Page last updated: 20th September 2023

SAPPHIRE Trial

Informed by the results of the TOPAZ trial (see below), SAPPHIRE is a randomised, double-blind, placebo-controlled Phase 3 study that aims to assess the safety and efficacy of apitegromab in conjunction with SPINRAZA® (nusinersen) or EVRYSDI® (risdiplam). Trial participants are non-ambulatory individuals aged 2-21 years living with Type 2 or Type 3 SMA. Patient enrolment for the SAPPHIRE trial was completed in September 2023.

Trial results and updates >

TOPAZ Trial

This Phase 2 study by Scholar Rock is a proof-of-concept trial designed to assess the safety and efficacy of apitegromab in conjunction with SPINRAZA® (nusinersen) or EVRYSDI® (risdiplam). Trial participants are non-ambulatory (unable to walk independently) individuals aged 2-21 years living with SMA Type 2 or Type 3.

ClinicalTrials.Gov >

Trial results and updates >

ONYX Trial

This is an open-label Phase 3 study by Scholar Rock designed to assess the long-term safety and efficacy of apitegromab in Patients with Type 2 and Type 3 SMA who have completed the TOPAZ or SAPPHIRE trial (see above).

ClinicalTrials.Gov >