Sildenafil Trial in Children and Young Adults With CF
Status:
Withdrawn
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
Cystic Fibrosis (CF), the most common inherited disease in Caucasians, is characterized by
chronic pulmonary inflammation and progressive loss of gas exchange units that eventually
results in respiratory failure. There is strong evidence that, in CF, abnormally low
perfusion carries a high risk of death independent from the presence of pulmonary
hypertension. However, the evolution of pulmonary vascular disease in CF and how it might
contribute to the rate of decline in lung function is not known. Our knowledge remains
limited to the results of old observational studies which concluded that the major causes of
pulmonary vascular remodeling and hypertension in CF are hypoxic respiratory failure and
destruction of lung tissue. Our recent data obtained by state-of-the-art Magnetic Resonance
Imaging (MRI) of the pulmonary circulation, challenges the existing paradigm. We demonstrate
that in the absence of hypoxia, significant changes in pulmonary perfusion and in surrogate
measures of vascular resistance as well as in collateral blood flow begin early in the course
of CF. Newly developed therapeutics have altered dramatically the course of patients
suffering from pulmonary vascular disease. Through this 8 week trial, we will examine by
Magnetic Resonance Imaging the effect of Sildenafil on pulmonary perfusion and systemic
vascularization of the lungs in subjects with mild to moderate disease.