Clinical Trial of Hydroxyurea Treatment Using Precision Dosing for Children Aged 6 Months to 12 Years with Sickle Cell Anemia in Africa

What this Trial Is About

Sickle cell anemia SCA is a common and serious blood disorder affecting over 300,000 newborns yearly, mostly in sub-Saharan Africa where many children die young due to lack of early diagnosis and care. Researchers are evaluating hydroxyurea, an oral medication already standard in high-resource countries, to improve treatment safety and effectiveness for children with SCA in Africa. This trial aims to test a personalized dosing approach that could allow safer use of hydroxyurea with limited laboratory monitoring in low-resource settings. The study involves 400 children aged 6 months to 12 years in Luanda, Angola, randomly assigned to receive either a fixed 25 mgkg starting dose of hydroxyurea or an individualized dose guided by pharmacokinetics using a battery-powered device and computer algorithm. This precision dosing method seeks to optimize benefits while minimizing toxicity. The trial will assess safety and clinical outcomes over approximately 24 months of treatment. Participants will be closely monitored for adverse events and blood responses through clinical assessments and laboratory tests during the first year and throughout treatment. The main outcomes measured include rates of serious sickle cell-related and non-related adverse events. This research will help create guidelines for hydroxyurea use in African children with SCA, supporting wider and safer access to this treatment in resource-limited areas.

Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa