| Primary | Participants Who Transitioned to Oral Feeding | 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. | | Posted | | Number | | Participants | | Weeks 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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. |
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| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
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| Analysis conducted using Generalized Linear Mixed Models with a logit link within SAS Proc GLIMMIX to model the binary outcome of transitioned to oral feeding (y/n) defined as at least 90 percent of calories consumed orally. Fixed effects (dummy variables) will be entered for each site as described by McNeish and Stapleton (2016) to account for the clustering of the participants within sites. This will result in essentially, a logistic regression model with appropriate standard errors. | | | | | Odds Ratio, log | 7.2218 | Standard Error of the Mean | 13083 | 2-Sided | | | | | | Participants in the megestrol group who successfully transitioned to oral feeding. | | |
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| Secondary | Number of Participants With Adrenal Insufficiency as Measured by Morning Cortisol Lab Value | The investigators will determine whether any of our participants in either group have adrenal sufficiency levels outside of the normal range at any time point. Should any participant have levels outside of the normal range, the investigators will descriptively examine the individual characteristics associated with this, including group assignment, medical diagnoses, sex, and age at initiation. These characteristics will be reported descriptively. | | Posted | | Count of Participants | | Participants | | 10 to 14 weeks | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | Placebo |
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| Secondary | Change in Child Quality of Life as Measured by the Infant Toddler Quality of Life Scale (ITQOL47) - Global Behavior (GB2) Subscale | The investigators will model child quality of life from Week 0 to Week 24 of the intervention. The Infant Toddler Quality of Life Questionnaire™ (ITQOL) was developed for use in infants and toddlers from 2-months-to-5 years of age. The Infant Toddler Quality of Life Questionnaire™ (ITQOL) adopts the World Health Organization's definition of health, as a state of complete physical, mental and social wellbeing and not merely the absence of disease. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Child Quality of Life as Measured by the Infant Toddler Quality of Life Scale (ITQOL47) - Combined Behavior Scale (CBE) Subscale | he investigators will model child quality of life from Week 0 to Week 24 of the intervention. The Infant Toddler Quality of Life Questionnaire™ (ITQOL) was developed for use in infants and toddlers from 2-months-to-5 years of age. The Infant Toddler Quality of Life Questionnaire™ (ITQOL) adopts the World Health Organization's definition of health, as a state of complete physical, mental and social wellbeing and not merely the absence of disease. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). | | Posted | | Least Squares Mean | Standard Error | scores on scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Child Quality of Life as Measured by the Child Health Questionnaire Parent Form - CHQ-PF50, 5.1 Behavior (BE) Subscale | Child quality of life in children aged ≥5 years will be assessed using the Child Health Questionnaire Parent Form - CHQ-PF50, a validated parent-reported measure of physical and psychosocial health for children ages 5-18. The CHQ-PF50 contains 50 items assessing 14 health concepts, including physical functioning, emotional/behavioral well-being, social functioning, and family impact. The Behavior (BE) subscale evaluates behavioral and emotional adjustment as observed by the parent. Subscale scores range from 0 to 100, with higher scores representing better behavioral health and fewer behavioral problems. CHQ-PF50 scores can be analyzed at the subscale level (CHQ Profile Scores) or combined into Summary Scores for physical and psychosocial health. Summary scores are calculated by averaging standardized subscale scores, also ranging from 0 to 100, with higher scores indicating better overall health-related quality of life. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Child Quality of Life as Measured by the Child Health Questionnaire Parent Form - CHQ-PF50 - 5.2 Global Behavior (GBE) Subscale | Child quality of life in children aged ≥5 years will be assessed using the Child Health Questionnaire Parent Form - CHQ-PF50, a validated parent-reported measure of physical and psychosocial health for children ages 5-18. The CHQ-PF50 includes 50 items covering 14 health concepts, such as physical functioning, emotional/behavioral well-being, social functioning, and family impact. The Global Behavior (GBE) subscale assesses overall behavioral and emotional functioning as reported by the parent. Subscale scores range from 0 to 100, with higher scores indicating better global behavioral health and fewer overall behavioral problems. CHQ-PF50 scores can be analyzed at the subscale level (CHQ Profile Scores) or combined into Summary Scores for physical and psychosocial health. Summary scores are calculated by averaging standardized subscale scores, also ranging from 0 to 100, with higher scores representing better overall health-related quality of life. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Parent Stress as Measured by the Pediatric Inventory for Parents (PIP36) - Total Frequency Score | The investigators will model parent stress levels from Week 0 to Week 24 of the intervention. The PIP is scored separately for each of the 4 domains (Communication, Emotional Distress, Medical Care, Role Function), across 2 scales: Frequency (F) and Difficulty (D). There is also a total score comprised of the sum for each of the 4 domains, yielding Total F and Total D scores. Items are scored as endorsed by respondents, ranging from 1-5. The range for each of the Total F and Total D scores is 42-210. Higher scores are indicative of worse parent stress outcomes. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Weeks 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Parent Stress as Measured by the Pediatric Inventory for Parents (PIP36) - Total Difficulty Score | The investigators will model parent stress levels from Week 0 to Week 24 of the intervention. The PIP is scored separately for each of the 4 domains (Communication, Emotional Distress, Medical Care, Role Function), across 2 scales: Frequency (F) and Difficulty (D). There is also a total score comprised of the sum for each of the 4 domains, yielding Total F and Total D scores. Items are scored as endorsed by respondents, ranging from 1-5. The range for each of the Total F and Total D scores is 42-210. Higher scores are indicative of worse parent stress outcomes. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Weeks 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Parent Quality of Life as Measured by the SF-36v2 - Emotional Well-Being Subscale | Parent quality of life will be assessed using the Medical Outcomes Study 36-Item Short Form Health Survey (SF-36v2), a widely used, validated self-report measure of general health-related quality of life. The SF-36v2 includes 36 items covering 8 domains: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health. The Emotional Well-Being (Mental Health) subscale measures overall emotional functioning, including mood, anxiety, and psychological distress. Raw subscale scores range from 0 to 100, with higher scores indicating better emotional well-being. For analysis, raw subscale scores will be converted to standardized Z-scores, where a Z-score of 0 represents the population mean, and each standard deviation above or below 0 represents deviation from the mean. Higher Z-scores indicate better emotional functioning, while lower Z-scores indicate worse emotional | | Posted | | Least Squares Mean | Standard Error | z-score | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Parent Depressive Symptoms Measured by the Patient Health Questionnaire-9 (PHQ-9) | Parent depressive symptoms will be assessed using the Patient Health Questionnaire-9 (PHQ-9), a validated 9-item self-report measure of depression severity based on the 9 criteria for Major Depressive Disorder (MDD) from the DSM. Each item is scored from 0 ("not at all") to 3 ("nearly every day"), yielding a total score ranging from 0 to 27. Higher PHQ-9 scores indicate greater severity of depressive symptoms, while lower scores indicate fewer or less severe depressive symptoms. Total score interpretation is commonly as follows: 0-4 = minimal or no depression, 5-9 = mild depression, 10-14 = moderate depression, 15-19 = moderately severe depression, 20-27 = severe depression. This measure can be used to assess both the presence of depressive symptoms and changes in symptom severity over time. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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| Secondary | Change in Parent Anxiety Symptoms Measured by the General Anxiety Disorder-7 (GAD-7). | Parent anxiety symptoms will be assessed using the Generalized Anxiety Disorder-7 (GAD-7), a validated 7-item self-report measure that screens for and assesses the severity of common anxiety disorders, including Generalized Anxiety Disorder, Panic Disorder, Social Phobia, and Post-Traumatic Stress Disorder. Each item is scored from 0 ("not at all") to 3 ("nearly every day"), yielding a total score range of 0 to 21. Higher scores indicate greater anxiety symptom severity, whereas lower scores indicate fewer or less severe anxiety symptoms. Total scores are interpreted as follows: 0-4 = minimal anxiety, 5-9 = mild anxiety, 10-14 = moderate anxiety, 15-21 = severe anxiety. The total GAD-7 score is calculated by summing the scores for all seven items. This measure can be used to assess the presence of clinically significant anxiety symptoms as well as changes in symptom severity over time. | | Posted | | Least Squares Mean | Standard Error | scores on a scale | | Week 0 to 24 | | | | ID | Title | Description |
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| OG000 | 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. |
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