A Study to Determine the Effectiveness of Escalating Doses of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Read our disclaimer for details.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study:
18 Years and older (Adult, Senior)
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