About The Pioneer Study
People with Sickle Cell Disease (SCD) have a disease that causes their red blood cells to be crescent (sickle)-shaped instead of round. These cells have difficulty carrying oxygen and can get stuck. This can cause pain, tiredness, and create difficulty breathing. In this study, researchers are exploring if an investigational oral medicine (Pociredir) still being studied is safe and has the desired biologic effect for which it was designed to treat SCD. The study medicine is investigational because it has not been approved for use except in clinical trials.
All people that are eligible to take part in the study will take medicine by mouth once a day for 12 weeks. The total time on the study may be up to 4 months, including follow- up with the doctor or study team 4 weeks after finishing study treatment.