Sickle Cell Disease
Conditions
Keywords
hydroxycarbamide, pediatric, acceptability
Brief summary
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).
Interventions
DRUGHydroxycarbamide
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.
Sponsors
Theravia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 6 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Acceptability score | At Day 1 (inclusion visit at study drug administration) | Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Distribution of the scores related to the ease of administration | At Day 1 (inclusion visit at study drug administration) | Score reported by parent(s), based on a 5-point Likert scale, |
| 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 | At Day 1 (inclusion visit at study drug administration) | Score reported by the parent(s) based on a 5-point Likert scale, |
| Percentage of children with acceptable acceptability score (neutral to positive scores) | At Day 1 (inclusion visit at study drug administration) | Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old) |
| Free comments collected by the investigator | At Day 1 (inclusion visit at study drug administration) | Questions from child/parent, reactions before/after drug intake |
| Number of adverse events | At Day 1 (inclusion visit at study drug administration) | Number of adverse events and percentage of patients reporting at least one adverse event |
| Score related to the usefulness of the dispersible form, compared with the tablets currently used | At Day 1 (inclusion visit at study drug administration) | Score reported by the parent(s), based on a 5-point Likert scale |
Countries
France
Outcome results
None listed