Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

Study Overview
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Brief Summary
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This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets).

Official Title
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An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: Hydroxycarbamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, Child with sickle cell disease, treated with 100 mg and/or 1000 mg Siklos® film-coated tablets at the same daily dose for more than 4 weeks, Child aged between 2 and 6 years old, Parents capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements, Children affiliated to a social security plan (including universal health coverage) or beneficiary of a similar insurance plan. Exclusion Criteria: Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding study inclusion, 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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: New formulation of hydroxycarbamide<br>Single arm study with a single administration of hydroxycarbamide | Drug: Hydroxycarbamide<br>* Single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Acceptability score | Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old) | At Day 1 (inclusion visit at study drug administration) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of children with acceptable acceptability score (neutral to positive scores) | Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old) | At Day 1 (inclusion visit at study drug administration) | 
| Distribution of the scores related to the ease of administration | Score reported by parent(s), based on a 5-point Likert scale, | At Day 1 (inclusion visit at study drug administration) | 
| Distribution of the scores related to the ease of preparation including the ease of constitution of the liquid form and the ease to follow the prescription | Score reported by the parent(s) based on a 5-point Likert scale, | At Day 1 (inclusion visit at study drug administration) | 
| Score related to the usefulness of the dispersible form, compared with the tablets currently used | Score reported by the parent(s), based on a 5-point Likert scale | At Day 1 (inclusion visit at study drug administration) | 
| Free comments collected by the investigator | Questions from child/parent, reactions before/after drug intake | At Day 1 (inclusion visit at study drug administration) | 
| Number of adverse events | Number of adverse events and percentage of patients reporting at least one adverse event | At Day 1 (inclusion visit at study drug administration) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hydroxycarbamide, pediatric, acceptability