Cystic FibrosisUnderstand-CFNCT05857709
Fitness, Body Composition, Physical Activity and Sleep Patterns in People With Cystic Fibrosis on Modulator Therapy.
An observational study of Cystic Fibrosis, run by University of Portsmouth at 1 site in United Kingdom.
Trial summary
Cystic fibrosis (CF) is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. It is well recognised that regular exercise is clinically important for people with CF. Read more about this study
- Who is it for?
- Ages 10 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 100 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 10 Jan 2023to Feb 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Participants will be recruited from adult and paediatric CF outpatient clinics within the Southampton CF network.
Who may be able to join
- Stable on the modulator therapy, Kaftrio®
- Males and females ≥ 10 years of age
- CF diagnosis based on clinical features, supported by a history of an abnormal sweat test (sweat chloride > 60 mmol·L-1 > 100 mg sweat), where possible, diagnostic genotyping would also be desired
- Can clearly state that they are not pregnant
- No contraindications to performing exercise
- Can understand and cooperate with the study protocol
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- No increase in symptoms or weight loss in the preceding 2 weeks
- Healthy males and females who are age- and sex-matched to the enrolled individuals with CF
- Can clearly state that they are not pregnant
- No clinical diagnosis of a chronic disease
- Can understand and cooperate with the study protocol
- No contraindications to performing exhaustive exercise
Who cannot join
- Any non-pulmonary conditions that may impair exercise ability, such as musculoskeletal disorders and cardiovascular disease.
- Unstable co-morbid asthma (daily pulmonary function variability of >20%)
- Is pregnant during the initial screening process
- Unable to understand or cooperate with the study protocol due to learning difficulties or otherwise
- Not of a suitable age for testing
- Is a smoker or inhales any other substances
Show all 16Show fewer
- Are taking vasoactive medications
- Any pulmonary, metabolic or cardiovascular conditions
- Any other diagnosed disease
- Any non-pulmonary conditions that may impair exercise ability, such as musculoskeletal disorders and cardiovascular disease
- Is pregnant during the initial screening procedure
- Presents with co-morbidities to performing exhaustive exercise
- Is a smoker or inhales any other substance
- Unable to understand or cooperate with the study protocol due to learning difficulties or otherwise
- Not an age- or sex-match for the CF group
- Are taking vasoactive medications
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
University of Portsmouth
Portsmouth, PO1 2EF
Zoe Saynor, 023 9284 3080 ext. 3080, zoe.saynor@port.ac.uk
Lauren Clayton, 023 9284 3085 ext. 3085, lauren.clayton@port.ac.uk
About this study
Official title: An Observational Study to Understand the Fitness, Body Composition, Physical Activity and Sleep Patterns in People With Cystic Fibrosis on Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy Compared to Healthy Controls
Cystic fibrosis (CF) is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. It is well recognised that regular exercise is clinically important for people with CF. Exercise function measured by the maximal oxygen consumption during a cardiopulmonary exercise test is often reduced in people with CF and this has been attributed to multiple factors including, altered heart and blood vessel function, muscle function, reduced physical activity levels and poorer sleep quality.
New medicine (modulators) have become available for many people with CF. Modulators appear able to reduce sweat chloride concentrations, improve lung function and reduce the frequency of pulmonary exacerbations in people with CF. Little evidence exists to show how they may have changed the fitness and underlying mechanisms responsible for this in people with CF.
Read more
This study aims to:
- determine the exercise function
- determine the blood vessel function
- determine body composition
- determine physical activity and sleep levels in people with CF on modulator therapy compared to a healthy controls group.
Read the detailed description
CF is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. CF affects the movement of salt and water across the body which leads to a thick sticky build up of mucus causing problems in the lungs and digestive system. CF also causes problems in other parts of the body for example, the heart, the blood vessels and muscles. This can impact a person with CFs' ability to exercise which can have impact prognosis, quality of life and increase the amount of times someone with CF is admitted to hospital due to a chest infection.
For many people with CF, new medicine (modulators) have lately become accessible bringing big changes to their health. Elexacaftor-tezacaftor-ivacaftor (ETI) is the most recently approved modulator for use in people with specific CF transmembrane conductance regulator gene mutations and is now the most widely used modulator therapy. So far clinical trials have largely looked at lung function, sweat chloride levels and body mass index. The wider reaching effects of modulator therapy on exercise function has not been studied. This study will see if people with CF have blood vessel and exercise dysfunction, abnormal body composition and reduced physical activity and sleep quality when they are taking modulator therapy compared to a healthy group.
The aims of this study are:
- Determine the aerobic exercise function and mechanisms underlying this in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the peripheral muscle function in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the functional exercise capacity in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the micro- and macrovascular endothelial function in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine body composition in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the physical activity levels and sleep duration and quality in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls
This study will recruit 50 people with CF and 50 healthy age- and sex-matched control participants who are older than 10 years of age. It will ask them to attend the University of Portsmouth for 2 visits. The first visit will last ~3 hours. During this time they will undergo measures on blood vessel function and aerobic exercise function. The second visit will last ~2 hours. During this time they will undergo a body composition scan and a series of muscle function test. At home, participants will wear an accelerometer for 7 days and complete a series of questionnaires.
- Conditions
- Cystic Fibrosis
- Keywords
- Exercise, Modulator therapy, Peripheral muscle function, Vascular function, Body composition, Physical activity
- Medical subject headings
- Cystic Fibrosis, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystic fibrosis | No intervention - only assessments. | |
| Healthy Control | No intervention - only assessments. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximal oxygen uptake
Between group differences in maximal oxygen uptake derived from a maximal cardiopulmonary exercise testing on a cycle ergometer
Measured: Day 1 - baseline
Secondary outcomes (24)
Peak minute ventilation
Between group differences in peak minute ventilation derived from a maximal cardiopulmonary exercise test on a cycle ergometer
Measured: Day 1 - baseline
Oxygen uptake at the gas exchange threshold
Between group differences in oxygen uptake at the gas exchange threshold derived from a maximal cardiopulmonary exercise test on a cycle ergometer
Measured: Day 1 - baseline
Peak power output
Between group differences in peak power output derived from a maximal cardiopulmonary exercise test on a cycle ergometer
Measured: Day 1 - baseline
Time to exhaustion
Between group differences in time to exhaustion derived from a maximal cardiopulmonary exercise test on a cycle ergometer
Measured: Day 1 - baseline
Heart rate
Between group differences in heart rate derived from a maximal cardiopulmonary exercise test on a cycle ergometer
Measured: Day 1 - baseline
Near-infrared spectroscopy derived deoxygenated [haemoglobin + myoglobin]
Between group differences in dynamics of near-infrared spectroscopy derived deoxygenated [haemoglobin + myoglobin]
Measured: Day 1 - baseline
Cardiac output
Between group differences in dynamics of non-invasive thoracic impedance cardiography
Measured: Day 1 - baseline
Stroke volume index
Between group differences in dynamics of non-invasive thoracic impedance cardiography
Measured: Day 1 - baseline
Forced Expiratory Volume in the 1st second (% predicted)
Between group differences in Forced Expiratory Volume in the 1st second measured using flow-volume loop spirometry
Measured: Day 1 - baseline
Forced Vital Capacity (%predicted)
Between group differences in Forced Vital Capacity measured using flow-volume loop spirometry
Measured: Day 1 - baseline
Total minutes of physical activity (light, moderate, moderate to vigorous physical activity)
Between group differences in physical activity assessed via wrist-worn accelerometery
Measured: Physical activity will be measured continuously for 7 days
Sleep efficiency (time in bed + time asleep)
Between group differences in sleep efficiency assessed via wrist-worn accelerometery
Measured: Sleep efficiency will be measured continuously for 7 days
Acetylcholine iontophoresis
Between group differences in acetylcholine iontophoresis measure of microvascular function
Measured: Day 1 - baseline
Insulin iontophoresis
Between group differences in insulin iontophoresis measure of microvascular function
Measured: Day 1 - baseline
Percentage change in brachial artery diameter taken from the Flow Mediated Dilation assessment
Between group differences in Flow Mediated Dilation assessment derived from baseline brachial artery diameter and peak brachial artery diameter from flow mediated dilation measure of macrovascular function
Measured: Day 1 - baseline
Maximum quadricep strength
Derived from a maximal voluntary contraction
Measured: Day 2 - baseline
Quadricep fatigability index
Derived from change in maximal voluntary contraction
Measured: Day 2 - baseline
Handgrip strength
Between group difference in handgrip strength
Measured: Day 2 - baseline
Sit to stand repetitions per minute
Between group difference in sit to stand repetitions per minute
Measured: Day 2 - baseline
Body composition (total fat-free mass and total fat mass)
Derived from dual-energy X-ray absorptiometry
Measured: Day 2 - baseline
Habitual activity estimation scale
Between group difference in habitual activity estimation scale - answers are varied and require numerical values, percentages and/or multiple choice questions
Measured: Day 2 - baseline
Cystic fibrosis questionnaire revised
Between group difference in health related quality of life on a 0 - 100 scale with higher scores indicating better health related quality of life
Measured: Day 2 - baseline
The Pittsburgh Sleep Quality Index
Between group differences in the Pittsburgh Sleep Quality Index on a 0 - 21 scale with higher scores indicating worse sleep quality
Measured: Day 2 - baseline
Perceived exertion (breathing effort, chest tightness, throat narrowing, perceived exertion)
Between group differences in perceived exertion using the Dalhousie Dyspnoea and Perceived Exertion Scales during a maximal cardiopulmonary exercise testing on a cycle ergometer on a 1 - 7 scale with a higher value indicating a worse perceived exertion
Measured: Day 1 - baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2023
- Study start10 Jan 2023
- Primary completionFeb 2024 (estimated)
- Study completionFeb 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Portsmouth
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital Southampton NHS Foundation Trust
- Responsible party
- Principal investigator, Dr Zoe Saynor, University of Portsmouth
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05857709
- Sponsor's protocol ID
- 313567
- Acronym
- Understand-CF
- Status last verified
- May 2023
- First submitted
- 23 Jan 2023
- Last updated
- 18 May 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study to Understand the Fitness, Body Composition, Physical Activity and Sleep Patterns in People With Cystic Fibrosis on Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy Compared to Healthy Controls
- ClinicalTrials.gov ID
- NCT05857709
- Acronym / sponsor ID
- Understand-CF / 313567
- Conditions
- Cystic Fibrosis
- Keywords
- Exercise, Modulator therapy, Peripheral muscle function, Vascular function, Body composition, Physical activity
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University of PortsmouthUniversity, hospital or other
- Collaborators
- University Hospital Southampton NHS Foundation Trust
- Study start
- 10 Jan 2023 (actual)
- Primary completion
- Feb 2024 (estimated)
- Study completion
- Feb 2024 (estimated)
- First posted
- 15 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cystic fibrosis (CF) is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. It is well recognised that regular exercise is clinically important for people with CF. Exercise function measured by the maximal oxygen consumption during a cardiopulmonary exercise test is often reduced in people with CF and this has been attributed to multiple factors including, altered heart and blood vessel function, muscle function, reduced physical activity levels and poorer sleep quality.
New medicine (modulators) have become available for many people with CF. Modulators appear able to reduce sweat chloride concentrations, improve lung function and reduce the frequency of pulmonary exacerbations in people with CF. Little evidence exists to show how they may have changed the fitness and underlying mechanisms responsible for this in people with CF.
This study aims to:
- determine the exercise function
- determine the blood vessel function
- determine body composition
- determine physical activity and sleep levels in people with CF on modulator therapy compared to a healthy controls group.
Detailed description
CF is the most common inherited condition in the United Kingdom, affecting approximately 10,837 people. CF affects the movement of salt and water across the body which leads to a thick sticky build up of mucus causing problems in the lungs and digestive system. CF also causes problems in other parts of the body for example, the heart, the blood vessels and muscles. This can impact a person with CFs' ability to exercise which can have impact prognosis, quality of life and increase the amount of times someone with CF is admitted to hospital due to a chest infection.
For many people with CF, new medicine (modulators) have lately become accessible bringing big changes to their health. Elexacaftor-tezacaftor-ivacaftor (ETI) is the most recently approved modulator for use in people with specific CF transmembrane conductance regulator gene mutations and is now the most widely used modulator therapy. So far clinical trials have largely looked at lung function, sweat chloride levels and body mass index. The wider reaching effects of modulator therapy on exercise function has not been studied. This study will see if people with CF have blood vessel and exercise dysfunction, abnormal body composition and reduced physical activity and sleep quality when they are taking modulator therapy compared to a healthy group.
The aims of this study are:
- Determine the aerobic exercise function and mechanisms underlying this in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the peripheral muscle function in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the functional exercise capacity in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the micro- and macrovascular endothelial function in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine body composition in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls;
- Determine the physical activity levels and sleep duration and quality in people with CF stable on ETI and whether this is different to healthy age- and sex-matched controls
This study will recruit 50 people with CF and 50 healthy age- and sex-matched control participants who are older than 10 years of age. It will ask them to attend the University of Portsmouth for 2 visits. The first visit will last ~3 hours. During this time they will undergo measures on blood vessel function and aerobic exercise function. The second visit will last ~2 hours. During this time they will undergo a body composition scan and a series of muscle function test. At home, participants will wear an accelerometer for 7 days and complete a series of questionnaires.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cystic fibrosis | – | –No intervention - only assessments. |
| Healthy Control | – | –No intervention - only assessments. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be recruited from adult and paediatric CF outpatient clinics within the Southampton CF network.
Key inclusion criteria (12 listed)
- Stable on the modulator therapy, Kaftrio®
- Males and females ≥ 10 years of age
- CF diagnosis based on clinical features, supported by a history of an abnormal sweat test (sweat chloride > 60 mmol·L-1 > 100 mg sweat), where possible, diagnostic genotyping would also be desired
- Can clearly state that they are not pregnant
- No contraindications to performing exercise
Key exclusion criteria (16 listed)
- Any non-pulmonary conditions that may impair exercise ability, such as musculoskeletal disorders and cardiovascular disease.
- Unstable co-morbid asthma (daily pulmonary function variability of >20%)
- Is pregnant during the initial screening process
- Unable to understand or cooperate with the study protocol due to learning difficulties or otherwise
- Not of a suitable age for testing
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Maximal oxygen uptake
Time frame: Day 1 - baseline
Between group differences in maximal oxygen uptake derived from a maximal cardiopulmonary exercise testing on a cycle ergometer
24 secondary outcomes
Peak minute ventilation
Time frame: Day 1 - baseline
Oxygen uptake at the gas exchange threshold
Time frame: Day 1 - baseline
Peak power output
Time frame: Day 1 - baseline
Time to exhaustion
Time frame: Day 1 - baseline
Heart rate
Time frame: Day 1 - baseline
Near-infrared spectroscopy derived deoxygenated [haemoglobin + myoglobin]
Time frame: Day 1 - baseline
Cardiac output
Time frame: Day 1 - baseline
Stroke volume index
Time frame: Day 1 - baseline
Forced Expiratory Volume in the 1st second (% predicted)
Time frame: Day 1 - baseline
Forced Vital Capacity (%predicted)
Time frame: Day 1 - baseline
Total minutes of physical activity (light, moderate, moderate to vigorous physical activity)
Time frame: Physical activity will be measured continuously for 7 days
Sleep efficiency (time in bed + time asleep)
Time frame: Sleep efficiency will be measured continuously for 7 days
Acetylcholine iontophoresis
Time frame: Day 1 - baseline
Insulin iontophoresis
Time frame: Day 1 - baseline
Percentage change in brachial artery diameter taken from the Flow Mediated Dilation assessment
Time frame: Day 1 - baseline
Maximum quadricep strength
Time frame: Day 2 - baseline
Quadricep fatigability index
Time frame: Day 2 - baseline
Handgrip strength
Time frame: Day 2 - baseline
Sit to stand repetitions per minute
Time frame: Day 2 - baseline
Body composition (total fat-free mass and total fat mass)
Time frame: Day 2 - baseline
Habitual activity estimation scale
Time frame: Day 2 - baseline
Cystic fibrosis questionnaire revised
Time frame: Day 2 - baseline
The Pittsburgh Sleep Quality Index
Time frame: Day 2 - baseline
Perceived exertion (breathing effort, chest tightness, throat narrowing, perceived exertion)
Time frame: Day 1 - baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by University of Portsmouth at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Zoe Saynor (02392843080, zoe.saynor@port.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximal oxygen uptake (time frame: Day 1 - baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2023.