Behavioral & Nutritional Treatment to Help CF Preschoolers Grow

Not Applicable

Completed

Conditions: Pancreatic Cystic Fibrosis
Cystic Fibrosis

Interventions: Behavioral: Education and Attention Control
Behavioral: Behavioral and Nutrition Treatment

Registration Number: NCT00241969

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary: 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: 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:

1. determine the impact of the behavioral intervention on energy intake and weight gain;

2. examine the durability of the behavioral intervention's impact on growth (weight and height) one year following treatment; and

3. 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 78

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Education and Attention Control	Education and Attention Control	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 and Nutrition Treatment	Behavioral and Nutrition Treatment	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.

Primary Outcome Measures

Name	Time	Method
Change in Height for Age Z-Score (HAZ) From Baseline to Follow Up	18 months	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.
Change in Weight for Age Z-Score (WAZ) From Baseline to Post Treatment	6 months	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.
Change in Energy Intake From Baseline to Post Treatment	6 months	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

Secondary Outcome Measures

Name	Time	Method

Trial Locations

Locations (5): Nationwide Children's Hospital
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Columbus, Ohio, United States
Cincinnati Children's Hospital Medical Center
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Cincinnati, Ohio, United States
Rainbows and Babies Children's Hospital
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Cleveland, Ohio, United States
University of Arizona
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Tucson, Arizona, United States
University of Michigan Health System
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Ann Arbor, Michigan, United States