The PTN’s hydroxyurea (HU) study is kicking into high gear, with sites reportedly expecting an influx of at least 5 newly enrolled patients over the upcoming spring holiday.
Hydroxyurea is the only major drug breakthrough for the treatment of sickle cell disease within the past 20 years. Although it is approved by the Food and Drug Administration (FDA) to treat adults with sickle cell anemia, few studies have been done with HU in children to tell us how it is absorbed and expelled in maturing bodies. The PTN study will fill those knowledge gaps.
The HU study will enroll eligible children aged 2–17 years with a confirmed diagnosis of sickle cell anemia. For children aged 5–17 years, it will also explore whether the body handles the drug differently in liquid versus pill form. Results will provide information to help the FDA in considering approval of HU for use in infants and children with sickle cell anemia.