Clinical Trial: Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

Brief Summary: This is a study to determine if early, long-term low dose sirolimus is effective for preventing progression to more advanced stages.

Detailed Summary: The primary objective of the MILED trial is to determine if early, long term (2 yr), low dose (fixed at 1 mg/day) treatment of patients with well-preserved lung function will prevent disease progression to more advanced stages. Sixty patients with FEV1>70% predicted will be enrolled and randomized to receive 1 mg/day sirolimus or placebo, and followed for a period of 2 years with pulmonary function testing every 4 months. The primary endpoint will be the between-group (placebo vs. sirolimus) difference in the rate of change in FEV1 (in liters) over two years. Secondary endpoints will include severity grade adverse events, time to 200cc or 10% FEV1 decline, forced vital capacity, lung volumes, diffusing capacity, serum VEGF-D, and early airflow obstruction assessed using hyper-polarized gas MRI. The study will be conducted through the Rare Lung Disease Clinic Network, a confederacy of clinics organized by the LAM Foundation that is currently following over 1300 U.S. LAM patients and conducting the Department of Defense sponsored Trial of an Aromatase Inhibitor in LAM (TRAIL) trial. The LAM Foundation will assist with study recruitment and dissemination of results, and the University of South Florida will function as the Data Coordinating Center. Successful completion of this study will define the safety and efficacy of low dose sirolimus in patients with normal lung function, and determine if sirolimus can be used to prevent disease progression to symptomatic stages.
Sponsor: University of Cincinnati

Current Primary Outcome: Forced Expiratory Volume in 1 Second (FEV1 slope) [ Time Frame: 2 years ]

Rate of lung function decline


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Diffusing Capacity for Carbon Monoxide (DLCO) [ Time Frame: 2 years ]
    Rate of decline in diffusing capacity
  • Total Lung Capacity (TLC) [ Time Frame: 2 years ]
    Rate of change in total lung capacity


Original Secondary Outcome: Same as current

Information By: University of Cincinnati

Dates:
Date Received: May 10, 2017
Date Started: June 6, 2017
Date Completion: June 6, 2021
Last Updated: May 10, 2017
Last Verified: May 2017