SRK-015 / Apitegromab (Scholar Rock)
SRK-015 / Apitegromab (Scholar Rock)
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 sites in the UK:
| Sapphire Trial of Apitegromab funded by Scholar Rock | ||
|---|---|---|
| All applicants must be:
-receiving risdiplam or nusinersen |
||
| Trial Centre | Current status / Contact | Cut-off date |
| Leeds | Recruiting:
aaidah.patankar@nhs.net |
March – April 2023. Internationally competitive recruitment. |
| Oxford | Approved – at set up stage | |
| Great Ormond Street | Approved – at set up stage | |
| Liverpool | Approved – at set up stage | |
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.
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).