Results of the SPR1NT trial: Zolgensma under study in presymptomatic

2 August 2022

This trial was conducted worldwide (but not in France) in 14 infants with presymptomatic SMN1-related proximal spinal muscular atrophy (SMA) with two copies of SMN2 over 18 months, and in 15 infants with three copies of SMN2 over 24 months.

The data from these two follow-up periods show that :

For the 14 infants with two copies of SMN2 :

  • all are able to sit up independently for at least 30 seconds,
  • 71% can stand up,
  • 71% can walk independently,
  • none of them required permanent ventilation, respiratory or nutritional support,
  • 13 of them maintained their weight.

For the 15 infants with three copies of SMN2 :

  • all can stand independently,
  • 14 of them can walk independently,
  • none of them needed permanent ventilation or respiratory or nutritional assistance.

These results confirm the importance of treating as early as possible and therefore the urgency of implementing newborn screening.

 

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Strauss KA, Farrar MA, Muntoni F et al. Nat Med. 2022 Jun 17:1–9.

 

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Strauss KA, Farrar MA, Muntoni F et al. Nat Med. 2022 Jun 17:1–8.

 

Further details on Clinicaltrials.gov: NCT03505099