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.
Cohort 1: Patients are eligible if their percentage of predicated forced expiratory
volume in1 second (FEV1) is 60% or lower at screening.
- Cohort 2: Patients are eligible if their percentage of predicted forced expiratory
volume in 1 second (FEV1) is 60% or greater at screening.
- 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 Visit 1
- No initiation of new chronic therapy (e.g., ibuprofen, azithromycin, inhaled
tobramycin, Cayston, CFTR modulator) within 28 days prior to and including Visit 1.
- 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
Visit 1.
- 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
Arm
Description
Assigned Intervention
Cohort 1
Forced expiratory volume in 1 second (FEV1) <60% 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.
Cohort 2
FEV1 ≥60% 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.
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.
