NCT01059318

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

An Exploratory, Open-label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: Female
• Age: 18 Years
• Healthy volunteers: Not accepted

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".

Conditions

• Lymphangioleiomyomatosis

Interventions

Type	Name	Description
DRUG	Everolimus	Everolimus was formulated as tablets in strengths of 2.5mg, 5mg and 10mg.

Timeline

• Start date: 2010-01-01
• Primary completion: 2012-06-01
• Completion: 2012-06-01
• First posted: 2010-01-29
• Last updated: 2020-11-19
• Results posted: 2020-11-19

Locations

4 sites across 3 countries: United States, France, Italy