Skip to main content
OpenTrials
Completed

NCT Number: NCT05470270

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

2 year–6 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

InterCommunal Hospital Centre of Creteil, Créteil, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. Acceptability score

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

Secondary outcomes

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

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

  2. Distribution of the scores related to the ease of administration

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

  3. 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

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

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

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

  5. Free comments collected by the investigator

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

  6. Number of adverse events

    Time frame: At Day 1 (inclusion visit at study drug administration)

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

Sponsors and collaborators

Lead sponsor

Theravia

Industry

Registry information

Official study 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.

Important dates

Study start
2022
Primary completion
2022
Study completion
2022
First posted
Jul 22, 2022
Registry last updated
Nov 23, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.