Pharmacogenetics and Model-Informed Optimisation of Hydroxyurea Study
Summary
The NIH registered NCT07543289, an observational prospective cohort pharmacokinetic study evaluating pharmacogenetics, pharmacodynamics, and pharmacokinetics of hydroxyurea therapy in 100 Nigerian patients with sickle cell disease. The study will develop PopPK and PK-PD models using Monolix and exposure-response analyses in R to support a precision medicine dosing algorithm for hydroxyurea in this population.
What changed
NIH published a clinical trial registration for a prospective cohort study of hydroxyurea pharmacogenetics in Nigerian sickle cell disease patients. The study will enroll 100 participants (both hydroxyurea-experienced and naive patients) and develop population pharmacokinetic and PK-PD models to characterise interindividual variability in drug response. Analytical methods for hydroxyurea quantification via HPLC will also be developed and validated.
Healthcare researchers and pharmaceutical companies with interests in sickle cell disease therapeutics should note this study as a potential contributor to evidence-based hydroxyurea dose-optimisation algorithms for African populations. The study's focus on genetic polymorphisms in metabolic enzymes and drug transporters may inform future pharmacogenomic labelling or clinical guidance for hydroxyurea use in patients of African descent.
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Apr 21, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Pharmacogenetics and Model-Informed Optimisation of Hydroxyurea
Observational NCT07543289 Kind: OBSERVATIONAL Apr 21, 2026
Abstract
The wide interindividual variability in clinical response to hydroxyurea therapy in the management of sickle cell disease has limited its use. These variabilities have been linked to differences in pharmacodynamics, pharmacokinetics, and pharmacogenetics. This study, therefore, aims to enhance understanding of these factors as they relate to hydroxyurea therapy, with the overall goal of developing a precision medicine algorithm.
The study will be a prospective cohort pharmacokinetic study of 100 Nigerian patients with sickle cell disease, including current hydroxyurea users and naive patients. Pharmacodynamic markers will be collected to evaluate response. PopPK and PK-PD models will be developed in Monolix, exposure-response relationships will be analysed in R, and pregnancy, lactation, and paediatrics PBPK models will be developed in Simcyp or PK-SIM to inform dose optimisation.
This study aims to build a pharmacometric model by integrating differences in pharmacokinetics, pharmacodynamics, and pharmacogenetics of hydroxyurea, which could aid the optimisation of hydroxyurea for sickle cell patients in Nigeria.
The objectives of the study are i. To determine the prevalence of genetic polymorphisms in metabolic enzymes and transporters relevant to the disposition of hydroxyurea in the Nigerian sickle cell disease population, ii. To develop and validate an analytical method for the quantification of hydroxyurea using high-performance liquid chromatography.
iii. To evalu...
Conditions: Sickle Cell Disease
Interventions: Hydroxyurea (HU)
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