Page checked: 29th November 2022
Last updated: 21st February 2022

The ENDEAR Trial¹’² was with 122 children with SMA Type 1 of whom two-thirds were treated with nusinersen. One third of these received sham treatment – a small needle prick to the skin over the lumbar area of the spine, which was covered with a bandage to simulate the appearance of a lumbar-puncture injection.

At a pre-specified time point in this study, the interim analysis of results showed a significantly higher number of patients in the nusinersen group had a motor-milestone response compared with patients in the sham group. This resulted in the trial being ended early after 13 months and patients from the sham group being moved on to active treatment. It is important to note that when this trial ended some children had completed the trial and had treatment for all 13 months, others were only part-way through.

The results for all the treated children, no matter how long their treatment had been, were that:

• 51% improved their developmental motor milestones as measured on the HINE scale for assessing motor function compared with 0% of the placebo group. These were the additional skills gained for the treated group:
• 22% of infants had head control;
• 10% could roll over;
• 8% could sit without support;
• 1% able to stand, compared with 0% of the placebo group.
• 71% of the treated group made a 4-point gain using the CHOP-INTEND (a second scale that also measures motor function) compared with 3% of the placebo group.
• 61% of the treated group did not require permanent ventilator support and were still alive. Permanent ventilator support is defined as more than 16 hours non-invasive ventilation support (NIV such as BiPap) for more than 21 days due to the impact of the condition. It does not include those who have to have NIV due to an ongoing infection. This compared with 32% of the placebo group.