Phase 3 STAND Trial Supports Safety and Tolerability of Crizanlizumab in SCD

Crizanlizumab is safe and well-tolerated in patients with sickle cell disease, according to results from the STAND trial published in The Lancet Haematology.

Results from the SUSTAIN study previously demonstrated a lower rate of vaso-occlusive crises in patients treated with 5.0 mg/kg crizanlizumab versus placebo. In the randomized, double-blind, phase 3 STAND study, Miguel R Abboud, MD, American University of Beirut Medical Center, and colleagues further evaluated the safety and efficacy of two doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) compared with placebo.

Of the 252 patients included in the study, 85 were randomized to the crizanlizumab 7.5 mg/kg group, 84 to the crizanlizumab 5.0 mg/kg group, and 83 to the placebo group. The primary endpoint was the annualized rate of vaso-occlusive crises that led to a healthcare visit over the first year of treatment. The secondary objective was safety.

The adjusted annualized rate of vaso-occlusive crises was 2.49 (95% CI, 1.90–3.26) in the crizanlizumab 5.0 mg/kg group, 2.04 (1.56–2.65) in the 7.5 mg/kg group, and 2.30 (1.75–3.01) in the placebo group. The rate of adjusted annualized rates of vaso-occlusive crises leading to healthcare visits was 1.08 (95% CI 0.76–1.55, P>0.999) for 5.0 mg/kg and 0.89 (0.62–1.27, P>0.999) for 7.5 mg/kg vs placebo.

The researchers noted that the safety profile was consistent among the three treatment groups. Grade 3 or higher adverse events (AEs) occurred in 27 (32%) patients in the placebo group, 32 (39%) in the crizanlizumab 7.5 mg/kg group, and 47 (56%) in the crizanlizumab 5.0 mg/kg group. The rates of serious AEs followed a similar trend across treatment groups, occurring in 26 (31%) of patients in the placebo group, 22 (27%) in the crizanlizumab 7.5 mg/kg group, and 35 (42%) in the crizanlizumab 5.0 mg/kg group.

While the rates of AEs were lower in the placebo and crizanlizumab 7.5 mg/kg groups than the crizanlizumab 5.0 mg/kg group, there were no differences in efficacy between crizanlizumab and placebo.

Reference

Abboud MR, et al. Lancet Haematol. 2025;12(4):e248-e257. doi:10.1016/S2352-3026(24)00384-3