Clinical trial NCT03815019: iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children
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
Overall status: COMPLETED
Phase: PHASE4
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
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
First posted: 2019-01-24
Enrollment: 71
Sex: ALL
Minimum age (years): 9
Maximum age (years): 108
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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).
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in % kilocalories/day obtained orally
timeFrame: Weeks 0 to 24
Secondary outcomes:
measure: Number of patients with adrenal insufficiency as measured by morning cortisol lab value
timeFrame: 10 to 14 weeks
measure: Change in parent stress as measured by the Pediatric Inventory for Parents (PIP36)
timeFrame: Weeks 0 to 24
measure: Change in child quality of life as measured by the Infant Toddler Quality of Life scale (ITQOL47)
timeFrame: Week 0 to 24
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Rady Children's Hospital - San Diego
state: California
city: Orlando
country: United States
facility: Arnold Palmer Hospital for Children
state: Florida
city: Indianapolis
country: United States
facility: Riley Hospital for Children at IU Health
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Nashville
country: United States
facility: Monroe Carell Jr. Children's Hospital at Vanderbilt
state: Tennessee
Study officials:
References:
citation: Choi KT, Illievich UM, Zornow MH, Scheller MS, Strnat MA. Effect of hyperglycemia on peri-ischemic neurotransmitter levels in the rabbit hippocampus. Brain Res. 1994 Apr 11;642(1-2):104-10. doi: 10.1016/0006-8993(94)90910-5.