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iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children (iKanEat)

Primary Purpose

Feeding Behavior, Feeding Disorder of Infancy or Early Childhood

Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Megestrol Acetate
iKanEat Behavioral Intervention
Sponsored by
University of Kansas Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Feeding Behavior focused on measuring Gastrostomy Tube, G-Tube, Oral Food Refusal, Behavioral Intervention, Megestrol, Transition to Oral Feedings, Weaning

Eligibility Criteria

9 Months - 108 Months (Child)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

  1. Males and females aged 9 months to 9y0m at the time of consent.
  2. Able to obtain parental or legal guardian written informed consent from subjects as applicable by local laws and regulations.
  3. Subjects must have a G or G/J tube.
  4. Subjects must receive over 80% of their total daily calorie needs from a tube in order to be classified as tube dependent.
  5. 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.
  6. Subjects must possess the oral motor skills necessary for eating. Subjects must possess behavioral skills necessary for mealtimes.

Exclusion Criteria:

  1. Children receiving oral or inhaled steroids.
  2. 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).
  3. 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).
  4. Children of non-English speaking parents.

Sites / Locations

  • Rady Children's Hospital - San DiegoRecruiting
  • Arnold Palmer Hospital for ChildrenRecruiting
  • Riley Hospital for Children at IU HealthRecruiting
  • Boston Children's HospitalRecruiting
  • Children's Mercy HospitalRecruiting
  • Children's Hospital of PhiladelphiaRecruiting
  • Rhode Island HospitalRecruiting
  • Monroe Carell Jr. Children's Hospital at VanderbiltRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Megestrol

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Change in % kilocalories/day obtained orally
What percentage of kilocalories come from oral feedings as opposed to g-tube feeding.

Secondary Outcome Measures

Number of patients 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.
Change in parent stress as measured by the Pediatric Inventory for Parents (PIP36)
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.
Change in child quality of life as measured by the Infant Toddler Quality of Life scale (ITQOL47)
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).

Full Information

First Posted
January 17, 2019
Last Updated
May 15, 2023
Sponsor
University of Kansas Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT03815019
Brief Title
iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children
Acronym
iKanEat
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 15, 2019 (Actual)
Primary Completion Date
June 1, 2024 (Anticipated)
Study Completion Date
June 1, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Kansas Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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).
Detailed Description
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. Hypothesis 2A: Children randomized to the megestrol group will not differ from control children in morning cortisol classification level (low, average, high) and will remain within the normal range at all time points. Analyses 2B: Exploratory analysis will determine which, if any, covariates (gender, age, and diagnoses at week 0, diagnoses at birth) are related to abnormal morning cortisol levels. Aim 3: To examine the effect of the transition from tube to oral feeding on parent stress and parent and child quality of life. Hypothesis 3A: The transition to oral feeding will temporarily increase parent stress at week 14 at the cessation of tube feeding, with a return to baseline by week 24. Hypothesis 3B: The transition to oral feeding will increase parent/child quality of life at 24 weeks compared to week 0. Children with feeding tubes have few options for treatment other than extensive inpatient stays and expensive day treatment programs. iKanEat offers an outpatient, less intensive, empirically supported effective treatment option that can improve the lives of children and families. It is imperative that the investigators determine the efficacy and safety of the protocol including 4 weeks of megestrol before the investigators move toward broad dissemination of the iKanEat protocol.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Feeding Behavior, Feeding Disorder of Infancy or Early Childhood
Keywords
Gastrostomy Tube, G-Tube, Oral Food Refusal, Behavioral Intervention, Megestrol, Transition to Oral Feedings, Weaning

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Model Description
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).
Masking
ParticipantCare ProviderInvestigator
Masking Description
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.
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Megestrol
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Megestrol Acetate
Other Intervention Name(s)
Megace, Megestrol
Intervention Description
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.
Intervention Type
Behavioral
Intervention Name(s)
iKanEat Behavioral Intervention
Intervention Description
The 24 week iKanEat intervention is composed of 4 clinic visits and a series of 12 tele-visits. All visits (clinic and tele-visits) will focus directly on the oral-motor and behavioral skills necessary for oral eating. Tele-visits will begin by building rapport and asking for a summary of all relevant information since the last point of contact, including parent perception of changes in weight, feeding habits, progress, stress of parent/child, and illness. The majority of the time left in the 30 minute session will be spent dealing with parent concerns, which our previous project indicates may include questions about measures, questions about implementation of the iKanEat protocol, and ensuring that children/families adhere to the oral-motor and behavioral guidelines for feeding.
Primary Outcome Measure Information:
Title
Change in % kilocalories/day obtained orally
Description
What percentage of kilocalories come from oral feedings as opposed to g-tube feeding.
Time Frame
Weeks 0 to 24
Secondary Outcome Measure Information:
Title
Number of patients with adrenal insufficiency as measured by morning cortisol lab value
Description
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.
Time Frame
10 to 14 weeks
Title
Change in parent stress as measured by the Pediatric Inventory for Parents (PIP36)
Description
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.
Time Frame
Weeks 0 to 24
Title
Change in child quality of life as measured by the Infant Toddler Quality of Life scale (ITQOL47)
Description
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).
Time Frame
Week 0 to 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
9 Months
Maximum Age & Unit of Time
108 Months
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ann M Davis, PhD, MPH, ABPP
Phone
913-588-6323
Email
adavis6@kumc.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Kelsey M Dean, RD, CCRP
Phone
913-588-3775
Email
kdean@kumc.edu
Facility Information:
Facility Name
Rady Children's Hospital - San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Matthew Brigger, MD
Phone
858-309-7701
Email
mbrigger@ucsd.edu
Facility Name
Arnold Palmer Hospital for Children
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Devendra Mehta, MD
Phone
321-841-3338
Email
devendra.mehta@orlandohealth.com
Facility Name
Riley Hospital for Children at IU Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ryan Pitman, MD
Phone
317-944-3774
Email
rtpitman@iu.edu
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rachel Rosen, MD
Phone
617-355-6055
Email
Rachel.Rosen@childrens.harvard.edu
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sarah Edwards, DO
Phone
816-234-3000
Email
sedwards1@cmh.edu
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hayat Mousa, MD
Phone
215-913-3906
Email
MOUSAH@chop.edu
Facility Name
Rhode Island Hospital
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carolina S Cerezo, MD
Phone
401-444-8306
Email
ccerezo@lifespan.org
Facility Name
Monroe Carell Jr. Children's Hospital at Vanderbilt
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Julia Anderson, MD
Phone
615-322-7449
Email
julia.anderson@vumc.org

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
33640012
Citation
Edwards S, Hyman PE, Mousa H, Bruce A, Cocjin J, Dean K, Fleming K, Romine RS, Davis AM. iKanEat: protocol for a randomized controlled trial of megestrol as a component of a pediatric tube weaning protocol. Trials. 2021 Feb 27;22(1):169. doi: 10.1186/s13063-021-05131-w.
Results Reference
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iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children

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