Table 2 Update on clinical trials for onasemnogene abeparvovec in spinal muscular atrophy (SMA)
Trial name (ID) | Phase/route | Patient recruited | Interventional model | Status | Outcome measure |
|---|---|---|---|---|---|
SMART (NCT04851873) | 3b/i.v | Any (symptomatic) SMA type, weighed ≧8.5 kg and ≦21 kg | Multi-center, open label, single group assignment | Completed | Safety, efficacy |
STEER (NCT05089656) | 3a/i.t | SMA type 2 (able to sit, never walked), aged 2 to ≦18 years | Multi-center randomized, sham-controlled double-blind study, cross over assignment | Ongoing | Safety, efficacy |
STRONG (NCT03381729) | 1/i.t | SMN2: 3 copies, aged ≧ 6 months and < 5 years | Parallel assignment, open label, non-randomized, multi-center | Terminated | Safety, tolerability |
STRENGTH (NCT05386680) | 3b/i.t | Discontinued treatment with nusinersen or risdiplam, aged 2 to < 18 years | Open-label, non-randomized, single arm, multi-center study | Ongoing | Safety, tolerability, efficacy |
SPECTRUM (NCT05335876) | 3/i.v. and i.t | Long-term follow-up of patients with SMA treated with onasemnogene abeparvovec in clinical trials | 15-years follow up from the date of onasemnogene abeparvovec administration | Ongoing | Safety, tolerability, efficacy |