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.

Condition	Intervention	Phase
Cystic Fibrosis Pancreatic Cystic Fibrosis	Behavior: Behavioral plus Nutrition Treatment	Phase III

Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Primary Outcomes: Energy intake at baseline, month 6 and 18; Weight for age z score at baseline and month 18; Height for age z score at baseline and month 18
Secondary Outcomes: Enzyme adherence at baseline, month 6 and 18; BMI at baseline, months 6 and 18; Body composition via DXA at baseline and month 18; Body composition via skinfolds at baseline and month 18
Expected Total Enrollment:  100

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 accredited CF Centers in Ohio (Cincinnati, Columbus, Cleveland), 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 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.

Ages Eligible for Study:  2 Years   -   6 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• confirmed diagnosis of cystic fibrosis based upon 2 of the following: a. sweat chloride by quantitative pilocarpine electrophoresis ≥60 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 ≤ 15 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 CF TDN

Please refer to this study by ClinicalTrials.gov identifier  NCT00241969

Leigh A Chamberlin, MEd      513-636-7735    lacham@cchmc.org
Stephanie M Sullivan, BS      513-636-7735    stephanie.sullivan@cchmc.org

Ohio
      Cincinnati Children''''s Hospital Medical Center, Cincinnati,  Ohio,  45229,  United States
      Columbus Children''''s Hospital, Columbus,  Ohio,  43205,  United States
      Rainbows and Babies Children''''s Hospital, Cleveland,  Ohio,  44106,  United States

Study chairs or principal investigators

Scott W. Powers, PhD,  Principal Investigator,  Children''''s Hospital Medical Center - Cincinnati   

Study ID Numbers:  DK 54915
Last Updated:  December 8, 2005
Record first received:  October 18, 2005
ClinicalTrials.gov Identifier:  NCT00241969
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2006-01-10