Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

Sickle Cell Disease Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05470270
• Other study ID #: SIK-FR-22-1
• Status: Completed
• Phase: Phase 2
• Start date: July 8, 2022
• Est. completion date: October 28, 2022

Study information

• Verified date: November 2022
• Source: ADDMEDICA SASA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 33
• Est. completion date: October 28, 2022
• Est. primary completion date: October 28, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 6 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Hydroxycarbamide
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.

Locations

Country	Name	City	State
France	InterCommunal Hospital Centre of Creteil	Créteil
France	Necker University Hospital	Paris
France	Robert Debré Hospital	Paris

Sponsors (1)

Lead Sponsor	Collaborator
ADDMEDICA SASA

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Acceptability score	Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)	At Day 1 (inclusion visit at study drug administration)
Secondary	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)	At Day 1 (inclusion visit at study drug administration)
Secondary	Distribution of the scores related to the ease of administration	Score reported by parent(s), based on a 5-point Likert scale,	At Day 1 (inclusion visit at study drug administration)
Secondary	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,	At Day 1 (inclusion visit at study drug administration)
Secondary	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	At Day 1 (inclusion visit at study drug administration)
Secondary	Free comments collected by the investigator	Questions from child/parent, reactions before/after drug intake	At Day 1 (inclusion visit at study drug administration)
Secondary	Number of adverse events	Number of adverse events and percentage of patients reporting at least one adverse event	At Day 1 (inclusion visit at study drug administration)