2 up to 24m of age and Weight ≤17 kg. |
N=16 |
ONGOING - AEs and SAEs, changes in vital signs, cardiac safety, and laboratory results/ development of motor milestones |
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