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

Primary Purpose

Sickle Cell Disease

Status

Completed

Phase

Phase 2

Locations

France

Study Type

Interventional

Intervention

Hydroxycarbamide

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring hydroxycarbamide, pediatric, acceptability

Eligibility Criteria

2 Years - 6 Years (Child)All SexesDoes not accept 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.

Sites / Locations

InterCommunal Hospital Centre of Creteil
Necker University Hospital
Robert Debré Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

New formulation of hydroxycarbamide

Arm Description

Single arm study with a single administration of hydroxycarbamide

Outcomes

Primary Outcome Measures

Acceptability score

Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)

Secondary Outcome Measures

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)

Distribution of the scores related to the ease of administration

Score reported by parent(s), based on a 5-point Likert scale,

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,

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

Free comments collected by the investigator

Questions from child/parent, reactions before/after drug intake

Number of adverse events

Number of adverse events and percentage of patients reporting at least one adverse event

Full Information

NCT ID

NCT05470270

First Posted

July 19, 2022

Last Updated

November 22, 2022

Sponsor

ADDMEDICA SASA

1. Study Identification

Unique Protocol Identification Number

NCT05470270

Brief Title

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

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Completed

Study Start Date

July 8, 2022 (Actual)

Primary Completion Date

October 28, 2022 (Actual)

Study Completion Date

October 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

ADDMEDICA SASA

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease

Keywords

hydroxycarbamide, pediatric, acceptability

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

33 (Actual)

8. Arms, Groups, and Interventions

Arm Title

New formulation of hydroxycarbamide

Arm Type

Experimental

Arm Description

Single arm study with a single administration of hydroxycarbamide

Intervention Type

Drug

Intervention Name(s)

Hydroxycarbamide

Intervention Description

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.

Primary Outcome Measure Information:

Title

Acceptability score

Description

Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)

Time Frame

At Day 1 (inclusion visit at study drug administration)

Secondary Outcome Measure Information:

Title

Percentage of children with acceptable acceptability score (neutral to positive scores)

Description

Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old)

Time Frame

At Day 1 (inclusion visit at study drug administration)

Title

Distribution of the scores related to the ease of administration

Description

Score reported by parent(s), based on a 5-point Likert scale,

Time Frame

At Day 1 (inclusion visit at study drug administration)

Title

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

Description

Score reported by the parent(s) based on a 5-point Likert scale,

Time Frame

At Day 1 (inclusion visit at study drug administration)

Title

Score related to the usefulness of the dispersible form, compared with the tablets currently used

Description

Score reported by the parent(s), based on a 5-point Likert scale

Time Frame

At Day 1 (inclusion visit at study drug administration)

Title

Free comments collected by the investigator

Description

Questions from child/parent, reactions before/after drug intake

Time Frame

At Day 1 (inclusion visit at study drug administration)

Title

Number of adverse events

Description

Number of adverse events and percentage of patients reporting at least one adverse event

Time Frame

At Day 1 (inclusion visit at study drug administration)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

6 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Bérengère Koel, MD

Organizational Affiliation

Hopital Universitaire Robert-Debre

Official's Role

Principal Investigator

Facility Information:

Facility Name

InterCommunal Hospital Centre of Creteil

City

Créteil

ZIP/Postal Code

94000

Country

France

Facility Name

Necker University Hospital

City

Paris

ZIP/Postal Code

75015

Country

France

Facility Name

Robert Debré Hospital

City

Paris

ZIP/Postal Code

75019

Country

France

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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