NCT05298085已完成2 期
Effect of Intranasal Oxytocin on Dysphagia Related to Oropharyngo-oesophageal Dysmotility in Children and Adolescents With Prader-Willi Syndrome: a Phase 2B Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 4
- 主要终点
- videofluoroscopic swallowing study (VFSS) score change
研究概览
简要总结
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS).
This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS patients aged 2-17 years and 5 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients between 2 years and 17 years and 5 months at inclusion.
- •Genetically confirmed diagnosis of PWS.
- •Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.
排除标准
- •A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex;
- •Intolerance of intranasal administrations (including when due to a major behavioural problem);
- •Hyponatremia (clinically relevant at the discretion of the investigator);
- •Hypokalaemia (clinically relevant at the discretion of the investigator);
- •Prolongation of the QT interval and/or family history of prolongation of the QT interval;
- •Concomitant treatment prolonging the QT interval;
- •Start of growth hormone (GH) treatment within the last 4 weeks before inclusion;
- •History of abnormal electrocardiogram (ECG) (validated by a cardiologist);
- •Pregnant girls; (for girls with childbearing potential who do not have contraception and are sexually active, a negative pregnancy test will be required)
- •Patient with clinical signs in the context of contact with COVID-19 infected person.
- •Patient included in another study protocol on a medicinal product within the last 6 months;
- •Administrative problems:
- •Inability to give parents (or legal representatives) expert medical information;
- •No coverage by a social security regime.
研究组 & 干预措施
OXYTOCIN nasal spray
Experimental
intranasal administration of Oxytocin
干预措施: Oxytocin nasal spray (Drug)
PLACEBO
Placebo Comparator
intranasal administration of placebo
干预措施: Placebo (Drug)
结局指标
主要结局
videofluoroscopic swallowing study (VFSS) score change
时间窗: after 12 weeks oxytocin (OT) / placebo (at V2)
Percentage of patient with at least one VFSS subscore's change from baseline
次要结局
未报告次要终点
研究者
研究点 (4)
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