💬In plain words
What the trial is, and whether it could suit you
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
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
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.
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