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

An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

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).

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Hydroxycarbamide

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Créteil, France, 94000
    • InterCommunal Hospital Centre of Creteil
  • Paris, France, 75019
    • Robert Debre Hospital
  • Paris, France, 75015
    • Necker University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 6 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: New formulation of hydroxycarbamide; Single arm study with a single administration of hydroxycarbamide	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)
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)
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)
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)
Free comments collected by the investigator	Questions from child/parent, reactions before/after drug intake	At Day 1 (inclusion visit at study drug administration)
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)

Collaborators and Investigators

• Sponsor: ADDMEDICA SASA
• Investigators: Principal Investigator: Bérengère Koel, MD, Hôpital Universitaire Robert-Debré

Study record dates

Study Major Dates

• Study Start (Actual): July 8, 2022
• Primary Completion (Actual): October 28, 2022
• Study Completion (Actual): October 28, 2022

Study Registration Dates

• First Submitted: July 19, 2022
• First Submitted That Met QC Criteria: July 21, 2022
• First Posted (Actual): July 22, 2022

Study Record Updates

• Last Update Posted (Actual): November 23, 2022
• Last Update Submitted That Met QC Criteria: November 22, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Keywords: pediatric; acceptability; hydroxycarbamide
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Molecular Mechanisms of Pharmacological Action; Nucleic Acid Synthesis Inhibitors; Enzyme Inhibitors; Antineoplastic Agents; Antisickling Agents; Hydroxyurea
• Other Study ID Numbers: SIK-FR-22-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No