Clinical Trial: Safety of Simvastatin in LAM and TSC

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional



Official Title: The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)

Brief Summary:

The purpose of this research study is to see if simvastatin can be taken safely in patients with either LAM or TSC, who are already being treated with everolimus or sirolimus. This is the first step in looking at simvastatin as a drug that may help patients, by impacting the growth and survival of cells that make up the lung lesions that cause problems in LAM and TSC patients. The study also seeks to learn more about how simvastatin works, when given to patients being treated with everolimus or sirolimus, and to evaluate the safety and any potential benefit to patients taking this 2-drug combination.

The primary objective of this study is to determine the safety of simvastatin in the treatment of LAM-S or LAM-TS in patients on a stable (for at least 3 months) dose of sirolimus or everolimus.

Secondary objectives include:

  • To assess the effect of simvastatin on forced expiratory volume in 1 second (FEV1).
  • To assess the effect of simvastatin on forced vital capacity (FVC).
  • To assess the effect of simvastatin on diffusing lung capacity (DLCO).
  • To assess the effect of simvastatin on vascular endothelial growth factor -D (VEGF-D) serum levels.
  • To assess the effect of simvastatin with questionnaire- based assessments of dyspnea, fatigue, and quality of life (QOL).
  • Assess signs of clinical benefit.