Planned for the beginning of 2021, the goal of the phase IV RESPOND clinical trial, being conducted by Biogen, is to provide answers to a topical question: does taking Spinraza® (nusinersen) improve the effects of Zolgensma® (onasemnogene abeparvovec) in infants or children with SMA for whom the latter has not been sufficiently efficacious? To achieve this goal, this open-label study lasting 2 years will be made up of 2 groups:
- one group consisting of 40 infants likely to develop type 1 SMA, who received Zolgensma® treatment before the age of 6 months, and who will be a maximum of 9 months of age at the time of the first Spinraza® injection;
- the other consisting of 20 children who will be a maximum of 3 years of age at the time of the first Spinraza® injection.
The inclusion criteria will relate to the CHOP INTEND score, the need for respiratory assistance, the ability to swallow or feed themselves, etc. For the moment, no information has been communicated regarding which countries will be involved in this study.