Drepanocytosis
Conditions
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
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of subjects with a time to reach LMA of less than 9 months | — |
Secondary
| Measure | Time 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