Effects of Intranasal Administrations of Oxytocin on Behavioural Troubles, Hyperphagia and Social Skills in Children With Prader-Willi Syndrome Aged From 3 to 12 Years.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment.
研究概览
简要总结
Positive results in preclinical and clinical studies in adults and infants with Prader-Willi syndrome lead investigators to set up a new study in children with Prader-Willi syndrome. The objective of this study is to document effects of oxytocin intranasal administrations on behavioural troubles in children with Prader-Willi syndrome aged from 3 to 12 years.
详细描述
Two groups of patients will be constituted according to their age; "Group 1" children aged from 3 to 6 years (n = 20) and "Group 2" children aged from 7 to 12 years (n = 20). Within each group, subjects will be randomized to receive either oxytocin or placebo for 12 consecutive weeks. A second period of 12 consecutive oxytocin treatment weeks will then be performed for all patients, followed by a 4-week observation period to document effects after discontinuation of treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
The packaging, presentation and labelling of the bottles used during the blind phase will guarantee the blindness to the healthcare team and the patient.
The allocation of the treatment arms to each treatment number will be carried out according to the randomization table established beforehand.
Until the end of the study, neither the investigating physician nor the patient will know the group to which the patient has been randomly assigned.
入排标准
- 年龄范围
- 3 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patient with a complete genetic diagnosis of Prader-Willi syndrome
- •patient treated by growth hormone for at least 1 year
- •patient naïve for oxytocin for at least 5 years
排除标准
- •patient who do not accept intranasal administrations (major behavioural trouble)
- •patient with hepatic insufficiency : serum transaminases (SGOT, SGPT) higher than 3 times normal values for age
- •patient with renal insufficiency : serum creatinine higher than 3 times normal values for age
- •patient with an antecedent of abnormal electrocardiogram
- •patient with arterial hypertension or hypotension
- •patient with type 1 or 2 diabetes
研究组 & 干预措施
Oxytocin
Daily intranasal administrations of oxytocin for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
干预措施: Oxytocin (Drug)
Placebo
Daily intranasal administrations of placebo for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
干预措施: Placebo (Drug)
Placebo
Daily intranasal administrations of placebo for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
干预措施: Oxytocin (Drug)
结局指标
主要结局
Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment.
时间窗: Week 12
It's variation between inclusion and 12 weeks of total score total problems from the Child Behavior Check List Questionnaire.
次要结局
- Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment.(Week 12)
- Evaluation of social skills after 12 weeks of oxytocin/placebo treatment.(Week 12)
- Evaluation of auto- and hetero-aggressive after 12 weeks of oxytocin/placebo treatment.(Week 12)
- Evaluation of psychopathology after 12 weeks of oxytocin/placebo treatment.(Week 12)
- Evaluation of global clinical status after 12 weeks of oxytocin/placebo treatment.(Weeks 12)
- Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment.(Week 12)
- Evaluation of attentional abilities after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.(Week 12)
- Evaluation of metabolic brain resting state after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.(Week 12)