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

NCT03815019 | Completed Phase 4 Results DMC FDA Drug

• 1 other identifier: STUDY00142352
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 8

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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.

  Weeks 0 to 24

Secondary Outcomes (10)

• 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

Study Arms (2)

Megestrol

EXPERIMENTAL

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.

Drug: Megestrol Acetate; Behavioral: iKanEat Behavioral Intervention

Placebo

PLACEBO COMPARATOR

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.

Behavioral: iKanEat Behavioral Intervention

Interventions

Megestrol Acetate DRUG

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.

Also known as: Megace, Megestrol

Megestrol

iKanEat Behavioral Intervention BEHAVIORAL

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.

Megestrol; Placebo

Eligibility Criteria

• Age: 9 Months - 108 Months
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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 of non-English speaking parents.

Sponsors & Collaborators

• University of Kansas Medical Center: lead

Study Sites (8)

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

Location

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

Location

Riley Hospital for Children at IU Health

Indianapolis, Indiana, 46202, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Monroe Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

Related Publications (1)

• 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.

  PMID: 33640012 BACKGROUND

MeSH Terms

Conditions

Feeding Behavior

Interventions

Megestrol Acetate; Megestrol

Condition Hierarchy (Ancestors)

Behavior, Animal; Behavior

Intervention Hierarchy (Ancestors)

Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Ann Davis, PhD, MPH, ABPP, Director, Center for Children's Healthy Lifestyles & Nutrition
• Organization: University of Kansas Medical Center

adavis6@kumc.edu

(913) 588-1227

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: 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).

Study Record Dates

• First Submitted: January 17, 2019
• First Posted: January 24, 2019
• Study Start: August 15, 2019
• Primary Completion: March 19, 2025
• Study Completion: March 19, 2025
• Last Updated: September 10, 2025
• Results First Posted: September 10, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)