Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

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)

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

Inclusion Criteria:

• 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,
• 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
• Affiliated to a social security plan 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.

Exclusion Criteria:

• 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,
• 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 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,
• Female patients who are pregnant or lactating,
• Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months.