The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age.
Participants will:
* Take Hydroxycarbamide twice a day every day for 12 months * Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months
Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent from the children,
HbSS or HbSβ0 SCD,
Aged between 9 months and 11 years old,
Hydroxycarbamide naïve,
Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding the inclusion visit,
Patients who have had chronic blood transfusion or transfusion in the last 3 months preceding the inclusion visit,
Patients treated with other SCD-modifying therapies,
Patient with a stage 3, 4 or 5 chronic kidney disease,
Single group
Hydroxycarbamid
DrugHydroxycarbamide Paediatric dispersible tablets will be provided in the form of film-coated dispersible tablets containing 50 mg of hydroxycarbamide. The IMP will be administered as half-strength twice daily, based on the body weight of the patient.
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax)
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax)
Absolute mean change from baseline in HbF levels
HC plasma concentrations and HbF levels
Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model
Proportion of patients with a relative difference in Cmax ≥ 30% from BID maintenance dose relative to the one simulated on a once daily regimen giving an equivalent AUC0-24h.
