Cystic FibrosisNCT03338595
Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF
An observational study of Cystic Fibrosis, run by Augusta University at 1 site in United States.
Trial summary
This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del. Our goal is to perform the exercise and vascular measurements every 3 months after a patient starts taking Orkambi. Read more about this study
- Who is it for?
- Ages 7 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to Oct 2020
- 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 7 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: CF patients who are homozygous F508del and have been prescribed Orkambi.
Who may be able to join
- Patients diagnosed with Cystic Fibrosis (homozygous deltaF508del)
- Prescribed Orkambi
- Men and women (> 18 yrs. old)
- Boys and girls (7 -17 yrs. old)
- FEV1 percent predicted > 40%
- Resting oxygen saturation (room air) >85%
Show all 10Show fewer
- Patients with or without CFRD
- Traditional CF-treatment medications
- Clinically stable for past 28 days (no exacerbations or change in medical status)
- Healthy Controls
Who cannot join
- Children 6 yrs. old and younger
- FEV1 percent predicted < 40%
- Resting oxygen saturation (room air) < 85%
- Clinical diagnosis of heart disease
- Pulmonary artery hypertension
- Febrile illness within two weeks of visit
Show all 12Show fewer
- Current smokers
- Currently pregnant or nursing
- Individuals on vaso-active medications (i.e. nitrates, beta blockers, ACE inhibitors, etc.)
- Use of VX-770 within 6 months prior to Visit 1
- History of solid organ transplantation
- Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the patient or the quality of the data.
Trial locations and contacts
1 site in 1 country.
United States1 site
Georgia Prevention Institute
Augusta, Georgia, 30912
About this study
This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del. Our goal is to perform the exercise and vascular measurements every 3 months after a patient starts taking Orkambi.
Read the detailed description
Cystic Fibrosis (CF) is the most common fatal genetic disease in North America. The most disturbing aspect of CF is the associated premature death, most often due to respiratory complications. Clinical manifestations of CF include not only lung dysfunction, but many other systemic consequences as well. Systemic oxidative stress and exercise intolerance are established phenotypes in patients with CF. Additionally, for the first time the investigators have recently published the presence of systemic endothelial dysfunction in a cohort of young patients with CF who exhibited normal oxygen saturation and spirometric function.
Exercise intolerance, the limitation of the ability to perform exercise at the expected level, has been shown to predict mortality in patients with CF independent of lung function. Exercise capacity (VO2 peak), an objective measurement of exercise tolerance, drops approximately 5-8% per year in patients with CF. This excessive decay in exercise capacity not only leads to more pulmonary infections and deterioration of lung function, it represents a 5-8 fold decline compared to healthy sedentary adults. Preventing the excessive annual reduction in exercise capacity is essential to increasing the quality of life and longevity of patients with CF. However, a critical barrier to improving exercise capacity in CF is the investigators lack of knowledge regarding the different physiological mechanisms that contribute to exercise intolerance. It is important to emphasize that decreases in lung function (FEV1) do not always contribute to reductions in VO2 peak. Furthermore, less than 2% of patients who have an FEV1 greater than 50% predicted will have a significant drop in hemoglobin oxygen saturation (SpO2) during maximal exercise. These data suggest that mechanisms other than lung function induced hypoxemia may be contributing to exercise intolerance in patients with CF.
- Conditions
- Cystic Fibrosis
- Keywords
- arterial stiffness, flow-mediated dilation, endothelial function, Orkambi, exercise capacity
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximal exercise capacity
Subjects will perform the maximal exercise tests on an electronically braked cycle ergometer using the Godfrey protocol. Expired gases will be collected using a Parvo Medics True One metabolic cart for determination of exercise capacity (VO2 peak).
Measured: 1 year
Secondary outcomes (1)
Flow mediated dilation
The brachial artery FMD test will be performed according to the recent tutorial on the ultrasonic assessment of FMD and shear rate will be calculated as the stimulus of the vasodilatory response. Briefly, subjects will lie in the supine position for 20 minutes to obtain hemodynamic steady state. A blood pressure cuff (Hokanson) will be placed around the forearm (distal to the Doppler transducer) and rapidly inflated to 250 mmHg for 5 minutes (circulatory arrest). Simultaneous ultrasound images of the vessel (B-mode) and Doppler waveforms will be collected 10 seconds prior to and for 2 minutes following deflation of the cuff.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2017
- Study startMay 2014
- Primary completionOct 2020
- Study completionOct 2020
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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Augusta University
- Sponsor type
- University, hospital or other
- Collaborators
- Vertex Pharmaceuticals Incorporated
- Responsible party
- Principal investigator, Ryan Harris, Augusta University
- Investigators
- Ryan Harris, PhD, Principal investigator, Augusta University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03338595
- Sponsor's protocol ID
- CF-Long
- Status last verified
- Jan 2021
- First submitted
- 31 Mar 2017
- Last updated
- 19 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF
- ClinicalTrials.gov ID
- NCT03338595
- Acronym / sponsor ID
- CF-Long
- Conditions
- Cystic Fibrosis
- Keywords
- arterial stiffness, flow-mediated dilation, endothelial function, Orkambi, exercise capacity
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Augusta UniversityUniversity, hospital or other
- Collaborators
- Vertex Pharmaceuticals Incorporated
- Study start
- May 2014
- Primary completion
- Oct 2020 (actual)
- Study completion
- Oct 2020 (actual)
- First posted
- 9 Nov 2017 (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.
This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del. Our goal is to perform the exercise and vascular measurements every 3 months after a patient starts taking Orkambi.
Detailed description
Cystic Fibrosis (CF) is the most common fatal genetic disease in North America. The most disturbing aspect of CF is the associated premature death, most often due to respiratory complications. Clinical manifestations of CF include not only lung dysfunction, but many other systemic consequences as well. Systemic oxidative stress and exercise intolerance are established phenotypes in patients with CF. Additionally, for the first time the investigators have recently published the presence of systemic endothelial dysfunction in a cohort of young patients with CF who exhibited normal oxygen saturation and spirometric function.
Exercise intolerance, the limitation of the ability to perform exercise at the expected level, has been shown to predict mortality in patients with CF independent of lung function. Exercise capacity (VO2 peak), an objective measurement of exercise tolerance, drops approximately 5-8% per year in patients with CF. This excessive decay in exercise capacity not only leads to more pulmonary infections and deterioration of lung function, it represents a 5-8 fold decline compared to healthy sedentary adults. Preventing the excessive annual reduction in exercise capacity is essential to increasing the quality of life and longevity of patients with CF. However, a critical barrier to improving exercise capacity in CF is the investigators lack of knowledge regarding the different physiological mechanisms that contribute to exercise intolerance. It is important to emphasize that decreases in lung function (FEV1) do not always contribute to reductions in VO2 peak. Furthermore, less than 2% of patients who have an FEV1 greater than 50% predicted will have a significant drop in hemoglobin oxygen saturation (SpO2) during maximal exercise. These data suggest that mechanisms other than lung function induced hypoxemia may be contributing to exercise intolerance in patients with CF.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- CF patients who are homozygous F508del and have been prescribed Orkambi.
Key inclusion criteria (10 listed)
- Patients diagnosed with Cystic Fibrosis (homozygous deltaF508del)
- Prescribed Orkambi
- Men and women (> 18 yrs. old)
- Boys and girls (7 -17 yrs. old)
- FEV1 percent predicted > 40%
Key exclusion criteria (12 listed)
- Children 6 yrs. old and younger
- FEV1 percent predicted < 40%
- Resting oxygen saturation (room air) < 85%
- Clinical diagnosis of heart disease
- Pulmonary artery hypertension
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 exercise capacity
Time frame: 1 year
Subjects will perform the maximal exercise tests on an electronically braked cycle ergometer using the Godfrey protocol. Expired gases will be collected using a Parvo Medics True One metabolic cart for determination of exercise capacity (VO2 peak).
1 secondary outcome
Flow mediated dilation
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2021.
- Individual participant dataSharing planned: Yes
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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.
Ryan Harris, PhD principal investigator
Augusta University
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 Augusta University at 1 site in United States.
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 10 inclusion and 12 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: finished, as of the sponsor's last update on 19 Jan 2021. 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 States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximal exercise capacity (time frame: 1 year). 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 19 Jan 2021.