Clinical Trials

Overview

Nusinersen (Spinraza™) – Biogen

In collaboration with researchers, nusinersen was developed by Ionis Pharmaceuticals and Biogen Idec.

Marketed as Spinraza™, it was the first potentially disease-modifying treatment available in Europe for 5q SMA. It is an antisense oligonucleotide (ASO) designed to modify the product of the SMN2 “backup” gene to produce more functional SMN protein.

About Spinraza™

Method of Action

ASOs are small snippets of synthetic genetic material that bind ribonucleic acid (RNA). They are often described as molecular patches because they can be specifically designed to target and affect how a particular gene is read.

ASOs have great potential for SMA, because they can accurately target the SMN2 gene“patching it up” to act more like SMN1. They do this by binding to the RNA template made by SMN2 and enhancing the inclusion of exon 7 into the SMN protein.

View Method of Action

Trials of Nusinersen

Trial Name
ASCEND
Age:
5 – 39 years old
Aim:
To evaluate higher dose (HD) Nusinersen in Participants with SMA previously treated with risdiplam
Status:
Not yet started
Locations:
Identifier:
NCT05067790
Trial Name
DEVOTE
Age:
Child, adult, older adult
Aim:
To evaluate the safety and efficacy of higher doses of nusinersen
Status:
Recruiting
Locations:
Estonia, France, Germany, Greece, Hungary, Ireland, Italy, Latvia, Poland, Russian Federation, Spain, UK, Australia, Brazil, Canada, Columbia, Israel, Japan, Korea, Lebanon, Saudi Arabia, Taiwan & USA
Identifier:
NCT04089566
Trial Name
RESPOND
Age:
≤ 3 years old
Aim:
To evaluate higher doses of nusinersen in people previously treated with gene therapy
Status:
Recruiting
Locations:
Italy, Spain & USA
Identifier:
NCT04488133
Trial Name
NURTURE
Age:
≤ 6 weeks old
Aim:
To assess the efficacy, safety, tolerability, and pharmacokinetics of multiple doses of nusinersen delivered intrathecally to subjects with presymptomatic SMA
Status:
Active, not recruiting
Locations:
Italy, Germany, Turkey, Australia, Canada Qatar, Taiwan & USA
Identifier:
NCT02386553
Trial Name
ENDEAR
Age:
≤ 210 days old
Aim:
To assess the efficacy and safety of nusinersen in infants with SMA
Status:
Terminated
Locations:
Identifier:
NCT02193074
Trial Name
CHERISH
Age:
2 to 12 years old
Aim:
To assess the efficacy and safety of nusinersen in participants with later-onset SMA
Status:
Completed
Locations:
Identifier:
NCT02292537
Trial Name
EMBRACE
Age:
Child, adult, older adult
Aim:
To assess the safety and tolerability of nusinersen in participants with SMA
Status:
Terminated
Locations:
Identifier:
NCT02462759

Community updates

General

  • Nusinersen safety and effects on motor function in adult SMA type 2 and 3 in Italy – 5th October 2020

Access

  • September 2020
  • September 2019
  • March 2019
  • June 2018
  • April 2018

 

 

Label

  • Label update 8th September 2018

DEVOTE

  • New global clinical trial DEVOTE – 18th September 2019
  • DEVOTE clinical trial – 17th April 2020

NURTURE

  • Nurture update – 2nd September 2019

RESPOND

  • 20th July 2020

Related pages & resources