Adjusting hydroxyurea dose using pharmacokinetics in children with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This interventional trial tests a pharmacokinetic–driven approach to hydroxyurea dosing in patients with sickle cell disease, comparing PK-guided dose increases to standard care. Group A uses PK results to adjust dose, Group B follows usual bloodwork-based adjustments, and Group C undergoes a single PK dose check after long-term HU use. The study aims to determine if PK-guided dosing can accelerate attainment of the maximum tolerated dose over a 12-month period. Participants are aged 6 months to 18 years and must have confirmed SCD, with specific eligibility criteria for each group.

Why it matters

Who can join: the full rules

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.

Where it's running

• CHU Sainte-Justine
  Montreal, Quebec, Canada Recruiting

Study contacts

• Study coordinator: Yves Pastore, MD
• Email: yves.pastore.med@ssss.gouv.qc.ca
• Phone: 514-345-4931

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Sickle cell disease registry at Ain Shams University

  This project creates an Ain Shams University registry of children and young people with sickle cell disease to track their health over time without providing a treatment.

  Recruiting Ain Shams University · Cairo and 1 other locations · NCT07800247

• Point-of-care newborn SCD screening and early care in Côte d'Ivoire

  This trial tests whether using a Gazelle-Multispectral point-of-care test at birth plus early, comprehensive SCD care can reduce illnesses and deaths for babies with SCD in the first years of life.

  Recruiting Phase 4 Swiss Tropical & Public Health Institute · Abidjan and 1 other locations · NCT07488520

• Sickle Cell Kidney Sample Bank

  This project will collect blood and urine from adults with sickle cell disease or sickle cell trait to see if linked samples and medical records can help researchers learn how kidney disease starts and progresses.

  Recruiting at 2 sites University of Texas Southwestern Medical Center · Dallas, Texas and 1 other locations · NCT07064174

• Comparing stem cell transplantation to disease-modifying therapy for children with sickle cell disease

  This study is trying to see how well stem cell transplants work compared to other treatments for children with sickle cell disease over three years.

  Recruiting at 16 sites University of Rochester · Birmingham, Alabama and 36 other locations · NCT06941389

• Examining megakaryocytes in patients with sickle cell disease

  This study looks at how certain blood cells called megakaryocytes behave in people with sickle cell disease to see if they play a role in the complications of the illness.

  Recruiting University Hospital, Toulouse · Toulouse, France · NCT06887907

• Depo-Provera (depot medroxyprogesterone acetate) for pain and biomarker changes in women with sickle cell disease

  This study will test whether an injectable birth control (Depo-Provera) can reduce pain in adult women with sickle cell disease.

  Recruiting Phase 4 University of Pennsylvania · Atlanta, Georgia and 1 other locations · NCT06665997

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.