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NCT Number: NCT06761560

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

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.

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Key information

Conditions

Sickle Cell Disease (SCD) Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

6 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

(Group A and B) :

  • 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.
  • 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.
  • Be 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.

Treatment and study plan

Pharmacokinetic based dosage change

Diagnostic Test

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

Diagnostic Test

Patient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint

Primary outcomes

  1. Evaluation of HU-PK at 6 months between group A and group B

    Time frame: At 6 months

    Pharmakocinetic dosage of hydroxyurea will be determined at 6 months in group A and group B. We hypothesize that HU-PK in group B may be lower (suboptimal) compared to group A.

Secondary outcomes

  1. Time to reach maximal tolerated dose (MTD)

    Time frame: 3, 6, 9 and 12 months

    Time (weeks) to achieve MTD in groups A and B will be determined by evaluating the % of patients reaching MTD (at 3 , 6, 9 and 12 months) in each group.

    MTD is defined by hematological parameters: Absolute neutrophile count 0.8-1.5x10*9/L or platelet 80-120x10*9/L or absolute reticulocyte count 50-80x10*9/L)

  2. Fetal hemoglobin

    Time frame: at 3, 6 and 12 months

    Comparing fetal hemoglobin between group A and B

  3. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) in group A and B

    Time frame: From enrollment to 12 months

    Evaluation of the incidence adverse events (AE) and serious adverse events (SAE) in both groups

  4. Evaluation of % of patients reaching AUC of 115 +/- 15mg*h/L at 12 months compared to the percentage of patients in group C reaching the same AUC

    Time frame: At 12 months

    Percentage of patients in the HU-AUC (group A) with an AUC of 115 mg*h/L at 12 months will be compared to the percentage of patients in group C with an AUC of 115 +/-15 mg*h/L.

Study contacts

Contact information is provided by the study sponsor or research team.

Bianka Courcelle, Research nurse, RN

CONTACT

[email protected]

514-345-4931 ext. 3757

Yves Pastore, MD

CONTACT

[email protected]

514-345-4931 ext. 5027

Sponsors and collaborators

Lead sponsor

Yves Pastore

Other

Collaborators

  • St. Justine's Hospital

Registry information

Acronym: OPTIMA

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Jan 7, 2025
Registry last updated
Jan 7, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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