Clinical trial NCT00241969: Behavioral & Nutritional Treatment to Help CF Preschoolers Grow
Official title: A Multi-Site Randomized Clinical Trial of Behavioral and Nutrition Treatment Designed to Help Preschoolers With Cystic Fibrosis Optimize Growth
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Pancreatic Cystic Fibrosis
Keywords: nutrition therapy
preschool children
cystic fibrosis
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
First posted: 2005-10-19
Enrollment: 78
Sex: ALL
Minimum age (years): 2
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in Energy Intake From Baseline to Post Treatment
timeFrame: 6 months
measure: Change in Weight for Age Z-Score (WAZ) From Baseline to Post Treatment
timeFrame: 6 months
measure: Change in Height for Age Z-Score (HAZ) From Baseline to Follow Up
timeFrame: 18 months
Secondary outcomes:
Other outcomes:
Locations:
city: Tucson
country: United States
facility: University of Arizona
state: Arizona
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbows and Babies Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Scott W. Powers, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Powers SW, Piazza-Waggoner C, Jones JS, Ferguson KS, Daines C, Acton JD. Examining clinical trial results with single-subject analysis: an example involving behavioral and nutrition treatment for young children with cystic fibrosis. J Pediatr Psychol. 2006 Jul;31(6):574-81. doi: 10.1093/jpepsy/jsj049. Epub 2005 Jul 13.
citation: Stark LJ, Opipari LC, Jelalian E, Powers SW, Janicke DM, Mulvihill MM, Hovell MF. Child behavior and parent management strategies at mealtimes in families with a school-age child with cystic fibrosis. Health Psychol. 2005 May;24(3):274-80. doi: 10.1037/0278-6133.24.3.274.
citation: Powers SW, Patton SR, Rajan S. A comparison of food group variety between toddlers with and without cystic fibrosis. J Hum Nutr Diet. 2004 Dec;17(6):523-7. doi: 10.1111/j.1365-277X.2004.00560.x.
citation: Mitchell MJ, Powers SW, Byars KC, Dickstein S, Stark LJ. Family functioning in young children with cystic fibrosis: observations of interactions at mealtime. J Dev Behav Pediatr. 2004 Oct;25(5):335-46. doi: 10.1097/00004703-200410000-00005.
citation: Powers SW, Patton SR. A comparison of nutrient intake between infants and toddlers with and without cystic fibrosis. J Am Diet Assoc. 2003 Dec;103(12):1620-5. doi: 10.1016/j.jada.2003.09.024.
citation: Powers SW, Patton SR, Byars KC, Mitchell MJ, Jelalian E, Mulvihill MM, Hovell MF, Stark LJ. Caloric intake and eating behavior in infants and toddlers with cystic fibrosis. Pediatrics. 2002 May;109(5):E75-5. doi: 10.1542/peds.109.5.e75.
citation: Stark LJ, Jelalian E, Powers SW, Mulvihill MM, Opipari LC, Bowen A, Harwood I, Passero MA, Lapey A, Light M, Hovell MF. Parent and child mealtime behavior in families of children with cystic fibrosis. J Pediatr. 2000 Feb;136(2):195-200. doi: 10.1016/s0022-3476(00)70101-6.
citation: Stark LJ, Mulvihill MM, Jelalian E, Bowen AM, Powers SW, Tao S, Creveling S, Passero MA, Harwood I, Light M, Lapey A, Hovell MF. Descriptive analysis of eating behavior in school-age children with cystic fibrosis and healthy control children. Pediatrics. 1997 May;99(5):665-71.
citation: Stark LJ, Mulvihill MM, Powers SW, Jelalian E, Keating K, Creveling S, Byrnes-Collins B, Harwood I, Passero MA, Light M, Miller DL, Hovell MF. Behavioral intervention to improve calorie intake of children with cystic fibrosis: treatment versus wait list control. J Pediatr Gastroenterol Nutr. 1996 Apr;22(3):240-53. doi: 10.1097/00005176-199604000-00005.
citation: Stark LJ, Jelalian E, Mulvihill MM, Powers SW, Bowen AM, Spieth LE, Keating K, Evans S, Creveling S, Harwood I, et al. Eating in preschool children with cystic fibrosis and healthy peers: behavioral analysis. Pediatrics. 1995 Feb;95(2):210-5.
citation: Stark LJ, Powers SW, Jelalian E, Rape RN, Miller DL. Modifying problematic mealtime interactions of children with cystic fibrosis and their parents via behavioral parent training. J Pediatr Psychol. 1994 Dec;19(6):751-68. doi: 10.1093/jpepsy/19.6.751.
citation: Stark LJ, Knapp LG, Bowen AM, Powers SW, Jelalian E, Evans S, Passero MA, Mulvihill MM, Hovell M. Increasing calorie consumption in children with cystic fibrosis: replication with 2-year follow-up. J Appl Behav Anal. 1993 Winter;26(4):435-50. doi: 10.1901/jaba.1993.26-435.
citation: Powers SW, Stark LJ, Chamberlin LA, Filigno SS, Sullivan SM, Lemanek KL, Butcher JL, Driscoll KA, Daines CL, Brody AS, Schindler T, Konstan MW, McCoy KS, Nasr SZ, Castile RG, Acton JD, Wooldridge JL, Ksenich RA, Szczesniak RD, Rausch JR, Stallings VA, Zemel BS, Clancy JP. Behavioral and nutritional treatment for preschool-aged children with cystic fibrosis: a randomized clinical trial. JAMA Pediatr. 2015 May;169(5):e150636. doi: 10.1001/jamapediatrics.2015.0636. Epub 2015 May 4.