NCT06761560

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

• Status: Not Yet Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 29 (estimated)
• Sponsor: Yves Pastore · Academic / Other
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Sickle Cell Disease (SCD)

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	Pharmacokinetic based dosage change	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
DIAGNOSTIC_TEST	Pharmacokinetic dosing	Patient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint

Timeline

• Start date: 2025-01-15
• Primary completion: 2027-11-25
• Completion: 2027-11-25
• First posted: 2025-01-07
• Last updated: 2025-01-07

Locations

1 site across 1 country: Canada