The TOPAZ trial tested the safety, tolerability and efficacy of the anti-myostatin apitegromab in SMA over one year in the United States and Europe (but not in France).
- The 58 participants, aged between 2 and 21 years, received apitegromab intravenously every 4 weeks for one year.
- The first cohort (open-label) included 23 participants with type III SMA, aged between 5 and 21 years, ambulant and receiving a high dose of apitegromab (20 mg/kg) with or without Spinraza.
- The second cohort (open-label) included 15 participants with type II or III SMA, aged 5 to 21 years, non-ambulatory, all treated with Spinraza and receiving a high dose of apitegromab (20 mg/kg).
- The third cohort (double-blind, randomised) consisted of 20 participants with type II SMA, aged 2 years or older, non-ambulatory and receiving either high-dose apitegromab (20 mg/kg) or low-dose apitegromab (2 mg/kg) in combination with Spinraza.
- Results after one year of treatment showed improved motor function on the Hammersmith scale, especially for participants in cohort 3.
An extension phase is ongoing and has already confirmed these positive results after three years of treatment. The other apitegromab trial, called SAPPHIRE, has completed recruitment of its participants.
Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial. Scholar Rock. Communiqué de presse du 19 septembre 2023
