Tests hydroxyurea dosing for children with sickle cell disease in Africa

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children with sickle cell anemia (HbSS or HbS/B0-thalassemia), aged 6 months to 12 years, to test a dosing approach for hydroxyurea in Africa.

Age

6 months to 12 years

Sex

Boys and girls can both join.

Disease stage

Sickle cell anemia, either HbSS or HbS/B0-thalassemia.

Earlier treatment

Cannot have used hydroxyurea in the past 6 months. Cannot have had a blood transfusion in the past 6 months (this is a temporary exclusion).

Main reasons for not taking part

• Enlarged spleen with signs of hypersplenism (platelet count <150,000, hemoglobin <5 g/dL or absolute neutrophil count <1.0 x10^9/L)
• Hydroxyurea use within the past 6 months
• Blood transfusion within the past 6 months (temporary)
• Pregnancy
• Severe blood count problems already present (temporary)

The registry criteria, word for word

What the trial wants to find out

Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year. Most infants with SCA are born in the low-resource settings of sub- Saharan Africa, where an estimated 50-90% will die before 5 years of age due to lack of early diagnosis and appropriate care. Hydroxyurea is a safe and effective once-daily oral medication that has become the standard of care for the treatment of children with SCA in high-resource settings. There is now a growing body of evidence to support the safety and clinical benefits of hydroxyurea for the treatment of SCA in sub-Saharan Africa. The requirement for frequent laboratory monitoring, uncertainties about appropriate, most effective dosing, and the concern for hematologic laboratory toxicities, however, will continue to limit widespread hydroxyurea utilization and real-world effectiveness. The investigators have recently developed and prospectively evaluated an individualized, pharmacokinetics-guided hydroxyurea dosing strategy for children with SCA that has demonstrated optimal clinical and laboratory benefits with minimal toxicity. In this research study, the investigators aim to extend this precision medicine approach to Africa.

The 2 groups in the trial

• Weight Based Starting DoseExisting treatment, for comparison

  25 mg/kg starting dose Hydroxyurea

  Treatment: Hydroxyurea

• PK-guided starting doseTreatment being tested

  Individualized, PK-guided starting dose Hydroxyurea

  Treatment: Hydroxyurea