Etavopivat for Sickle Cell Disease (HIBISCUS,HEM-301)

“This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients, age 12 to 65 years, with sickle cell disease (SCD).”

This study is investigating a new medication called etavopivat for children with sickle cell disease. Etavopivat is taken as a tablet and is intended to activate a specific enzyme in red blood cells, making them more stable and less likely to break down, thereby alleviating anemia and the typical symptoms of the disease. In the study, participants are randomly assigned to receive either etavopivat or a placebo, without them or their treating physicians knowing who is receiving which treatment; this allows for a particularly reliable assessment of efficacy and safety. The study primarily investigates whether hemoglobin levels improve, whether there are fewer pain crises and hospitalizations, and what side effects occur.

The goal of the study is to determine whether etavopivat is a viable long-term, easy-to-use treatment option for patients with sickle cell disease.
 

Patient enrollment in Heidelberg is possible since July 2023.