Cystic Fibrosis
Conditions
Brief summary
The main objective of this clinical pilot study is to evaluate the feasibility and impact of a home-based exercise program on clinical and patient-centered outcomes in adult with cystic fibrosis (CF) and inform the design of a larger clinical trial.
Detailed description
This pilot and feasibility study is a randomized controlled trial comparing exercise intervention with a usual care group. Participants will be randomized based on a randomization table developed by a biostatistician who is not involved in the study. The exercise intervention group will be delivered with a telehealth platform using internet (Zoom HIPAA-Compliant video), an app, phone, and email/text support. Participants will be randomly allocated to either an exercise coaching program or a usual care arm. The exercise intervention arm will follow guidelines established by the American Association of Cardiovascular and Pulmonary Rehabilitation (AACVPR) and complement best clinical practices established by the American Thoracic Society/European Respiratory Society (ATS/ERS). The usual care arm will be provided a handout on resources for engaging in physical activity. Aim 1. Examine the feasibility of a home-based Tele-rehab intervention. Indicators of feasibility will include recruitment and adherence. Hypothesis 1: Based on previous trials, the investigators predict that \>50% of eligible participants will consent to the study. Hypothesis 2: Again, based on previous trials, \>70% of participants in the intervention arm will adhere to \>70% of their weekly prescribed exercise. Also, the investigators will assess reasons for; 1) eligible patients who don't enroll, 2) participants' non-adherence, and 3) non-completers. Aim 2. Evaluate the impact of a home-based exercise program in adults with CF for improving clinical and patient-centered outcomes. Hypothesis 1: There will be a greater improvement in exercise capacity (measured by a modified shuttle test), a novel measure of lung function 129Xenon MRI (129Xe MRI), forced expiratory volume in 1 second (FEV1), and quality of life with participants in the intervention arm when compared to usual care. Hypothesis 2: Changes in exercise capacity and 129Xe MRI will be associated with exercise adherence, as noted in Aim 1.
Interventions
DIAGNOSTIC_TESTXenon MRI
Study participants will be randomized to a 12 week exercise intervention versus usual care. Participants will have Xenon MRI at study start and completion to evaluate impact of exercise on lung function. The exercise intervention for all participants will be delivered by two pulmonary rehabilitation coaches (PR-coach) including a registered respiratory therapist and clinical registered dietitian who've been trained in exercise training as recommended by the AACVPR and American College of Sports Medicine (ACSM). Each TR participant will be assigned a PR-coach, and receive one weekly exercise consulting session during the 12-week intervention.
Sponsors
University of Kansas Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* cystic fibrosis patients 18 years of age and older, confirmed diagnosis of CF (two CF mutations or sweat chloride \> 60 mmol/L) * stable while either on/off Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy and no plan to start/discontinue CFTR modulator therapy * clearance from their CF physician to participate in exercise * have access to the internet * not involved in an exercise intervention in the previous 6 months, and not performing structured exercise \> 150 minutes per week.
Exclusion criteria
* pregnancy * history of solid organ transplant * active treatment for mycobacterial infections * significant untreated hypoxemia, oxygen dependent at rest or with exercise * FEV1 \< 40% of predicted or clinical evidence of cor pulmonale * untreated arterial hypertension (resting systolic blood pressure \>140 mm Hg, diastolic blood pressure \> 90 mmHg) * systolic blood pressure less than 90 mm Hg while standing * congestive heart failure * active treatment for Allergic Bronchopulmonary Aspergillosis (ABPA) * acute upper or lower respiratory infection or pulmonary exacerbation within 4 weeks prior to Day 1 * changes in therapy (including antibiotics) for pulmonary disease within 4 weeks prior to Day 1 * significant hemoptysis within 4 weeks prior to Day 1 (≥ 5 mL of blood in one coughing episode or \> 30 mL of blood in a 24 hour period * ongoing participation in an investigational drug study within 60 days prior to Day 1
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cardiorespiratory Fitness Assessment- Modified Shuttle Walk Test Result | 3 months | change in number of modified shuttles completed between baseline/enrollment and study completion at 3 months |
| Ventilation Defect Percentage (VDP) as Detected by 129Xenon MRI | 3 months | Hyperpolarized 129Xe ventilation MRI will be performed to assess VDP at baseline (within 2 weeks of initiation of study) and after completion of the intervention/usual care-control period |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Forced Expiratory Volume in 1 Second | 3 months | lung function will be assessed at baseline/enrollment and study completion at 3 months |
| Quality of Life Assessment. | 3 months | Cystic Fibrosis questionnaire-revised (CFQR) will be assessed at baseline/enrollment and study completion at 3 months. Scale 0-100 (high score indicates better quality of life) |
| Exercise Time | 3 months | assess weekly adherence to prescribed exercise time completed |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Xenon MRI Only, Without Exerercise Intervention Participants will receive usual standard of care and undergo a xenon MRI.
Xenon MRI: Study participants will be randomized to a 12 week exercise intervention versus usual care. Participants will have Xenon MRI at study start and completion to evaluate impact of exercise on lung function. The exercise intervention for all participants will be delivered by two pulmonary rehabilitation coaches (PR-coach) including a registered respiratory therapist and clinical registered dietitian who've been trained in exercise training as recommended by the AACVPR and American College of Sports Medicine (ACSM). Each TR participant will be assigned a PR-coach, and receive one weekly exercise consulting session during the 12-week intervention. | 9 |
| Xenon MRI With Exercise Coaching Participants will receive weekly coaching intervention and undergo a xenon MRI
Xenon MRI: Study participants will be randomized to a 12 week exercise intervention versus usual care. Participants will have Xenon MRI at study start and completion to evaluate impact of exercise on lung function. The exercise intervention for all participants will be delivered by two pulmonary rehabilitation coaches (PR-coach) including a registered respiratory therapist and clinical registered dietitian who've been trained in exercise training as recommended by the AACVPR and American College of Sports Medicine (ACSM). Each TR participant will be assigned a PR-coach, and receive one weekly exercise consulting session during the 12-week intervention. | 12 |
| Total | 21 |
Baseline characteristics
| Characteristic | Xenon MRI With Exercise Coaching | Total | Xenon MRI Only, Without Exerercise Intervention |
|---|---|---|---|
| Age, Continuous | 32 years | 30.6 years | 28 years |
| Race and Ethnicity Not Collected | — | 0 Participants | — |
| Region of Enrollment United States | 12 participants | 21 participants | 9 participants |
| Sex: Female, Male Female | 7 Participants | 8 Participants | 1 Participants |
| Sex: Female, Male Male | 5 Participants | 13 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 12 |
| other Total, other adverse events | 0 / 9 | 0 / 12 |
| serious Total, serious adverse events | 0 / 9 | 0 / 12 |
Outcome results
Primary
Cardiorespiratory Fitness Assessment- Modified Shuttle Walk Test Result
change in number of modified shuttles completed between baseline/enrollment and study completion at 3 months
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xenon MRI Only, Without Exerercise Intervention | Cardiorespiratory Fitness Assessment- Modified Shuttle Walk Test Result | -6.67 change shuttles (1 shuttle is 10 meters) | Standard Deviation 9.33 |
| Xenon MRI With Exercise Coaching | Cardiorespiratory Fitness Assessment- Modified Shuttle Walk Test Result | -7.63 change shuttles (1 shuttle is 10 meters) | Standard Deviation 9.84 |
Primary
Ventilation Defect Percentage (VDP) as Detected by 129Xenon MRI
Hyperpolarized 129Xe ventilation MRI will be performed to assess VDP at baseline (within 2 weeks of initiation of study) and after completion of the intervention/usual care-control period
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xenon MRI Only, Without Exerercise Intervention | Ventilation Defect Percentage (VDP) as Detected by 129Xenon MRI | 20.7 percentage of lung with low ventilation | Standard Deviation 9 |
| Xenon MRI With Exercise Coaching | Ventilation Defect Percentage (VDP) as Detected by 129Xenon MRI | 24.2 percentage of lung with low ventilation | Standard Deviation 12.6 |
Secondary
Exercise Time
assess weekly adherence to prescribed exercise time completed
Time frame: 3 months
Population: Participants in the Xenon MRI only without exercise intervention group did not record exercise minutes as they did not have exercise coaching intervention
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Xenon MRI Only, Without Exerercise Intervention | Exercise Time | 1290 Minutes |
Secondary
Forced Expiratory Volume in 1 Second
lung function will be assessed at baseline/enrollment and study completion at 3 months
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xenon MRI Only, Without Exerercise Intervention | Forced Expiratory Volume in 1 Second | 1.2 change pre/post FEV1 percent predicted | Standard Deviation 4.66 |
| Xenon MRI With Exercise Coaching | Forced Expiratory Volume in 1 Second | -0.38 change pre/post FEV1 percent predicted | Standard Deviation 3.42 |
Secondary
Quality of Life Assessment.
Cystic Fibrosis questionnaire-revised (CFQR) will be assessed at baseline/enrollment and study completion at 3 months. Scale 0-100 (high score indicates better quality of life)
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xenon MRI Only, Without Exerercise Intervention | Quality of Life Assessment. | -2.0 change pre/post CFQR score | Standard Deviation 10.82 |
| Xenon MRI With Exercise Coaching | Quality of Life Assessment. | -4.91 change pre/post CFQR score | Standard Deviation 7.06 |