Clinical Trial: A Study to Determine the Effectiveness of Escalating Doses of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Exploratory, Open Label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangio
Brief Summary: This is an exploratory study. The purpose of the study is to determine whether escalating doses of RAD001 (everolimus) is safe and effective in patients with Lymphangioleiomyomatosis
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Assess safety, pharmacokinetics (PK) and pharmacodynamics (PD), Measure: Safety (vital signs, ECGs, safety labs, AEs), PK (concentration of everolimus), PD (serum VEGF-D) [ Time Frame: 26 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Clinical efficacy of everolimus Measure: improvement in pulmonary function (FVC) [ Time Frame: 26 weeks ]
- Clinical efficacy of everolimus Measure: improvement in other measures of pulmonary function (FEV-1, lung volumes (TLC, TGV, RV), DLCO) [ Time Frame: 26 weeks ]
- Clinical efficacy of everolimus Measure: improvement in exercise capacity (6-minute walk test), and change in O2 saturation (with supplemental oxygen unchanged) [ Time Frame: 26 weeks ]
Original Secondary Outcome: Same as current
Information By: Novartis
Dates:
Date Received: January 28, 2010
Date Started: December 2009
Date Completion:
Last Updated: October 22, 2012
Last Verified: October 2012
