The STR1VE clinical trial was set up following the positive results of the first trial concerning Zolgensma (onasemnogene abeparvovec) which evaluated its efficacy in 15 participants with type I SMA aged less than 6 months. In this new american, phase III, open-label clinical trial, 22 participants with type I SMA presenting one or two copies of the SMN2 gene and aged less than 6 months at the time of the single intravenous injection of Zolgensma were followed for once a week during 4 weeks, then once a month until the end of the study (at the age of 18 months of the participants).
A significant improvement
The results of this trial published in April 2021 show that:
- 1% of participants do not need permanent ventilation by the age of 14 months compared to 26% in a natural history cohort,
- 59% are able to sit alone for at least 30 seconds by the age of 18 months compared to 0% in this same natural history cohort,
- Zolgensma was well tolerated. Among the most frequently reported side effects are bronchiolitis (due in particular to respiratory syncytial virus), pneumonia and respiratory distress. Two patients have a high level of transaminases.
In Europe, the STR1VE-EU trial, designed with the same design, took place for France at the I-Motion Institute at Trousseau Hospital (Paris) with 33 participants. The data is being analyzed.
