Therapeutic Response Evaluation and Adherence Trial (TREAT)
Status:
Recruiting
Trial end date:
2024-10-01
Target enrollment:
Participant gender:
Summary
The primary objectives of this prospective study of hydroxyurea for children with sickle cell
anemia are 1) Develop and prospectively evaluate a population
pharmacokinetic/pharmacodynamics model to predict the maximum tolerated dose (MTD); 2)
Identify urine biomarkers of hydroxyurea adherence using a novel metabolomics approach; 3)
Identify pharmacogenomics modifiers of hydroxyurea MTD; and 4) Longitudinal monitoring of the
effect of hydroxyurea upon organ function and quality of life.