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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
33
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.
InterCommunal Hospital Centre of Creteil
Créteil, France
Necker University Hospital
Paris, France
Robert Debré Hospital
Paris, France
Acceptability score
Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)
Time frame: At Day 1 (inclusion visit at study drug administration)
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)
Time frame: 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,
Time frame: 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,
Time frame: 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
Time frame: At Day 1 (inclusion visit at study drug administration)
Free comments collected by the investigator
Questions from child/parent, reactions before/after drug intake
Time frame: At Day 1 (inclusion visit at study drug administration)
Number of adverse events
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Number of adverse events and percentage of patients reporting at least one adverse event
Time frame: At Day 1 (inclusion visit at study drug administration)