A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis

Completed · Phase 2

Conditions studied: Lymphangioleiomyomatosis

In brief

This was an exploratory study to determine whether escalating doses of RAD001 (everolimus) were safe and effective in patients with Lymphangioleiomyomatosis

Key facts

Study ID

NCT01059318

Run by

Novartis Pharmaceuticals

People needed

24

Starts

2010-01-01

Expected to finish

2012-06-01

Last updated by the study team

2020-11-19

Who can join

Age: 18 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Center for LAM Research and Clinical Care — Boston, Massachusetts, United States
• University of Cincinnati, Department of Internal Medicine, Pulmonary, Critical Care & Sleep Medicine, — Cincinnati, Ohio, United States
• Novartis Investigative Site — Lyon, France
• Novartis Investigative Site — Milan, Italy

Full record on ClinicalTrials.gov

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