iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children 9 Months to 9 Years 0 Months of Age
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 8
- 主要终点
- Participants Who Transitioned to Oral Feeding
研究概览
简要总结
This is a multi-center, randomized, placebo-controlled, double-blind clinical trial. The primary focus of the study is the evaluation of the effectiveness of treatment with megestrol as part of a 24 week behavioral feeding protocol in transitioning from tube to oral feedings in a pediatric population. Approximately 60 pediatric subjects matching the criteria for eligibility will be enrolled in the study and randomized to receive either megestrol (n=30) or placebo (n=30).
详细描述
The primary aim of the current study is to conduct a randomized controlled trial of a 4-week course of megestrol, the only remaining medication that is part of the iKanEat protocol, to ensure that the addition of megestrol results in improved child outcomes. The second aim is to assess the safety of megestrol as part of the iKanEat protocol. Our previous work (as well as work by others) suggests that a 6 week course of megestrol can lead to adrenal insufficiency in some children, so as part of the current protocol, the investigators will assess the safety of a 4 week course of this drug.
Finally, parents of tube fed children encounter multiple psychosocial stressors regarding tube feeding. These include concerns about their child's survival due to their underlying medical issues, feelings of "failure" due to their inability to feed their child orally, increased feelings of stress around the tube feeding and decreased support from others due to the tube feeding. Our research indicates that quality of life can be poor in tube fed children, even more so than children with cancer or burns. Given the significance of these issues, the third aim of the study is to examine the effect of the transition from tube to oral feeding on parent stress and parent and child quality of life.
Aim 1: To assess the efficacy of megestrol as part of the 24 week iKanEat protocol.
Hypothesis 1: Children randomized to the megestrol group will be significantly more successful in making the transition to oral feeding (defined as obtaining at least 90% of calories orally) than children randomized to the placebo group.
Aim 2: To assess the safety of 4 weeks of megestrol as part of the 24 week iKanEat protocol.
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
The drug will be dispensed by the investigational pharmacy at each site, who will be the only unblinded part of the team. They will receive the random codes prepared by the study statistician, and will dispense placebo/megestrol as appropriate.
入排标准
- 年龄范围
- 9 Months 至 108 Months（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Males and females aged 9 months to 9y0m at the time of consent.
- •Able to obtain parental or legal guardian written informed consent from subjects as applicable by local laws and regulations.
- •Subjects must have a G or G/J tube.
- •Subjects must receive over 80% of their total daily calorie needs from a tube in order to be classified as tube dependent.
- •Subjects must have a ≥ 3 month history of feeding problems as identified by a diagnosis from a multidisciplinary feeding team, and must have permission from the physician on the team to ensure that they are medically stable enough to participate in a weaning study.
- •Subjects must possess the oral motor skills necessary for eating. Subjects must possess behavioral skills necessary for mealtimes.
排除标准
- •Children receiving oral or inhaled steroids.
- •Parent has a known developmental delay or cognitive impairment that may make participation in the study difficult (children with these issues will not be excluded).
- •Children receiving intensive (defined as more than one session per month) behavioral feeding therapy with a licensed psychologist (previous behavioral feeding therapy is not an exclusion criterion; neither is current oral-motor, sensory, or speech therapy).
- •Children of non-English speaking parents.
研究组 & 干预措施
Megestrol
Megestrol is a steroid and progestational drug FDA approved for treating anorexia or weight loss in patients with acquired immunodeficiency syndrome. Its use in the current protocol is "off label" to stimulate appetite in tube-fed infants and toddlers who are weaning from tube feedings and learning to eat. The precise mechanism of action that leads to increased appetite and weight gain is unknown, but is probably related to megestrol's glucocorticoid effect.
The proposed study will use megestrol 6 mg/kg/day in two doses because this dose has been effective and safe in two previous studies using megestrol to stimulate appetite in children transitioning from tube to oral feedings. The megestrol will be dosed at full dose weeks 10-11, at 66% dose week 12, at 33% dose week 14, and fully tapered at the end of week 14. Megestrol is absorbed from the small bowel, so feeding it through the tube will be acceptable.
干预措施: Megestrol Acetate (Drug)
Megestrol
Megestrol is a steroid and progestational drug FDA approved for treating anorexia or weight loss in patients with acquired immunodeficiency syndrome. Its use in the current protocol is "off label" to stimulate appetite in tube-fed infants and toddlers who are weaning from tube feedings and learning to eat. The precise mechanism of action that leads to increased appetite and weight gain is unknown, but is probably related to megestrol's glucocorticoid effect.
The proposed study will use megestrol 6 mg/kg/day in two doses because this dose has been effective and safe in two previous studies using megestrol to stimulate appetite in children transitioning from tube to oral feedings. The megestrol will be dosed at full dose weeks 10-11, at 66% dose week 12, at 33% dose week 14, and fully tapered at the end of week 14. Megestrol is absorbed from the small bowel, so feeding it through the tube will be acceptable.
干预措施: iKanEat Behavioral Intervention (Behavioral)
Placebo
Subjects randomized to the placebo protocol will receive a placebo syrup identical in taste and smell to megestrol at the same intervals as those in the megestrol group but the syrup will contain no active ingredients.
干预措施: iKanEat Behavioral Intervention (Behavioral)
结局指标
主要结局
Participants Who Transitioned to Oral Feeding
时间窗: Weeks 0 to 24
The efficacy of megestrol as part of the 24 week iKanEat protocol will be measured by participants who successfully transitioned to oral feeding, defined at at least 90% calories consumed orally.
次要结局
- Number of Participants With Adrenal Insufficiency as Measured by Morning Cortisol Lab Value(10 to 14 weeks)
- Change in Child Quality of Life as Measured by the Infant Toddler Quality of Life Scale (ITQOL47) - Global Behavior (GB2) Subscale(Week 0 to 24)
- Change in Child Quality of Life as Measured by the Infant Toddler Quality of Life Scale (ITQOL47) - Combined Behavior Scale (CBE) Subscale(Week 0 to 24)
- Change in Child Quality of Life as Measured by the Child Health Questionnaire Parent Form - CHQ-PF50, 5.1 Behavior (BE) Subscale(Week 0 to 24)
- Change in Child Quality of Life as Measured by the Child Health Questionnaire Parent Form - CHQ-PF50 - 5.2 Global Behavior (GBE) Subscale(Week 0 to 24)
- Change in Parent Stress as Measured by the Pediatric Inventory for Parents (PIP36) - Total Frequency Score(Weeks 0 to 24)
- Change in Parent Stress as Measured by the Pediatric Inventory for Parents (PIP36) - Total Difficulty Score(Weeks 0 to 24)
- Change in Parent Anxiety Symptoms Measured by the General Anxiety Disorder-7 (GAD-7).(Week 0 to 24)
- Change in Parent Quality of Life as Measured by the SF-36v2 - Emotional Well-Being Subscale(Week 0 to 24)
- Change in Parent Depressive Symptoms Measured by the Patient Health Questionnaire-9 (PHQ-9)(Week 0 to 24)
研究者
Ann Davis, PhD, MPH, ABPP
Professor
University of Kansas Medical Center