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

NCT05470270 | Completed Phase 2

• 1 other identifier: SIK-FR-22-1
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 3

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).

Conditions

Sickle Cell Disease

Keywords

hydroxycarbamide; pediatric; acceptability

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

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

  At Day 1 (inclusion visit at study drug administration)

• Distribution of the scores related to the ease of administration

  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

  At Day 1 (inclusion visit at study drug administration)

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

  At Day 1 (inclusion visit at study drug administration)

• Free comments collected by the investigator

  At Day 1 (inclusion visit at study drug administration)

Study Arms (1)

New formulation of hydroxycarbamide

EXPERIMENTAL

Single arm study with a single administration of hydroxycarbamide

Drug: Hydroxycarbamide

Interventions

Hydroxycarbamide 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.

New formulation of hydroxycarbamide

Eligibility Criteria

• Age: 2 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Theravia: lead

Study Sites (3)

InterCommunal Hospital Centre of Creteil

Créteil, 94000, France

Location

Necker University Hospital

Paris, 75015, France

Location

Robert Debré Hospital

Paris, 75019, France

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Study Officials

• Bérengère Koel, MD

  Hopital Universitaire Robert-Debre

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 19, 2022
• First Posted: July 22, 2022
• Study Start: July 8, 2022
• Primary Completion: October 28, 2022
• Study Completion: October 28, 2022
• Last Updated: November 23, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3)