Actively Recruiting
Optimizing the Management of Sickle Cell Patients on Hydroxyurea Using Therapeutic Pharmacological Monitoring
Led by University Hospital, Strasbourg, France · Updated on 2025-02-13
30
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Sickle cell disease is a serious condition marked by repeated painful episodes called vaso-occlusive crises (CVO), early complications, and high risks of illness and death. Hydroxyurea (HU) is a treatment used to reduce these crises by increasing fetal hemoglobin, which can improve patients' health and lifespan. This study aims to find the best way to adjust HU doses quickly and safely to reach the maximum tolerated dose (MTD), potentially reducing the time patients spend under suboptimal treatment and lowering the frequency of painful crises. The study involves two groups of patients aged 2 to 35 years with severe sickle cell disease who need HU treatment or dose adjustment. One group will have their dose adjusted based on standard blood tests, while the other group will have a personalized dose adjustment using pharmacological monitoring of drug exposure (AUC) and blood tolerance. Patients will take HU orally, starting at a standardized dose, and undergo regular visits every 3 months up to 15 months for clinical and biological assessments, dose adjustments, and blood tests to monitor treatment tolerance. Participants will have multiple visits including initial information, consent, randomization, and follow-up assessments at months 3, 6, 9, 12, and 15. At each visit, medical history, physical exams, blood tests, and pharmacokinetic measurements will be done to guide dose adjustments. Patients will continue their usual care with their doctor during and after the study. The main outcome measured is the time taken to reach the maximum tolerated dose, with the goal of improving treatment management and reducing complications from sickle cell disease.
CONDITIONS
Brief Title
Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 2 and 35 years
- Severe sickle cell disease with HbSS genotype
- Hospitalized for vaso-occlusive crisis in last 3 months or requiring initiation or adjustment of hydroxyurea treatment
- For women of childbearing potential: negative pregnancy test and acceptance of effective contraception during and 182 days after study
- Informed consent signed by patient and/or legal guardians
- Affiliated with a social health insurance scheme
- Able to understand study objectives and risks
You will not qualify if you...
- Already achieved maximum tolerated dose of hydroxyurea or dosage above 35 mg/kg/day
- Refusal to use effective contraception during and after treatment
- Pregnancy or breastfeeding
- Severe liver or kidney impairment
- Low neutrophils (<1500/mm3) or low platelets (<80,000/mm3)
- Hemoglobin below 4.5 g/dL or low reticulocytes if hemoglobin below 9 g/dL
- Recent transfusion or erythropoietin treatment within 3 months
- HIV infection
- Unable to provide informed consent or follow medical follow-up
- Under guardianship, curatorship, or court protection
- Currently in an exclusion period from another study
- Concurrent participation in another drug study
- Planning pregnancy within 18 months after study start or treatment end
- Hypersensitivity to hydroxyurea or its components
- Signs of severe myelosuppression at screening
- Geographical, social, or psychological reasons preventing follow-up participation
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) including information, eligibility check, and consent signing
Duration - Approximately 15 months
Participants take hydroxyurea with dose adjustments based on their assigned group and monitoring results, including pharmacological monitoring and haematological tolerance assessments.
5 visits approximately at months 0, 3, 6, 9, and 12 with additional blood tests at 15 days after dose adjustments
Duration - After treatment until study completion
Participants continue to be followed by their referring physician every 3 months as part of usual care.
Regular consultations every 3 months in routine care settings
Trial Site Locations
Total: 1 location
1
Strasbourg University Hospital
Strasbourg, France, 67091
Actively Recruiting
Research Team
M
Mariem DRIDI
M
Myriam DURAND
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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