Table 4 TOPAZ trial – study design

Design

• n = 23; age 5–21 years

• n = 15; age 5–21 years

• n = 20; age ≥ 2 years

• Open-label, single-arm

• Open-label, single-arm

• Double-blind, randomized (1:1) to 2 mg/kg or 20 mg/kg

• 20 mg/kg SRK-015 IV Q4W

• 20 mg/kg SRK-015 IV Q4W

• SRK-015 IV Q4W

• 12-month treatment period

• 12-month treatment period

• 12-month treatment period

Patients

• Ambulatory Type 3 SMA

• Type 2 or nonambulatory Type 3 SMA

• Type 2 SMA

• Concomitant therapy with approved SMN upregulator (n = 12) or monotherapy (n = 11)

• Concomitant therapy with approved SMN upregulator started ≥ 5 years

• Initiated treatment with approved SMN upregulator before age 5 years

• RHS Scores ≤ 63

• HFMSE Scores ≥ 10

• HFMSE Scores ≥ 10

Primary Objectives

• Safety

• Safety

• Safety

• Mean change from baseline in RHS

• Mean change from baseline in HFMSE

• Mean change from baseline in HFMSE