ACTIVE_NOT_RECRUITING

Pulmonary Vascular Disease in CF

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Pulmonary Vascular Disease in Cystic Fibrosis

Quick Facts

Study Start:2020-07-29
Study Completion:2026-11-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04549077

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:5 Years to 21 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * 5-21 years of age
  2. * diagnosis of CF based on a positive sweat test and genetic testing
  3. * 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.
  4. * Subjects should be able to perform an acceptable and reproducible spirometry
  5. * Study population will be equally divided between three groups based on FEV1%, (FEV1% ≥ 90); moderate (60 ≤ FEV1% \< 90)
  1. * Enrollment in clinical trials of CFTR correctors and or potentiator
  2. * Enrollment in gene therapy trial
  3. * Pregnancy.

Contacts and Locations

Study Locations (Sites)

Riley Hospital for Children
Indianapolis, Indiana, 46204-3509
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3026
United States

Collaborators and Investigators

Sponsor: Children's Hospital Medical Center, Cincinnati

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-07-29
Study Completion Date2026-11-30

Study Record Updates

Study Start Date2020-07-29
Study Completion Date2026-11-30

Terms related to this study

Keywords Provided by Researchers

  • Cystic Fibrosis
  • Pulmonary Disease
  • Pediatric

Additional Relevant MeSH Terms

  • Cystic Fibrosis