Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
- Registration Number
- NCT05470270
- Lead Sponsor
- Theravia
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 33
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description New formulation of hydroxycarbamide Hydroxycarbamide Single arm study with a single administration of hydroxycarbamide
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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)
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,
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,
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
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
Trial Locations
- Locations (3)
InterCommunal Hospital Centre of Creteil
🇫🇷Créteil, France
Necker University Hospital
🇫🇷Paris, France
Robert Debré Hospital
🇫🇷Paris, France