SMA Newsroom

Sep 20, 2022

Community update Scholar Rock: SAPPHIRE clinical trial sites in Europe

A community update has been shared by Scholar Rock regarding the sites for SAPPHIRE clinical trials in Europe. 

The SAPPHIRE study intends to enroll approximately 204 individuals with SMA across Europe and the USA. The plan is to have European trial sites in Belgium, France, Germany, Italy, Netherlands, Poland, Spain, and the United Kingdom. 

Scholar Rock has begun enrolling patients in Europe, with the first clinical trial sites up and running in Spain. The team is working on opening additional sites.

About apitegromab

Apitegromab is an antibody that selectively inhibits the activation of myostatin (a protein found in skeletal muscles that restricts muscle growth), which may improve muscle growth and motor function in people living with SMA.

SAPPHIRE trial overview

SAPPHIRE is a global Phase 3 study which aims to assess the safety and efficacy of apitegromab in conjunction with SPINRAZA® (nusinersen) or EVRYSDI® (risdiplam) in individuals aged 2-21 years with Type 2 or Type 3 non-ambulatory (unable to walk independently) SMA. The trial will have 2 groups of participants with the following estimated number of participants:

  • 156 individuals aged 2-12 years.
  • 48 individuals aged 13-21 years.

When enrolled, study participants will be randomly assigned to either apitegromab or placebo while continuing their existing treatment with either SPINRAZA® or EVRYSDI® under the supervision of their healthcare provider.

The treatment period (when participants will receive apitegromab or placebo) in the trial is planned to be 12 months long. After completing the 12 months, participants will have a 20-week follow-up period or have the option to enrol into an extension study.

During the follow-up period, participants will stop taking apitegromab but will continue their treatment with either SPINRAZA® or EVRYSDI®.

All participants in the extension study will receive apitegromab while continuing their treatment with either SPINRAZA® or EVRYSDI®.