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Optimization of the management of drepanocytosis patients treated with hydroxyurea: Interest of the pharmacological therapeutic follow-up

Status
Completed
Phases
Phase 4
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-515562-14-00
Enrollment
30
Registered
2024-07-19
Start date
2024-07-19
Completion date
2025-10-27
Last updated
2024-07-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Drepanocytosis

Brief summary

Proportion of subjects with a time to reach LMA of less than 9 months

Detailed description

Clinical parameters of efficacy in both arms: has. a. Number of vaso-occlusive seizures b. Number of complications related to sickle cell disease and/or hydroxyurea c. Number of hospitalizations d. Time elapsed before the need for transfusion e. Percentage of HbF, Biological parameters of toxicity in both arms: has. a. Complete blood count, reticulocytes, ferritin b. Renal function (glomerular filtration rate estimated by cystatin C, plasma creatinine and urea) c. Liver function (AST, ALT, total and conjugated bilirubin)., Non-compliance parameters: has. a. Mean blood cell volume (MCV) b. Percentage of HbF, Population pharmacokinetic parameters of the 2 arms: a. AUC b. Clearance and volume of distribution of the drug., Pharmacokinetic analysis: has. a. Pharmacokinetic modeling of the population and identification of parameters involved in the inter- and intra-individual variability of the pharmacokinetics of the HU and allowing to better predict the individual dosage adaptation from our population study: i. Renal function ii. Age iii. Weight iv. Body surface v. % HbF b.Correlation between the concentrations obtained at the different sampling times and the AUC

Interventions

DRUGHYDREA 500 mg
DRUGgélule
DRUGSiklos 100 mg film-coated tablet.

Sponsors

Les Hopitaux Universitaires De Strasbourg
Lead SponsorOTHER

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Proportion of subjects with a time to reach LMA of less than 9 months

Secondary

MeasureTime frame
Clinical parameters of efficacy in both arms: has. a. Number of vaso-occlusive seizures b. Number of complications related to sickle cell disease and/or hydroxyurea c. Number of hospitalizations d. Time elapsed before the need for transfusion e. Percentage of HbF, Biological parameters of toxicity in both arms: has. a. Complete blood count, reticulocytes, ferritin b. Renal function (glomerular filtration rate estimated by cystatin C, plasma creatinine and urea) c. Liver function (AST, ALT, total and conjugated bilirubin)., Non-compliance parameters: has. a. Mean blood cell volume (MCV) b. Percentage of HbF, Population pharmacokinetic parameters of the 2 arms: a. AUC b. Clearance and volume of distribution of the drug., Pharmacokinetic analysis: has. a. Pharmacokinetic modeling of the population and identification of parameters involved in the inter- and intra-individual variability of the pharmacokinetics of the HU and allowing to better predict the individual dosage adaptation from ou

Countries

France

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026