Key Clinical Trial Results
SMA Type 1 (ENDEAR Trial)
In August 2016, Biogen and Ionis announced that as an interim analysis of this trial with 20 children had met its primary endpoint, it would end early. All 20 children were transitioned into SHINE (see below). Biogen then opened its global Expanded Access Programme (EAP) for SMA Type 1.
- 15th December 2017 - last clinical trial update following 121 children. Read this and other previous reports on ENDEAR, here.
SMA Type 2 (CHERISH Trial)
On November 7th 2016, Biogen and Ionis announced that as an interim analysis of this trial with 126 children with later onset SMA Type 2 had met its primary endpoint, it would end early. All the children were transitioned into SHINE (see below). In February 2018, the prestigious New England Journal of Medicine published the trial results.
- March 28th 2018 - last clinical trial update following 126 children. Read this and other previous reports on CHERISH, here.
Pre-symptomatic SMA (NURTURE Trial)
This phase 2 trial 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.
- 10th May 2019 – latest and previous clinical trial updates
- 24th October 2019 - Updated NURTURE Study Results for the European Community
‘Biomarkers’ research to find ways to tell whether a medication has been successful
At the 23rd International Annual Congress of the World Muscle Society on 8th October 2018, Biogen presented the findings of blood tests done on over 300 participants receiving nusinersen in their clinical trials. They were looking at finding a way of telling whether a medication had been successful using a marker in the blood; these are called ‘biomarkers’. There was evidence that levels of phosphorylated neurofilament heavy chain (pNF-H) reduced immediately after treatment with nusinersen and stayed similar to levels of that seen in someone without SMA.
At the American Academy of Neurology 2019 conference, an update on this was given which you can read here.
SMA Types 1-3 (SHINE Trial)
This open-label, phase 3 study extends the treatment regime of patients who have completed the ENDEAR or CHERISH trials.
- 23rd October 2015
This Phase 2 trial enrolled a small number of SMA patients who were ineligible for inclusion in the ENDEAR and CHERISH Phase 3 trials.
- 15th November 2017 - (Poster 2)
DEVOTE Clinical Trial
Announced publicly via press release at the 13th Congress of the European Paediatric Neurology Society (EPNS), in Athens, the DEVOTE study will evaluate if higher dosing of nusinersen can provide improved efficacy in the treatment of SMA across a broad population of patients.
- 18th September 2019