Behavioral & Nutritional Treatment to Help CF Preschoolers Grow

Study Overview
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Brief Summary
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The primary objective of this NIH funded clinical trial is to conduct a multi-center, randomized, controlled trial comparing two interventions: a behavioral plus nutrition intervention to a nutrition intervention. This study will (a) determine the impact of the behavioral intervention on energy intake and weight gain; (b) examine the durability of the behavioral intervention's impact on growth (weight and height) one year following treatment; and (c) explore the relation between physical activity and growth.

Detailed Description
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Evidence-based nutritional interventions that achieve and sustain optimal growth in young children with cystic fibrosis (CF) do not exist, despite an urgent need. Such an intervention could positively change the course of clinical lung disease and enhance survival for these children. The primary objective of this NIH funded clinical trial is to conduct a multi-center, randomized, controlled trial comparing two interventions: a behavioral plus nutrition intervention to a nutrition (attention control) intervention. All subjects will receive nutritional care consistent with the 2001 CF Consensus Conference guidelines for pediatric nutrition. The specific aims are to: determine the impact of the behavioral intervention on energy intake and weight gain; examine the durability of the behavioral intervention's impact on growth (weight and height) one year following treatment; and explore the relation between physical activity and growth. The central hypothesis is that behavioral intervention will lead to better growth as measured by change in weight and height for age z scores. From three CF Centers in Ohio, (Cincinnati Children's, Columbus Children's, Rainbow Babies and Children's Hospital in Cleveland), two referral centers in Ohio (Dayton Children's and Akron Children's), one CF Center in Michigan (University of Michigan-Ann Arbor), and one CF Center in Arizona (University of Arizona-Tucson), 100 preschoolers with CF and pancreatic insufficiency age 2 to 6 years will be randomized to one of the two conditions. The two groups will be stratified so that they are similar at the initiation of treatment on weight for age z score. Other critical variables such as history of Pseudomonas aeruginosa infection and gender will be used as covariates in the statistical analysis plan. Outcome data (energy intake measured by 7-day diet record, weight, height) will be obtained at baseline, post-treatment (6 months), and after a 12-month follow-up (18 months post baseline). Secondary measures will include body mass index, body composition measured by dual energy x-ray absorptiometry (DXA) and skinfolds, and growth velocity. Behavioral treatment will maximize adherence to a high energy diet and enzyme replacement therapy, and motivate children to increase their energy intake. It involves 7 weekly sessions followed by 4 monthly sessions. The attention condition controls for time of contact and number of assessments conducted. This study advances the investigation of early nutritional interventions for young children with CF and directly addresses the need for controlled, longitudinal assessment of behavioral intervention on growth. The long-range goal is to change the standard of nutritional care for young children with CF because behavioral intervention leads to optimal growth and ultimately improves lung health and survival.

Official Title
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A Multi-Site Randomized Clinical Trial of Behavioral and Nutrition Treatment Designed to Help Preschoolers With Cystic Fibrosis Optimize Growth

Conditions
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Cystic Fibrosis, Pancreatic Cystic Fibrosis

Intervention / Treatment
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* Behavioral: Behavioral and Nutrition Treatment
* Behavioral: Education and Attention Control


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: confirmed diagnosis of cystic fibrosis based upon 2 of the following: a. sweat chloride by quantitative pilocarpine electrophoresis ≥60 milliequivalent/Liter (mEq/L), b. two clinical features consistent with CF, or c. genetic testing demonstrating two mutations associated with CF confirmation of pancreatic insufficiency based upon fecal elastase of ≤ 100 micrograms per gram of stool (or an undetectable level) age at enrollment to the trial of 2.0 years to 6.0 years at least 6 months post CF diagnosis consuming an unrestricted fat diet Exclusion Criteria: diagnosis of developmental delay (i.e., autism, cerebral palsy, or mental retardation) receiving supplemental enteral nutrition via nasogastric tube, gastrostomy, or total parenteral nutrition diagnosed with another disease/condition (e.g., insulin dependent diabetes, congenital heart disease, significant renal disease, history of bowel resection or short bowel syndrome, colonic strictures) known to affect growth taking a medication (e.g., insulin, growth hormone, chronic use of systemic steroids) known to affect growth screening assessment shows genetic potential for height as acceptable according to the 2001 Consensus Conference guidelines and diet diary indicates daily Dietary Reference Intake (DRI) of energy average of 140% or greater (DRI of 100% will be determined as the estimated energy requirement [EER] based upon the child's age, gender, and an active physical activity level weight z score (age and gender adjusted) of > 1.0 prior participation in the pilot intervention studies conducted by the PI during the prior period of R01 funding or current participation in an intervention trial conducted by the Cystic Fibrosis Therapeutics Development Network.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Behavioral and Nutrition Treatment<br>The behavioral and nutrition treatment combines individualized nutritional counseling that targeted increasing energy and fat intake and parent training in behavioral child-management skills based on social learning theory to improve meal-time behaviors. | Behavioral: Behavioral and Nutrition Treatment<br>* This intervention will combine individualized nutrition counseling that targets increasing energy and fat intake and parent training of effective behavioral child management skills. The treatment is delivered through 8 weekly sessions followed by 4 monthly sessions, each lasting around 60 minutes.<br>|
| Active Comparator: Education and Attention Control<br>The education and attention control treatment provides education and served as a behavioral placebo in terms of controlling for attention and contact frequency provided. Families are provided with information including general nutrition, enzyme therapy, respiratory infection control, and typical child development anticipatory guidance and safety for preschool- aged children. | Behavioral: Education and Attention Control<br>* This intervention will provide information about a number of aspects of their child's CF care and also provides anticipatory guidance for preschoolers.The treatment is delivered through 8 weekly sessions followed by 4 monthly sessions, each lasting around 60 minutes.<br>* Other names: control arm;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Energy Intake From Baseline to Post Treatment | This primary outcome measure compared change in energy intake from baseline to post treatment between the behavioral and nutrition treatment and the education and attention control treatment. Energy intake was assessed using a 7-day diet diary recorded by parents and analyzed using Nutrition Data System for Research Software, Version 2011. Data were examined as average kilocalories per day over the 7 day period at baseline and post treatment. The mean (SD) change in energy intake was compared between baseline to post treatment was | 6 months | 
| Change in Weight for Age Z-Score (WAZ) From Baseline to Post Treatment | This outcome measure examines the change in weight for age Z-score (WAZ) from baseline to post treatment. Weight was measured in kilograms, measured to the nearest 100 grams, obtained using a digital scale by trained study staff. All measurements were obtained in triplicate and then the mean used for analyses. Weight for age Z score was calculated using the mean measurement and the Centers for Disease Control and Prevention Anthropometric Software Program. The z score is a measure of the number of standard deviations that an observation is above or below the mean. A positive z score indicates that the observation is above the mean, a negative z score that the observation is below the mean. | 6 months | 
| Change in Height for Age Z-Score (HAZ) From Baseline to Follow Up | This outcome measure examines the change in height for age Z-score (HAZ) from baseline to follow up. Height was measured standing unless the child was unwilling to stand, then a supine measurement was obtained. All measurements were obtained in triplicate and then the mean used for analyses. Height for age Z score was calculated using the mean measurement and the Centers for Disease Control and Prevention Anthropometric Software Program. The z score is a measure of the number of standard deviations that an observation is above or below the mean. A positive z score indicates that the observation is above the mean, a negative z score that the observation is below the mean. | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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nutrition therapy, preschool children, cystic fibrosis