Purpose

The goal of the study is to examine multiple markers of anthropometrics, body composition, sarcopenia and frailty and compare them to dual energy X-ray absorptiometry (DXA) output, which is considered the current clinical gold-standard tool to measure body composition. The result of this study will provide detailed data regarding the nutrition and body composition within this Cystic Fibrosis population and also provide a baseline evaluation for use of these biomarkers in the future studies including evaluation of nutritional intervention. Further, the study will also include psychosocial and other patient-reported outcomes and medical contributors to understand their contributions to the nutritional failure in the adult advanced lung disease population. Finally, the study will evaluate both established and emerging nutritional and body composition parameters and link them to clinical outcomes in adults with CF across the spectrum of pulmonary function.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Cohort 1: Participants are eligible if their percentage of predicated forced expiratory volume in 1 second (ppFEV1) is <70% during the 12 months prior to enrollment (>50% of measurements, eliminating periods of exacerbation) - Cohort 2: Participants are eligible if their percentage of predicted forced expiratory volume in 1 second (ppFEV1) is 70% or greater during the 12 months prior to enrollment (>50% of measurements, eliminating periods of exacerbation). - Both cohorts match by age, gender, race and CFTR genotype severity.

Exclusion Criteria

  • No prior solid organ transplantation - No initiation of an investigation drug within 28 days prior to and including Baseline Visit. - No initiation of new chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin, Cayston, CFTR modulator, etc.) within 28 days prior to and including Baseline visit. - No acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 14 days prior to and including Baseline visit. - For the BIA sub-study - Individuals with an implanted pacemaker will be excluded. - No initiation of a drug for weight loss (such as a GLP-1 receptor agonist) or bariatric surgery within 6-months prior to and including the Baseline visit. - Patients with continued rapid change or extreme GI symptoms related to weight loss therapy should be excluded at the discretion of the study investigator.

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Cohort 1 Forced expiratory volume in 1 second (FEV1) <70% predicted during the 12 months prior to enrollment (>50% of measurements, eliminating periods of exacerbation). If no stable spirometry data are available in the 12 months prior to enrollment, from the prior 24 months will be used.
  • Diagnostic Test: BMI and lean mass index from DXA
    Estimate and compare correlation between lean mass index from DXA and BMI
  • Diagnostic Test: Anthropometric Measurements
    Skinfold will be assessed with calipers. Circumference will be measured with a tape measure.
  • Diagnostic Test: Hand-grip strength
    A small, handheld dynamometer will be used to measure grip strength, a measure of function, on each hand.
  • Diagnostic Test: Six-minute walk Test
    This will be another functional assessment of fitness. Participants will be asked to walk at their normal pace for 6 minutes.The total distance walked in that time will be measured.
  • Diagnostic Test: Sit-to-Stand Test
    This will be used as a functional assessment of fitness and lower extremity strength. Participants will start seated on a chair and be asked to complete as many sit-to-stand repetitions without using their arms for one minute.
  • Diagnostic Test: Short physical performance battery (SPPB)
    This is an assessment of frailty. The test is completed in approximately 8 minutes. The test consists of 3 assessments: 1) Balance tests where the participant stands and tries to balance with their feet in various positions for 10 seconds each without assistance (side-by-side, heel-to-side, and heel-to-toe); 2) Two 4-meter gait speed tests; and 3) Chair-stand tests (single chair stand, 5 chair stands).
  • Diagnostic Test: BIA Sub-study
    A minimum of 50 individuals will be enrolled. Body composition will be assessed at each study visit using study bioelectrical impendence analysis (BIA).
  • Diagnostic Test: Accelerometry to assess physical activity
    Participants will be provided with a wrist-worn accelerometer/heart rate monitor at the baseline study visit and asked to wear it continuously for at least 3-10 days (two weekdays, one weekend day). Approximately every 3 months, the participant will be asked to again wear the accelerometer. Participants will be able to keep their accelerometers.
  • Other: Gastrointestinal (GI) and nutrition questionnaires:
    Subjects will complete surveys regarding gastrointestinal (GI) symptoms, including the Patient Assessment of Constipation (PAC-SYM) Score, Patient Assessment of Gastrointestinal Symptoms (PACGI-SYM) Score, the Bristol Stool Chart, and the scored Patient-Generated Subjective Global Assessment (PG-SGA).
  • Other: Psychosocial questionnaire: PHQ-8
    This is an 8-item scale that measures depressive symptoms over the past two weeks.
  • Other: Psychosocial questionnaire: GAD-7
    This is a 7-items scale that measures anxiety symptoms over the past two weeks, from not at all to nearly every day.
  • Other: Psychosocial questionnaire: The Treatment Self-Regulation Questionnaire (TSRQ)
    This is a 15-item scale that assesses the degree to which participants' motivation is autonomous or self-regulated.
  • Other: Psychosocial questionnaire: CF Fatalism Scale
    The CF Fatalism Scale measures the belief in a lack of personal power or control over one's future and has 13 items.
  • Other: Psychosocial questionnaire: Body Esteem Scale for Adolescents and Adults (BESAA)
    The Body Esteem Scale for Adolescents and Adults (BESAA) is 23-item measurement of self-evaluations of one's body or appearance.
  • Other: 12-month Questionnaire
    A short questionnaire designed to explore participants' perspectives of the acceptability and feasibility of nutritional assessments used in STRONG-CF will be given to all study participants at the end of the study.
  • Other: Oral glucose tolerance testing (OGTT)
    For subjects without previously diagnosed CFRD, an OGTT will be performed at baseline and 12-months with samples analyzed at the central lab.
  • Device: Continuous glucose monitoring (CGM)
    Subjects will wear a Dexcom G6Pro sensor in blinded mode for three 10-day periods to collect comprehensive glucose data.
  • Diagnostic Test: Chest CT scans (When available within the past 6 months in medical records)
    Standard of Care chest CT images will be drawn from the medical record, as available, from enrolled participants. FEV1 measurements prior to chest CT scans will be recorded to account for possible illness occurring at the time of scanning. Quantitative assessment of the pectoralis muscle area will be performed on the first single axial image above the aortic arch. Any additional standard of care chest CT's that are performed while the participant is enrolled in the study will also be collected and have a quantitative assessment of the pectoralis muscle performed.
  • Diagnostic Test: Hologic Dual X-Ray Absorptiometry (DXA)
    DXA measurements for whole body, total hip and lumbar spine will be acquired using the Hologic DXA and standard positioning as noted in the DXA Manual of Operations. DXA will be used to estimate total and regional body composition, which will include body fat and lean body mass. This will be used as the gold standard from which to validate BIA and MAMC.
  • Diagnostic Test: Ultrasound Sub-study of assessment of appendage muscles using ultrasound
    A minimum of 50 individuals will be enrolled. At each study visit, participants will undergo ultrasound muscle measurements (cross-sectional area and muscle thickness) of the biceps and quadriceps on each (left and right) side of the body (4 total areas). These measurements will be obtained in triplicate for each patient.
  • Diagnostic Test: Psychosocial questionnaire: Hunger Vital Sign questionnaire
    Screening tool for identifying households at risk of food insecurity and poor health outcomes linked to food insecurity with 3-items over the last 12 months.
  • Other: Respiratory symptom questionnaire: CRISS
    Chronic Respiratory Infection Symptom Score (CRISS) is an 8-item respiratory symptom questionnaire covering the last 24-hours.
  • Diagnostic Test: Spirometry
    Spirometry will be performed in accordance with the current American Thoracic Society recommendations for the performance and interpretation of tests.
  • Other: Psychosocial questionnaire: Additional Health Questionnaire
    Single question over the last 3 months/90 days about cannabis use for GI problems and appetite. The clinical sites will not see the responses to this question. It will be completed by the participant online either during the study visit or at home.
  • Other: CF Management Questionnaire
    Assessment of prior 12-month management of cystic fibrosis, including treatments, medication intake and hospitalizations. This questionnaire will only be completed at 24- and 36- month visits.
  • Other: Food Frequency Questionnaire
    Assessment of prior 12-month nutritional intake being completed at Baseline (no later than 6-month visit) and 12-month visit.
Cohort 2 FEV1 ≥70% predicted during the 12 months prior to enrollment (>50% of measurements, eliminating periods of exacerbation).
  • Diagnostic Test: BMI and lean mass index from DXA
    Estimate and compare correlation between lean mass index from DXA and BMI
  • Diagnostic Test: Anthropometric Measurements
    Skinfold will be assessed with calipers. Circumference will be measured with a tape measure.
  • Diagnostic Test: Hand-grip strength
    A small, handheld dynamometer will be used to measure grip strength, a measure of function, on each hand.
  • Diagnostic Test: Six-minute walk Test
    This will be another functional assessment of fitness. Participants will be asked to walk at their normal pace for 6 minutes.The total distance walked in that time will be measured.
  • Diagnostic Test: Sit-to-Stand Test
    This will be used as a functional assessment of fitness and lower extremity strength. Participants will start seated on a chair and be asked to complete as many sit-to-stand repetitions without using their arms for one minute.
  • Diagnostic Test: Short physical performance battery (SPPB)
    This is an assessment of frailty. The test is completed in approximately 8 minutes. The test consists of 3 assessments: 1) Balance tests where the participant stands and tries to balance with their feet in various positions for 10 seconds each without assistance (side-by-side, heel-to-side, and heel-to-toe); 2) Two 4-meter gait speed tests; and 3) Chair-stand tests (single chair stand, 5 chair stands).
  • Diagnostic Test: BIA Sub-study
    A minimum of 50 individuals will be enrolled. Body composition will be assessed at each study visit using study bioelectrical impendence analysis (BIA).
  • Diagnostic Test: Accelerometry to assess physical activity
    Participants will be provided with a wrist-worn accelerometer/heart rate monitor at the baseline study visit and asked to wear it continuously for at least 3-10 days (two weekdays, one weekend day). Approximately every 3 months, the participant will be asked to again wear the accelerometer. Participants will be able to keep their accelerometers.
  • Other: Gastrointestinal (GI) and nutrition questionnaires:
    Subjects will complete surveys regarding gastrointestinal (GI) symptoms, including the Patient Assessment of Constipation (PAC-SYM) Score, Patient Assessment of Gastrointestinal Symptoms (PACGI-SYM) Score, the Bristol Stool Chart, and the scored Patient-Generated Subjective Global Assessment (PG-SGA).
  • Other: Psychosocial questionnaire: PHQ-8
    This is an 8-item scale that measures depressive symptoms over the past two weeks.
  • Other: Psychosocial questionnaire: GAD-7
    This is a 7-items scale that measures anxiety symptoms over the past two weeks, from not at all to nearly every day.
  • Other: Psychosocial questionnaire: The Treatment Self-Regulation Questionnaire (TSRQ)
    This is a 15-item scale that assesses the degree to which participants' motivation is autonomous or self-regulated.
  • Other: Psychosocial questionnaire: CF Fatalism Scale
    The CF Fatalism Scale measures the belief in a lack of personal power or control over one's future and has 13 items.
  • Other: Psychosocial questionnaire: Body Esteem Scale for Adolescents and Adults (BESAA)
    The Body Esteem Scale for Adolescents and Adults (BESAA) is 23-item measurement of self-evaluations of one's body or appearance.
  • Other: 12-month Questionnaire
    A short questionnaire designed to explore participants' perspectives of the acceptability and feasibility of nutritional assessments used in STRONG-CF will be given to all study participants at the end of the study.
  • Other: Oral glucose tolerance testing (OGTT)
    For subjects without previously diagnosed CFRD, an OGTT will be performed at baseline and 12-months with samples analyzed at the central lab.
  • Device: Continuous glucose monitoring (CGM)
    Subjects will wear a Dexcom G6Pro sensor in blinded mode for three 10-day periods to collect comprehensive glucose data.
  • Diagnostic Test: Chest CT scans (When available within the past 6 months in medical records)
    Standard of Care chest CT images will be drawn from the medical record, as available, from enrolled participants. FEV1 measurements prior to chest CT scans will be recorded to account for possible illness occurring at the time of scanning. Quantitative assessment of the pectoralis muscle area will be performed on the first single axial image above the aortic arch. Any additional standard of care chest CT's that are performed while the participant is enrolled in the study will also be collected and have a quantitative assessment of the pectoralis muscle performed.
  • Diagnostic Test: Hologic Dual X-Ray Absorptiometry (DXA)
    DXA measurements for whole body, total hip and lumbar spine will be acquired using the Hologic DXA and standard positioning as noted in the DXA Manual of Operations. DXA will be used to estimate total and regional body composition, which will include body fat and lean body mass. This will be used as the gold standard from which to validate BIA and MAMC.
  • Diagnostic Test: Ultrasound Sub-study of assessment of appendage muscles using ultrasound
    A minimum of 50 individuals will be enrolled. At each study visit, participants will undergo ultrasound muscle measurements (cross-sectional area and muscle thickness) of the biceps and quadriceps on each (left and right) side of the body (4 total areas). These measurements will be obtained in triplicate for each patient.
  • Diagnostic Test: Psychosocial questionnaire: Hunger Vital Sign questionnaire
    Screening tool for identifying households at risk of food insecurity and poor health outcomes linked to food insecurity with 3-items over the last 12 months.
  • Other: Respiratory symptom questionnaire: CRISS
    Chronic Respiratory Infection Symptom Score (CRISS) is an 8-item respiratory symptom questionnaire covering the last 24-hours.
  • Diagnostic Test: Spirometry
    Spirometry will be performed in accordance with the current American Thoracic Society recommendations for the performance and interpretation of tests.
  • Other: Psychosocial questionnaire: Additional Health Questionnaire
    Single question over the last 3 months/90 days about cannabis use for GI problems and appetite. The clinical sites will not see the responses to this question. It will be completed by the participant online either during the study visit or at home.
  • Other: CF Management Questionnaire
    Assessment of prior 12-month management of cystic fibrosis, including treatments, medication intake and hospitalizations. This questionnaire will only be completed at 24- and 36- month visits.
  • Other: Food Frequency Questionnaire
    Assessment of prior 12-month nutritional intake being completed at Baseline (no later than 6-month visit) and 12-month visit.

Recruiting Locations

University of Arizona
Tucson 5318313, Arizona 5551752 85724
Contact:
Cori Daines
cdaines@arizona.edu

University of Arkansas for Medical Sciences (UAMS)
Little Rock 4119403, Arkansas 4099753 72205
Contact:
Rajani Jagana
5016865525
RJagana@uams.edu

Emory
Atlanta 4180439, Georgia 4197000 30324
Contact:
William Hunt
4047787929
randy.hunt@emory.edu

Northwestern University
Chicago 4887398, Illinois 4896861 60611
Contact:
Adam Stein
3126954077
adam.stein@northwestern.edu

University of Kentucky
Lexington 4297983, Kentucky 6254925 40508
Contact:
Mark Wurth
859-257-5087
mark.wurth@uky.edu

John Hopkins University
Baltimore 4347778, Maryland 4361885 21287
Contact:
Noah Lechtzin
4109551167
nlechtz@jhmi.edu

Massachusetts General Hospital (MGH)
Boston 4930956, Massachusetts 6254926 02114
Contact:
Melissa Putman
msputman@partners.org

Boston Children's Hospital and Brigham and Women's CF Center
Boston 4930956, Massachusetts 6254926 02120
Contact:
Gregory Sawicki
6173551834
Gregory.Sawicki@childrens.harvard.edu

University of Minnesota
Minneapolis 5037649, Minnesota 5037779 55455
Contact:
Joanne Billings
612-625-7995
billi001@umn.edu

Washington University School of Medicine (St. Louis)
St Louis 4407066, Missouri 4398678 63110
Contact:
Jeffrey Atkinson
3147472940
atkinsonj@wustl.edu

Rutgers Health
New Brunswick 5101717, New Jersey 5101760 08901
Contact:
Sarah McCracken
7326670746
sem322@rwjms.rutgers.edu

New York Medical College (NYMC)
Hawthorne 5120284, New York 5128638 10532
Contact:
Allen Dozor
9144040152
Allen_dozor@nymc.edu

University of Cincinnati
Cincinnati 4508722, Ohio 5165418 45220
Contact:
Veronica Indihar
513-558-7036
indihama@ucmail.uc.edu

University Hospitals
Cleveland 5150529, Ohio 5165418 44106
Contact:
Kimberly McBennett
216-286-0709
kimberly.McBennett@uhhospitals.org

University of Oklahoma Sciences Center
Oklahoma City 4544349, Oklahoma 4544379 73104
Contact:
Nighat F Mehdi
405-271-6216
nighat-mehdi@ouhsc.edu

Oregon Health and Science University
Portland 5746545, Oregon 5744337 97239
Contact:
Aaron Trimble
503-418-8108
trimblea@ohsu.edu

University of Pittsburgh Medical Center
Pittsburgh 5206379, Pennsylvania 6254927 15224
Contact:
Joseph Pilewski
412-692-5873
pilewskijm@upmc.edu

Medical University of South Carolina
Charleston 4574324, South Carolina 4597040 29425
Contact:
Christina Mingora
8437923710
mingora@musc.edu

University of Virginia Cystic Fibrosis Center
Charlottesville 4752031, Virginia 6254928 22908
Contact:
Linsay Somerville
434-297-7773
LL7Y@hscmail.mcc.virginia.edu

More Details

NCT ID
NCT05639556
Status
Recruiting
Sponsor
Jaeb Center for Health Research

Study Contact

Judy Sibayan
(813) 975-8690
jsibayan@jaeb.org

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.