Oxytocin Trial in Prader-Willi Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 3
- 主要终点
- Safety of intranasal oxytocin in children with Prader-Willi syndrome
研究概览
简要总结
Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).
The research questions are:
- Does intranasal oxytocin cause any side effects in children with PWS?
- Does intranasal oxytocin administration alter appetite or behaviors in PWS?
详细描述
This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children with genetically confirmed PWS
- •Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
- •Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
- •Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
- •Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
- •Priority will be given to children currently enrolled in the RDCRN Natural History study
排除标准
- •Inability to tolerate intranasal administration of medication
- •Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
- •Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
- •History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
- •Child not receiving growth hormone treatment
- •Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)
- •Diabetes mellitus
- •Pregnant or lactating.
- •Schizophrenia or psychosis
- •Taking any psychotropic medications
研究组 & 干预措施
Intranasal oxytocin
Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.
干预措施: Intranasal oxytocin (Drug)
Placebo
Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.
干预措施: Placebo (Other)
结局指标
主要结局
Safety of intranasal oxytocin in children with Prader-Willi syndrome
时间窗: 3 months
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
次要结局
- Evaluation of food intake in Prader-Willi syndrome(3 months)