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

Last updated: January 5, 2025
Sponsor: Yves Pastore
Overall Status: Active - Not Recruiting

Phase

2/3

Condition

N/A

Treatment

Pharmacokinetic based dosage change

Pharmacokinetic dosing

Clinical Study ID

NCT06761560
2023-4674
  • Ages 6-18
  • All Genders

Study 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 Criteria

Inclusion

Inclusion Criteria (Group A and B) :

  • Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab withhemoglobin 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.

  • Have consented for participation in the study.

Inclusion Criteria (Group C) :

  • Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab withhemoglobin electrophoresis.

  • Be patients with SS, SBThal0.

  • Have taken hydroxyurea for a period of at least 12 months, and have received HU at astable 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

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 participationin the study will be excluded.

Study Design

Total Participants: 29
Treatment Group(s): 2
Primary Treatment: Pharmacokinetic based dosage change
Phase: 2/3
Study Start date:
January 15, 2025
Estimated Completion Date:
November 25, 2027

Connect with a study center

  • CHU Sainte-Justine

    Montreal, Quebec H3T 1C5
    Canada

    Site Not Available

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