Safety of Simvastatin in LAM and TSC — SOS  

Tuberous Sclerosis Complex Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial Description

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). ;

Related Conditions & MeSH terms

• Lymphangioleiomyomatosis
• Sclerosis
• Tuberous Sclerosis
• Tuberous Sclerosis Complex

• NCT number: NCT02061397
• Study type: Interventional
• Source: University of Pennsylvania
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: March 2014
• Completion date: December 13, 2019