Outcomes Related to Body Composition in Teens and Adults With Cystic Fibrosis (ORBIT-CF)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Emory University
- Enrollment
- 90
- Locations
- 2
- Primary Endpoint
- Change in Disposition Index
Study Overview
Brief Summary
Nutrition and body composition, the amount of muscle and fat in the body, has a role in overall health. This study wants to learn more about how nutrition and body composition affects health outcomes like glucose tolerance and lung function in patients with cystic fibrosis (CF) who are ages 16-30 years old. 60 adolescents and young adults with CF will be recruited, and 30 volunteers without cystic fibrosis. A total of 40 of these study participants with CF will be asked to return for annual study visits for 2 years after the first visit.
The long-term goal of this study is to use the information collected to make decisions about future nutrition monitoring and interventions which help maintain optimal health for individuals with CF.
Detailed Description
This is a prospective, observation study to test the central hypothesis that individuals with cystic fibrosis (CF) have a higher propensity to increased visceral adipose tissue (VAT) accumulation and decreased lean body mass (LBM) compared to healthy controls, and this dysregulation in adipose and protein deposition exacerbates glucose intolerance and lung function decline. A sub-set of participants with CF will be followed longitudinally for two years (n=40). The investigators will conduct detailed body composition, fat distribution, metabolic, and nutritional phenotyping in this cohort. Body fat distribution will be assessed with MRI. Whole body composition will be assessed with DEXA. Glucose tolerance will be assessed with an oral glucose tolerance test (OGTT) and mathematical modeling of the C-peptide and insulin response to glucose. Lung health will be assessed by objective clinical data and self-reported symptoms.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 16 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •CF inclusion criteria
- •confirmed CF diagnosis based on sweat testing by pilocarpine iontophoresis and/or CFTR genotyping with two disease causing mutations
- •be aged ≥ 16 yrs
- •clinically stable, defined as no changes in medical regimen (including medications) for at least 21 days prior to study visit
- •participation in the CFF Patient Registry
- •Longitudinal study inclusion:
- •CF participants who have normal glucose tolerance results after their initial study oral glucose tolerance test (OGTT).
- •Healthy controls inclusion criteria:
- •male or female ages 16 years and older
- •clinically stable. Healthy controls will be recruited who are similar in age, gender, and BMI as the participants with CF.
Exclusion Criteria
- •CF exclusion criteria:
- •diagnosis of CF-related diabetes (CFRD)
- •nocturnal tube feeds
- •life expectancy <6 months
- •history of or on waiting list for lung transplant
- •un-removable metal that is incompatible with MRI
- •inability or unwillingness to perform major study activities (OGTT, DEXA, MRI) due to claustrophobia, fear of blood draw, or other reasons
- •current pregnancy or lactation
- •study visit falls between window of 1 week to 8 weeks of initiation of CFTR modulator
- •use of chronic oral corticosteroids,
- •in the opinion of the CF Care Team or study physician, participant should not participate in the study, or
- •inability to provide informed consent or assent.
- •Healthy controls exclusion criteria:
- •malignant neoplasm (other than localized basal cell cancer of the skin) during the previous 5 years
- •respiratory (including asthma), endocrine (including diabetes), autoimmune, or other chronic disease
- •HIV or other chronic infection
- •current use of any medications to treat an acute or chronic disease or illness (anti-depressants, anti-anxiety medications are acceptable),
- •acute illness within the past 3 weeks
- •intravenous or oral antibiotics or use of systemic corticosteroids within the past 3 weeks
- •inability or unwillingness to perform major study activities due to claustrophobia, fear of blood draw, or other reasons
- •current pregnancy or lactation, or
- •inability to provide informed consent or assent.
Arms & Interventions
Subjects with Cystic Fibrosis
n=60 patients with CF ages 16-30
Healthy Controls
n=30 healthy controls matched to participants with CF for age, sex, BMI, and race.
Outcomes
Primary Outcomes
Change in Disposition Index
Time Frame: Baseline, 1 year, 2 year
The disposition index (DI) is a measure of the ability of B-cells to compensate for insulin resistance. A lower DI indicates a loss of B-cell function, which means decreased pancreatic function. The disposition index will be assessed with an oral glucose tolerance test (OGTT) and mathematical modeling of the C-peptide and insulin response to glucose. This study seeks to determine if glucose intolerance is associated with body composition and fat distribution in CF subjects.
Change in Visceral Adipose Tissue volume (VAT) by Magnetic Resonance Imaging (MRI)
Time Frame: Baseline, 1 year, 2 year
Body fat distribution and body composition in 60 individuals with Cystic Fibrosis (CF) and 30 matched, healthy control will be assessed by Magnetic Resonance Imaging (MRI)
Change in Forced Expiratory Volume in the first second (FEV1%)
Time Frame: Baseline, 1 year, 2 year
Clinical spirometry is a test of lung function that will be used to assess the progression of lung disease with the baseline Forced Expiratory Volume (FEV%) predicted within the past year. Baseline is defined as the average of the best FEV1% for each quarter of the calendar year. FEV1% predicted is a method of determining the severity of pulmonary disease and declines as disease severity increases.
Secondary Outcomes
- Number of pulmonary exacerbations needing intravenous (IV) antibiotics within previous five years(Baseline)
- Change in Thigh perimuscular adipose tissue (PMAT)(Baseline, 1 year, 2 year)
- Change in Body Composition Analysis(Baseline, 1 year, 2 year)
- Change in Whole body insulin sensitivity index (WBISI)(Baseline, 1 year, 2 year)
- Number of Perceived respiratory symptoms measured with the Cystic Fibrosis Questionnaire-Revised (CFQ-R)(Baseline, 1 year, 2 year)
- Change in Pancreatic lipid(Baseline, 1 year, 2 year)
- Change in Hepatic lipid(Baseline, 1 year, 2 year)
- Change in Insulin secretion(Baseline, 1 year, 2 year)
- Annual rate of Forced Expiratory Volume in the first second (FEV1%) decline(Baseline, 1 year, 2 year)
Investigators
Jessica Alvarez
Assitant professor
Emory University