Assessment of Fat Free Mass Index and Its Impact on Health in Children With Cystic Fibrosis (GAIA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by University of Arkansas
Sponsor:
Collaborator:
Arkansas Children's Hospital Research Institute
Information provided by (Responsible Party):
University of Arkansas
ClinicalTrials.gov Identifier:
NCT01401439
First received: July 22, 2011
Last updated: August 27, 2014
Last verified: August 2014
  Purpose

The purpose of this study is to find out whether measurement of body composition has a prognostic value on clinical and overall outcome in pediatric patients with cystic fibrosis.


Condition
Cystic Fibrosis

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Longitudinal Assessment of Fat Free Mass Index and Its Impact on Lung Health and Overall Health Metrics in Pediatric Patients With Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by University of Arkansas:

Primary Outcome Measures:
  • body composition (bone mineral density) [ Time Frame: change from baseline to 18 months ] [ Designated as safety issue: No ]
    Compare Dual Energy X-Ray Absorptiometry (DEXA) analysis to GAIA device


Secondary Outcome Measures:
  • Biomarkers: physical performance [ Time Frame: change from baseline to 18 months ] [ Designated as safety issue: No ]
    Physical performance includes skeletal muscle function,respiratory muscle function, lung function

  • Biomarkers: quality of life [ Time Frame: change from baseline to 18 months ] [ Designated as safety issue: No ]
    Quality of life includes nutrition, activity levels and general well-being measured by questionnaires

  • Biomarkers: blood [ Time Frame: change from baseline to 18 months ] [ Designated as safety issue: No ]
    Blood will be analyzed for inflammatory mediators, bone mineral markers, hormone levels, analysis of concentrations of amino acids, glucose and parathormone


Biospecimen Retention:   Samples Without DNA

Blood sample


Estimated Enrollment: 70
Study Start Date: June 2012
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Detailed Description:

Cystic fibrosis (CF) is one of the most common life-threatening inherited disorders affecting 1/35000 individuals in the US. In the past decades, the median predicted survival of CF patients has increased significantly from 10-12 to over 37 years (1). One of the major factors contributing to increased survival has been the understanding of the role of optimal nutrition on overall health status of individuals with CF.

Forced expiratory volume in one second (FEV1) is currently the best predictor of pulmonary health in CF, and malnutrition in a person of any age with CF has a negative impact on lung function with a more rapid yearly decline in FEV1. Recent data from the Cystic Fibrosis Foundation (CFF) patient registries in the United States and Canada were analyzed to determine whether any association existed between pulmonary function as measured by FEV1 and Body mass index (BMI) for age. BMI-for-age percentile values >50th percentile were associated with well-preserved lung function and lower values of BMI were associated with incrementally lower values of FEV1 (2). Thus, the CFF recommended that children with CF between the ages of 2 and 20 years maintain a BMI a≥50th percentile for age and sex. However, several publications have demonstrated that fat free muscle (FFM) wasting is evident even in individuals with a BMI > 50th percentile, and it is unknown whether BMI percentile adequately reflects changes in body composition in this group of patients; therefore, BMI might not be the best marker indicating an optimal nutritional status.

Limitations of BMI as a marker of optimal Nutrition: fat mass versus muscle mass In individuals with CF, BMI is used to assess appropriateness of weight to height for age and sex. Since there is a strong association between BMI and overall health metrics, especially lung health, the CFF recommends that children with CF maintain a BMI ≥50th percentile for age and sex. However, an apparent loss of skeletal muscle may occur in CF patients who retain their normal body weight, indicating that BMI does not provide any information about body compartments and may fail to detect subtle alterations in body composition. Although several epidemiologic studies showed that CF patients who maintain BMI> 50th percentile will have improved lung functions and survival, recent data indicate that the preserved FFM instead of BMI may have an impact on overall survival in CF (7). Because of the variability in the levels of both fat mass and fat-free mass, and the many combinations of fat mass index and FFM index associated with the same BMI, this weight-height index needs to be carefully interpreted. Because high levels of BMI-for-age are associated with substantial increases in fat mass, BMI is most useful as a measure of obesity. In chronic inflammatory diseases, a change in regional fat distribution is observed with a shift to a more central fat distribution. The latter is associated with an increase in low-grade chronic inflammation, insulin resistance, and cardiovascular risk (8). Therefore, BMI is a good index for assessment of obesity in otherwise normal children, but not adequate or sufficient in the CF population.

In summary, some pediatric patients with CF may have a decreased Fat-free Mass Index (FFMI) with a well preserved BMI of ≥50th percentile. Conversely, some patient might have a BMI<50th percentile with normal FFMI associated with overall well-being. Current practice forces CF patients to consume high fat and caloric nutrients to maintain a BMI >50th percentiles incurring the costs of causing anxiety, stress, daily struggle in the family with a potential increase in the pro-inflammatory state due to central fat accumulation. The outcome of the proposed study will contribute to the understanding of the relationships between BMI, muscle wasting and markers of health and wellbeing. If our study demonstrates that a well maintained FFMI rather than the BMI correlates better with good health status in CF, it may result in changes of current nutrition practice in CF at the national or even international level.

  Eligibility

Ages Eligible for Study:   7 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Children with cystic fibrosis 7-17 years followed at Arkansas Children's hospital Cystic Fibrosis Care Center

Criteria

Inclusion Criteria:

  • Subjects who already have a diagnosis of CF based on universal diagnostic criteria
  • Age 7 to 17 years at the time of enrollment
  • Under routine medical control at the CF center of Arkansas Children's Hospital(ACH)
  • Clinically stable in CF symptoms as determined by investigators

Exclusion Criteria:

  • Unstable current diagnosis of metabolic diseases including liver (cirrhosis) or renal disease
  • Failure to give informed consent
  • Individuals with pacemakers
  • Pregnancy in females of childbearing age. A pregnancy test will be performed on females who are post-menarche before performing a DEXA scan
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01401439

Contacts
Contact: Pat M. Brady, BSN 501-364-1544 bradypatriciam@uams.edu

Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Principal Investigator: Gulnur Com, MD         
Sub-Investigator: Ariel Berlinski, MD         
Sub-Investigator: John Carroll, MD         
Sponsors and Collaborators
University of Arkansas
Arkansas Children's Hospital Research Institute
Investigators
Principal Investigator: Gulnur Com, MD University of Arkansas for Medical Sciences/Arkansas Children's Hospital
  More Information

Additional Information:
Publications:

Responsible Party: University of Arkansas
ClinicalTrials.gov Identifier: NCT01401439     History of Changes
Other Study ID Numbers: 131677
Study First Received: July 22, 2011
Last Updated: August 27, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by University of Arkansas:
cystic fibrosis
body mass index
fat free mass
Children with cystic fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on September 16, 2014