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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

33 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-08

Study Completion Date

2022-10-28

Brief Summary

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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).

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Detailed Description

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Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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New formulation of hydroxycarbamide

Single arm study with a single administration of hydroxycarbamide

Group Type EXPERIMENTAL

Hydroxycarbamide

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No