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

Trial statusNot yet recruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age6-18
SponsorYves Pastore

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

Treatment groups

29 Participants
are divided into 3 treatment groups

Sponsors and collaborators

Yves Pastore

Lead sponsor

St. Justine's Hospital

Sponsor institution

St. Justine's Hospital

Collaborator