Efficacy and Safety of Sirolimus for Treating Lymphangioleiomyomatosis (LAM)
Status:
Unknown status
Trial end date:
2011-09-01
Target enrollment:
Participant gender:
Summary
Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It
results in the uncontrolled growth and proliferation of an unusual type of smooth muscle
cell. These cells invade lung tissue, including the airways, blood vessels, and lymph
vessels, and restrict the flow of air, blood, and lymph, respectively. Respiratory failure,
lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the
disease. This study will evaluate the safety and effectiveness of sirolimus, an
immunosuppressive medication, in stabilizing or improving lung function in people with LAM.