NCT05470270已完成2 期
An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Theravia
- 入组人数
- 33
- 试验地点
- 3
- 主要终点
- Acceptability score
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
New formulation of hydroxycarbamide
Experimental
Single arm study with a single administration of hydroxycarbamide
干预措施: Hydroxycarbamide (Drug)
结局指标
主要结局
Acceptability score
时间窗: 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)
次要结局
- 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 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))
- Distribution of the scores related to the ease of administration(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))
- Number of adverse events(At Day 1 (inclusion visit at study drug administration))
研究者
研究点 (3)
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