About this trial
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.
Eligibility criteria
Qualifiers
Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.
Be patients with SS, SBThal0.
Agree to take hydroxyurea for a period of 12 months
Be between age of 6months old and 18 years old.
Disqualifiers
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
Trial design
Treatments tested in this trial
- Pharmacokinetic based dosage change
- Pharmacokinetic dosing