Beirut

Researchers are evaluating roxadustat, an oral medicine, to treat anemia in children and teenagers with chronic kidney disease (CKD). Anemia is a condition where the body has a low level of red blood cells. Currently, anemia is often treated with injections called erythropoietin stimulating agents (ESAs), but these have some safety concerns. Roxadustat is already licensed for adults with CKD, and this study aims to learn how it affects anemia in younger patients, providing a potential alternative to injections. Participants in this open-label Phase 3 study will take roxadustat three times a week for up to 52 weeks. The study starts with 10 teenagers taking a fixed dose for 4 weeks to determine the best dose, followed by 10 children doing the same. After dosing is established, the rest of the children and teenagers will take roxadustat at the suitable dose. Blood samples will be taken regularly, and doses may be adjusted to keep hemoglobin levels just below the normal range. Visits occur every 2 weeks for the first month, then every 4 weeks, with a final visit 4 weeks after treatment ends. During the study, participants will have their vital signs checked, including blood pressure, temperature, and heart rate. Those on dialysis will have fluid levels monitored. Blood and urine tests will be done regularly, including checks for hemoglobin, iron, liver enzymes, and heart function through ECGs. Medical exams occur before treatment and at 6 and 13 months. Researchers will monitor safety, treatment effects, and hemoglobin changes throughout the study.