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Efficacy and Safety of Sirolimus for Treating Lymphangioleiomyomatosis (LAM)


Lymphangioleiomyomatosis Efficacy and Safety Trial




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.

Study Status: Active, not recruiting


Condition Intervention Phase
Lymphangioleiomyomatosis Drug: Sirolimus
Drug: Placebo sirolimus
Phase 3

Verified by Office of Rare Diseases (ORD) August, 2009

Sponsored by: Office of Rare Diseases (ORD)
Information provided by: Office of Rare Diseases (ORD) identifier: NCT00414648

Study Type: Interventional

Study Design: Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Outcomes Assessor), Primary Purpose: Treatment

Cleveland Clinic Foundation
Cleveland, Ohio 44195
United States

Frank McCormack, MD., Principal Investigator
Bruce Trapnell, MD., Principal Investigator

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