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

addmedica

Status and phase

Completed

Phase 2

Conditions

Sickle Cell Disease

Treatments

Drug: Hydroxycarbamide

Study type

Interventional

Funder types

Industry

Identifiers

NCT05470270

SIK-FR-22-1

Details and patient eligibility

About

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).

Enrollment

33 patients

Sex

All

Ages

2 to 6 years old

Volunteers

No Healthy Volunteers

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.