Understanding pulmonary vascular disease in cystic fibrosis

Pulmonary Vascular Disease in Cystic Fibrosis

This study is trying to understand how problems with blood vessels in the lungs affect people with cystic fibrosis and how it relates to their overall lung health.

Quick facts

• Study type: Observational
• Enrollment: 193 (estimated)
• Ages: 5 Years to 21 Years
• Sex: All
• Sponsor: Children's Hospital Medical Center, Cincinnati (Academic / other)
• Locations: 2 sites (Indianapolis, Indiana and 1 other locations)
• Trial ID: NCT04549077 on ClinicalTrials.gov

What this trial studies

This observational study aims to investigate the role of endothelial cells in lung disease associated with cystic fibrosis (CF). Researchers will conduct a cross-sectional analysis to assess the morphology of the pulmonary circulation in CF patients, focusing on the impact of dysfunctional CFTR on vascular and epithelial functions. The study will test hypotheses regarding the early loss of small pulmonary blood vessels and the interactions between VEGFR2 and CFTR in endothelial cells. By utilizing advanced imaging techniques, the study seeks to clarify the relationship between pulmonary vascular changes and lung function in CF.

Who should consider this trial

Good fit: Ideal candidates include cystic fibrosis patients aged 5-21 years with stable pulmonary function.

Not a fit: Patients currently enrolled in clinical trials for CFTR correctors or gene therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved understanding and management of pulmonary complications in cystic fibrosis patients.

How similar studies have performed: While this approach is novel in its specific focus on endothelial cell interactions in CF, similar studies have shown promising results in understanding pulmonary vascular changes in other conditions.

Eligibility criteria

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

Where this trial is running

Indianapolis, Indiana and 1 other locations

• Riley Hospital for Children — Indianapolis, Indiana, United States (Recruiting)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)

Study contacts

• Study coordinator: Raouf Amin, MD

Conditions Cystic Fibrosis; Pulmonary Disease; Pediatric