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

• Status: Completed
• Trial end date: 2012-06-01
• Target enrollment: 0
• Participant gender: Female

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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