Trials for later-onset SMA

CS2/CS12

Phase 1/2a, open-label, multiple-dose, dose-escalation analysis across 2 studies1 :

  • 28 ambulatory and nonambulatory patients aged 2 to 16 years at time of first dose with later-onset (Types 2 and 3) spinal muscular atrophy (SMA) over a 3-year period (~36 months)1,2
  • Safety and tolerability of SPINRAZA® (nusinersen) and the long-term effects of treatment with SPINRAZA on motor function were assessed1


Patients received up to 3 doses of SPINRAZA in study CS2 and up to 4 doses in study CS12. The dosage (3 mg to 12 mg in CS2 and 12 mg in CS12) and administration schedule differed from the approved SPINRAZA regimen. The median interval between the last dose in CS2 and the first dose in CS12 was 319 days.1,2

Limitations of this study include that it was open-label with no internal control groups. Because the 28 patients received their first dose in CS2 and then transferred to the second phase, CS12, the interval time between studies ranged from ~7 to ~13 months. The dosing and administration schedule in these studies differed from the approved SPINRAZA regimen.1,3

Improved motor
function1

Increased ambulation1

Image of Lauren aged 20 with Type 3 SMA Wheelchair

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.