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

Status: Recruiting
Location: See location...
Intervention Type: Diagnostic test
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

• 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.

Locations
Other Locations
Canada
CHU Sainte-Justine
RECRUITING
Montreal
Contact Information
Primary
Yves Pastore, MD
yves.pastore.med@ssss.gouv.qc.ca
514-345-4931
Backup
Bianka Courcelle, Research nurse, RN
bianka.courcelle.hsj@ssss.gouv.qc.ca
514-345-4931
Time Frame
Start Date: 2026-02-03
Estimated Completion Date: 2028-11-25
Participants
Target number of participants: 29
Treatments
Experimental: Group A
Patient starting hydroxyurea that will have PK sample procurements at different timpoints over a period of 12 months until HU-AUC shows MTD
No_intervention: Group B
Patient starting hydroxuyrea that will be followed as per standard of care over a period of 12 months until MTD has been reached
Experimental: Group C
Patient will undergo one PK sample procurement to evaluate level of HU-AUC after 12 months of taking hydroxyurea.
Sponsors
Leads: Yves Pastore
Collaborators: St. Justine's Hospital

This content was sourced from clinicaltrials.gov

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