The SAPPHIRE trial is an international, randomised, double-blind, placebo-controlled phase III trial that will evaluate the effects of a dual treatment :
- apitegromab (an anti-myostatin developed by Scholar Rock) injected intravenously every 4 weeks for one year,
- in addition to treatment with nusinersen or risdiplam,
- in non-walking patients with SMA type II or III, 156 aged 2 to 12 years and 48 aged 13 to 21 years.
Apitegromab has already shown positive results on motor function in the TOPAZ trial in 58 patients with SMA type II or III, aged 2 to 21 years.
Clinicaltrials.gov : NCT05156320
