Lymphangioleiomyomatosis Clinical Trial
Official title:
The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)
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.
After providing written informed consent, study related tests/procedures will be done to ensure eligibility for the study. If found to be eligible, the participant will be given simvastatin at a starting dose of 20 mg, to be taken each evening by mouth. If after 2 months the simvastatin 20 mg dose is tolerated, the dose of simvastatin will be increased to 40 mg each evening by mouth. Doses may be adjusted as needed, should the participant experience side effects from simvastatin. The participant's dose of everolimus or sirolimus is not expected to change, as this is a dose that has been previously tolerated. If side effects occur as a result of the combination of drugs, the dosages may be adjusted by the study physician (investigator). ;
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