Crizanlizumab for Sickle Cell Disease

(STAND Trial)

This trial tests the effectiveness of two different doses of crizanlizumab, a treatment for sickle cell disease, compared to a placebo in individuals with sickle cell disease (SCD) who have experienced painful vaso-occlusive crises (VOCs) requiring healthcare visits. Researchers aim to determine if crizanlizumab can reduce these painful crises and ensure its safety. The trial seeks participants who have had at least two healthcare visits for VOCs in the past year and may already be on other treatments like hydroxyurea but still experience pain. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for SCD.

The trial requires that if you are taking hydroxyurea (HU/HC), L-glutamine, or an erythropoietin stimulating agent, you must have been on a stable dose for at least 3 months before the trial and plan to continue it during the study. If you are not taking these medications, you must not have taken them for at least 6 months before joining the trial.

Studies have shown that crizanlizumab is generally well-tolerated by people with sickle cell disease. Research indicates that both the 5.0 mg/kg and 7.5 mg/kg doses of crizanlizumab are safe and help reduce the number of painful episodes requiring hospital visits. In one study, the 5.0 mg/kg dose reduced the likelihood of these painful episodes by 45%.

Other research shows that crizanlizumab works by blocking a protein called P-selectin and has a tolerable safety profile. Most participants in these studies did not experience severe side effects. While some side effects occurred, they were usually mild and manageable.

Crizanlizumab is unique because it targets a specific protein called P-selectin, which plays a key role in the painful blockages that occur in sickle cell disease. Unlike standard treatments like hydroxyurea, which works by increasing fetal hemoglobin, Crizanlizumab prevents the adhesion of sickled cells to blood vessels, potentially reducing the frequency of painful crises. Researchers are excited about Crizanlizumab because it offers a new mechanism of action, giving hope for more effective management of sickle cell disease symptoms and improving patients' quality of life.

Research suggests that crizanlizumab, one of the treatments under study in this trial, may help reduce emergency visits for people with sickle cell disease (SCD), particularly those frequently requiring hospital care. Studies have shown that crizanlizumab works by blocking a protein called P-selectin, which can cause blood cells to clump and obstruct blood flow. However, some research indicated that crizanlizumab did not significantly reduce the number of painful episodes or related healthcare visits in certain trials. While crizanlizumab shows promise, its effectiveness in reducing sickle cell crises remains under investigation. Participants in this trial will receive either crizanlizumab at different dosages or a placebo to further evaluate its impact.¹²⁵⁶⁷