A Study to Determine the Effectiveness of Escalating Doses of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Female aged >/= 18 years with a diagnosis of LAM
Pulmonary function abnormalities as follows:
FEV1 of ≤ 80% of the predicted value following administration of a standard dose of a short acting β2-agonist (*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) OR
FEV1 < 90% of the predicted value of bronchodilator following administration of a standard dose of a short acting β2-agonist (*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) and DLco (uncorrected) <80% predicted.
Female patients including those of childbearing potential will be included in this study.
Negative pregnancy test at screening and baseline
FEV1<50% of predicted post-bronchodilator.
Change in FVC (ml) > ± 15% of screening value at baseline visit (not less than 14d after screening visit).
Use of any medicine containing estrogen in the 4 months prior to the screening visit and for the duration of the study
Significant hematologic, renal, hepatic laboratory abnormality or amylase > 1.5x the upper limit of the normal range at the screening or baseline visits
Fasting blood glucose > 126mg/dl or random blood glucose >200mg/dl at screening and/or baseline
Recent surgery (involving entry into a body cavity or requiring sutures) within 2 months of the screening visit or any evidence of unhealed surgical wound.
Uncontrolled hyperlipidemia (defined as persistent elevation of total cholesterol or triglycerides >6.5nM/L) or a history of clinical atherosclerotic disease including heart attack, angina, peripheral vascular disease or stroke.
Previous organ transplantation
Inability to give informed consent
Inability to perform pulmonary function or 6 minute walk tests and imaging assessments
Other protocol-defined inclusion/exclusion criteria may apply