Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children with Sickle Cell Disease: a single-group, non-randomised, open-label study (KID-BID)

Prior treatment

• Patients treated with other SCD-modifying therapies

Other illness

• Patient with a stage 3, 4 or 5 chronic kidney disease (estimated glomerular filtration rate < 60 mL/min per 1.73 m2
• Patients known to be infected with human immunodeficiency virus, hepatitis B virus, or hepatitis C virus
• Known hypersensitivity or allergy to the excipients
• Any surgical or medical condition or any significant illness (of which severe hepatic impairment [Child-Pugh classification C], severe renal impairment, toxic ranges of myelosuppression) that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study
• Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months.

Other

• 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
• Female patients who are pregnant or lactating

• 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
• HC-naïve (absence of previous or current treatment with HC)
• Parent(s) or legally acceptable representative(s) capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements
• Contraception criterion, if applicable: for patients who are sexually active Female patients of childbearing potential (post menarche) at screening agreeing to use a highly effective form of contraception (oral, injected or implanted hormonal contraception, intrauterine device, o abstinence) during the trial and for 3 months after HC discontinuation. Male patients with partners of childbearing potential agreeing to use a highly effective contraception during the trial and for 3 months after HC discontinuation. Male patients with pregnant or lactating partners should be advised to use a barrier method of contraception (condom) to prevent the foetus or breastfed infant from exposure to HC.
• Affiliated to a social security plan (including universal health coverage) or beneficiary of a similar insurance plan
• Patient must meet the following laboratory values : Absolute Neutrophil Count ≥ 1,0x109/L, Platelets ≥ 75x109/L and Haemoglobin (Hgb) > 5,5 g/dL
• Transcranial Doppler (TCD) in the last 12 months indicating low risk for stroke is required for children over 18 months of age.