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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.

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SMA Types 2-3 (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.

  • 9th October 2018 – last clinical trial update. Read this and one other previous report, here.

‘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. 

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

ISIS PHARMACEUTICALS INITIATES NEW ISIS-SMNREXTENSION STUDY, SHINE

EMBRACE Study

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)

Safety and Efficacy of Nusinersen in Infants/Children with SMA: Part 1 of the Phase 2 EMBRACE Study

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