Recruiting
Phase 2
Phase 3

Hydroxyurea

Sponsor:

Yves Pastore

Code:

NCT06761560

Conditions

Sickle Cell Disease (SCD)

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

Interventions

Pharmacokinetic based dosage change

Pharmacokinetic dosing

Study Details

Brief 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)

Study ID

NCT06761560

Start date

Feb 3, 2026

Status verified date

Aug, 2026

Completion date

Nov 25, 2028

Anticipated

Primary completion date

Nov 25, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

29 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Other

Interventions and Outcome Measures

Arms

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.

Interventions

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

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

Primary outcome measure

  • Evaluation of HU-PK at 6 months between group A and group B [ Time Frame: At 6 months ]

Central Contacts and Locations

Central contacts

Yves Pastore, MD

514-345-4931yves.pastore.med@ssss.gouv.qc.ca

Bianka Courcelle, Research nurse, RN

514-345-4931bianka.courcelle.hsj@ssss.gouv.qc.ca

Locations

CHU Sainte-Justine

Recruiting

Montreal, Quebec, Canada, H3T 1C5

Contacts

Yves Pastore, MD

514-345-4931yves.pastore.med@ssss.gouv.qc.ca

Karyne Daigle, RN

514-345-4931karyne.daigle.hsj@ssss.gouv.qc.ca

More Information

Sponsor

Yves Pastore

Last update posted

Aug 21, 2026

Last verified

Aug, 2026

Keywords

  • hydroxyurea
  • pharmacokinetic
  • sickle cell disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Yves Pastore on 2026-08-21.