Conditions: Lymphangioleiomyomatosis
Keywords: Lymphangioleiomyomatosis, High Resolution CT scan, chest x-ray, 6 minute walk test, pulse oximetry, renal MRI, Pikometer
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 24
Healthy volunteers: no

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

Detailed description:
In addition to the data collected in this study, historical data from 43 patients treated with placebo from the multicenter trial of sirolimus in LAM (MILES) study (NCT00414648) were down weighted to an effective sample size of 18 for comparison of FEV1 and FVC endpoints. Reference to the publication of the MILES study has been provided under "Result Publication".

Arms:
arm 1: All patients received a starting dose of everolimus 2.5mg/day for 4 weeks, followed by a dose of 5 mg/day for 4 weeks and finally a dose of 10mg/day for 18 weeks.

The 26 week treatment period was followed by an optional extension period wherein patients continued therapy until the last patient had completed 26-weeks of treatment. The longest period a patient participated in the study was 62 weeks.

Interventions: intervention 1: Everolimus
Intervention descriptions: intervention 1: Everolimus was formulated as tablets in strengths of 2.5mg, 5mg and 10mg.

Protocol document: