Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus Trial (The MILES Trial)

INTERVENTION: Intervention type:DRUG. Intervention1:Sirolimus, Dose form:TABLET, Route of administration:ORAL, intended dose regimen:12 months on, 12 months off. Control intervention1:Placebo, Dose form:TABLET, Route of administration:ORAL, Intended dose regimen:12 months on, 12 months off. CONDITION: Lymphangioleiomyomatosis (LAM) PRIMARY OUTCOME: FEV1 response. Timepoint:Every three months the first year and then every six months the second year. SECONDARY OUTCOME: FVC response, six minute walk, diffusing capacity, lung volumes, dyspnea (shortness of breath) scores by questionnaire, mortality, 3 dimensional CT, adverse events. Timepoint: during September‐November, 2010. INCLUSION CRITERIA: 1 Age 18 or over 2 Signed and dated informed consent 3 Diagnosis of LAM as determined by biopsy; or chest CT scan findings compatible with LAM in the setting of tuberous sclerosis, angiomyolipoma or chylous pleural effusion 4 Abnormal lung function i.e., postbronchodilator FEV1 less than or equal to 70% of predicted