Intranasal Oxytocin for Treatment of Infants and Children With Prader-Willi Syndrome in Nutritional Phase 1a - Phase 2 Study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
研究概览
简要总结
The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies will be performed on treatment day 1 and on the day following treatment morning of day 6.
详细描述
The overall objective of this Phase 2 trial is to compare the change from baseline to morning of day 6 of Internasal Oxytocin (IN-OT) on suck and swallow competency in infants/children with Prader-Willi Syndrome (PWS) who are in nutritional phase 1a.
Study Hypothesis 1: The Study team hypothesize that replacing Oxytocin (OT) in infants and children who are in nutritional phase 1a will improve their suck and swallow, potentially even eliminating the need for gastrostomy tubes and nasogastric tubes for feeding, and decreasing the risk of aspiration with oral feeding.
Study Hypothesis 2: The Study team hypothesize that replacing OT in infants and children with PWS will result in improved eye contact, daytime alertness, and feelings of bonding between the parents and the infant.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Double (Care Provider, Outcomes Assessor)
盲法说明
This will be a double-blinded study, as no studies to date have compared this treatment in infants with placebo. Board-certified speech and language pathologist who will be blinded to the treatment arm of each patient.
入排标准
- 年龄范围
- 1 Week 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI
- •Physical exam and laboratory results that are within the normal range.
- •Presence of a parent/caregiver/guardian that is able to consent for their participation.
排除标准
- •Exposure to any investigational agent in the 30 days prior to randomization.
- •Prior chronic treatment with oxytocin.
- •A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
研究组 & 干预措施
Intranasal oxytocin
Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.
干预措施: Oxytocin (Drug)
IN-placebo
Intervention: 4 IU/day of placebo via nasal spray device each morning.
干预措施: Placebo (Drug)
结局指标
主要结局
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
时间窗: baseline to day 5
Swallow study Overall improvement
次要结局
未报告次要终点