Pulmonary Vascular Disease in CF (NCT04549077) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Pulmonary Vascular Disease in CF
United States86 participantsStarted 2020-07-29
Plain-language summary
In this project, the investigators seek to understand the role of endothelial cells in Cystic Fibrosis (CF) lung disease. This objective will be achieved by conducting a cross sectional clinical study to define the morphology of the pulmonary circulation across a range of lung function coupled with a mechanistic study of the effect of dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) in endothelial cells on vasculogenesis, epithelial morphogenesis and epithelial CFTR function. Toward that end, the investigators propose the following hypotheses; (a). Loss of pulmonary small blood vessels begins early in the CF lung and worsens with disease progression, (b).VEGFR2-CFTR interactions happen at the plasma membrane of endothelial cells and is likely to be involved in transendothelial ion transport (c) impaired VEGFR2-CFTR interactions on the endothelial cells will have a profound effect on vasculogenesis, epithelial morphogenesis and ion transport.
The first hypotheses will be tested through this clinical study. The following 2 hypotheses will be tested through laboratory studies that do not involve human subjects.
Who can participate
Age range
5 Years – 21 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Cystic Fibrosis Patients:
Inclusion Criteria:
* 5-21 years of age
* diagnosis of CF based on a positive sweat test and genetic testing
* Baseline pulmonary condition defined as a) Absence of signs and symptoms of pulmonary exacerbation, b) Baseline pulmonary function test (PFT) defined as FEV1% that is no less than 5% of the best PFT in the previous 6 months, c) Patients should be off acute antibiotics for 2 weeks or longer.
* Subjects should be able to perform an acceptable and reproducible spirometry
* Study population will be equally divided between three groups based on FEV1%, (FEV1% ≥ 90); moderate (60 ≤ FEV1% \< 90)
Exclusion Criteria:
* Enrollment in clinical trials of CFTR correctors and or potentiator
* Enrollment in gene therapy trial
* Pregnancy.
Historical Controls
Inclusion Criteria:
* solid tumor diagnosis
* had chest CT scan to survey possible metastasis or any other lung disease
* age and gender matched to Cystic Fibrosis patients
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The ratio (BV5/TBV) of blood volume in small < 5mm2 blood vessels (BV5) to the total pulmonary blood volume (TBV).
Timeframe: At baseline
2
The change in the the ratio (BV5/TBV) of blood volume in small < 5mm2 blood vessels (BV5) to the total pulmonary blood volume (TBV).
Timeframe: For a subset of 31 patients, the change in ratio will be measured between the baseline visit and 6 months post Trikafta therapy.
Trial details
NCT IDNCT04549077
SponsorChildren's Hospital Medical Center, Cincinnati