Drepanocytosis MedDRA version: 21.0 Level: LLT Classification code 10051835 Term: Drepanocytosis System Organ Class: 100000004850
Conditions
Interventions
Product Name: Hydroxycarbamide
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: HYDROXYCARBAMIDE
CAS Number: 127-07-01
Concentration unit: mg milligram(s)
Concentration type: equal
Concent
Sponsors
Addmedica
Eligibility
Sex/Gender
All
Inclusion criteria
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® filmcoated 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. Are the trial subjects under 18? yes Number of subjects for this age range: 45 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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 |
|---|---|
| Main Objective: The primary objective is to evaluate the acceptability of the hydroxycarbamide 50 mg film-coated dispersible tablets in 2- to 6- year old children with sickle cell disease.;Secondary Objective: • To calculate the proportion of children with an acceptable acceptability score (neutral to positive scores), • To evaluate the ease of administration of the hydroxycarbamide dispersible tablets, reported by the parent(s), • To evaluate the ease of preparation of the solution reported by the parent(s), • To assess the usefulness of the new form (hydroxycarbamide dispersible tablets) compared with the form currently used (Siklos® 100 mg and 1000 mg film-coated tablets), • To collect free comments (collection by the investigator) about the study product and administration made by the child/parent(s), • To assess the safety profile of the hydroxycarbamide dispersible tablets.;Primary end point(s): The acceptability score reported by the parent(s) of the child (2-6 years old) on a 5-point Likert scale and reported by the child (4-6 years old) on a 5-point hedonic scale will be summarised using descriptive statistics.;Timepoint(s) of evaluation of this end point: Visit inclusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • A neutral to positive acceptability score (scores 3-5) will be considered as acceptable. The percentages of children with acceptable acceptability scores, reported by the child (based on a 5-point hedonic scale) and reported by the parent (based on a 5-point Likert scale), will be calculated. A value of 80% of the sample population in agreement that the product is acceptable is generally considered to be the current standard requirement (8). • Distribution of the scores related to the ease of administration, reported by parent(s), based on a 5-point Likert scale, will be analysed in the FAS and in the PPS. • 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 (calculation of the number of tablets), reported by the parent(s) based on a 5-point Likert scale, will be analysed in the FAS and in the PPS. • Score related to the usefulness of the hydroxycarbamide dispersible form, reported by the parent(s), based on a 5-point Likert scale (when asked to compare it with the usual Siklos® tablets) will be analysed in the FAS and in the PPS. • Free comments, reactions before/after drug intake and questions by the child and the parents will be displayed in a listing of comments and not further analysed statistically. • Safety analysis: the frequency and severity of AEs will be analysed in the SS using descriptive statistics and displayed by system organ class (SOC) and preferred term (PT) according to the Medical Dictionary for Regulatory Activities (MedDRA) terminology.;Timepoint(s) of evaluation of this end point: Visit inclusion | — |
Countries
France
Contacts
Public ContactLaura Fabre Thomas-Bourgneuf
ADDMEDICA
Outcome results
None listed