LIBRARilzabrutinib for Sickle-Cell Disease in Patients Aged 10 to 65

This study is focused on finding out how effective and safe a drug called rilzabrutinib is for people aged 10 to 65 who have sickle-cell disease, a condition that affects the shape and function of red blood cells. The study aims to see if rilzabrutinib can help improve the health of patients with this disease over the course of a year. This research is crucial because sickle-cell disease can cause pain and other serious health problems, and current treatment options are limited. Participants in the study are randomly assigned to receive either the drug rilzabrutinib or a placebo, which is an inactive substance, for 52 weeks. The study is "double-blind," meaning neither the participants nor the researchers know who is receiving the drug or the placebo to ensure unbiased results. After completing the initial 52 weeks, participants may continue in an open-label phase where everyone receives rilzabrutinib. The effects of the treatment are monitored through regular visits and assessments to determine its impact on participants' health and safety.