Clinical trial NCT05470270: Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
Official title: An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: hydroxycarbamide
pediatric
acceptability
Lead sponsor: Theravia
Sponsor class: INDUSTRY
First posted: 2022-07-22
Enrollment: 33
Sex: ALL
Minimum age (years): 2
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility 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® 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.

Primary outcomes:
measure: Acceptability score
timeFrame: At Day 1 (inclusion visit at study drug administration)
Secondary outcomes:
measure: Percentage of children with acceptable acceptability score (neutral to positive scores)
timeFrame: At Day 1 (inclusion visit at study drug administration)
measure: Distribution of the scores related to the ease of administration
timeFrame: At Day 1 (inclusion visit at study drug administration)
measure: 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
timeFrame: At Day 1 (inclusion visit at study drug administration)
measure: Score related to the usefulness of the dispersible form, compared with the tablets currently used
timeFrame: At Day 1 (inclusion visit at study drug administration)
measure: Free comments collected by the investigator
timeFrame: At Day 1 (inclusion visit at study drug administration)
measure: Number of adverse events
timeFrame: At Day 1 (inclusion visit at study drug administration)
Other outcomes:
Locations:
city: Créteil
country: France
facility: InterCommunal Hospital Centre of Creteil
city: Paris
country: France
facility: Necker University Hospital
city: Paris
country: France
facility: Robert Debré Hospital
Study officials:
affiliation: Hopital Universitaire Robert-Debre
name: Bérengère Koel, MD
role: PRINCIPAL_INVESTIGATOR
References: