Results and Updates for Each Nusinersen Trial
Results and Updates for Each Nusinersen Trial
A preliminary trial with 20 infants with SMA Type 1 age 3 weeks – 6 months was ended early due to its interim positive results. These children were transitioned into the ENDEAR study Phase 3 which followed 122 children with SMA Type 1.
This was with 126 children with SMA Type 2 aged 2 – 12 years The first child was treated in November 2014. As a result of the positive interim results, it was ended early in November 2016 as it was considered unethical to continue to give the sham procedure to a group of children when nusinersen was effective at treating symptoms.
This is a long-term study, that in November 2015 enrolled 292 children who had previously participated in the ENDEAR and CHERISH phase III clinical trials. All participants continued to receive nusinersen. Study completion date is August 2023.
These studies, from October 2012 until 2019, were of children aged 2 – 15 years who had SMA Type 2 or Type 3 and had initially received nusinersen as part of the early Phase 1 and 2 studies.
This phase 2 trial started in May 2015 and aims to evaluate the safety and tolerability of nusinersen in 25 pre-symptomatic newborns who, based on their genetics, are very likely to develop SMA. It is due to complete in January 2025.
This 14 months Phase 2 study from August 2015 – September 2018 enrolled 21 infants and children with SMA who were ineligible for inclusion in the ENDEAR and CHERISH Phase 3 trials.
This trial started in March 2020, to evaluate the safety, tolerability and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved (12mg) for the treatment of SMA. It’s estimated that there are 172 participants. Primary completion is the end of November 2022; study completion early July 2023.
This phase 4 study, due to be completed in September 2024 is evaluating the benefit of Spinraza (nusinersen) in patients treated with Zolgensma (onasemnogene abeparvovec). The study plans to include 60 children who have unmet clinical needs after receiving gene therapy (as identified by a doctor). It is not recruiting in the UK.
Announced in September 2021, Biogen plans to initiate this new clinical trial to evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes and address unmet medical needs in people with later-onset SMA who were previously treated with risdiplam. Up to 135 later-onset, non-ambulatory (unable to walk independently) people with SMA aged 5-39 years will be enrolled.
All participants must have been treated with the maximum dose of 5 mg of risdiplam before joining the study and be willing and able to change their treatment regimen to a higher dose of nusinersen. Further eligibility criteria also apply.