RECRUITING Phase 2 / Phase 3 ClinicalTrials.gov

Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia

Sponsor: Yves Pastore • ClinicalTrials.gov ID: NCT06761560

Registry updated: 2026-08-21

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Description

The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted. Patient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up. Group C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time.

Interventions

Pharmacokinetic based dosage change (Diagnostic test)
This study will compare 2 groups of sickle cell patients that are receiving hydroxyurea. Group A will have an increase in their dosage based on the pharmacokinetic result over a period of 12 months and Group B will have an increase in their dosage based on the standard of care follow-up over a period of 12 months. The aim is to evaluate if the group A can reach MTD faster than than the Group B
Pharmacokinetic dosing (Diagnostic test)
Patient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint

Eligibility criteria

Inclusion Criteria (Group A and B) : * Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis. * Patients with SS, SBThal0. * Agree to take hydroxyurea for a period of 12 months * Be between age of 6months old and 18 years old. * Have consented for participation in the study. Inclusion Criteria (Group C) : * Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis. * Patients with SS, SBThal0. * Have taken hydroxyurea for a period of at least 12 months, and have received HU at a stable dose and at MTD for at least 6 months. * Be between age of 6months old and 18 years old. * Have consented for participation in the study. Exclusion Criteria: * Patients with sickle cell genotype other than SS or SBThal0 (SC, SBThal+, SE or SD) * Patients on chronic transfusion program * Patients have received a blood transfusion in the last 4 weeks of study enrollment. * Have received a hematopoietic stem-cell transplantation * Creatinine \>2x normal for age * ALT\>2x normal for age * Sexually active females unwilling to comply with reliable method of birth control * Pregnancy * Conditions which in the opinion of the investigator, would compromise participation in the study will be excluded.

Conditions

Sickle Cell Disease (SCD)

Locations

CHU Sainte-Justine — Montreal, Quebec, Canada

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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