Safety of Simvastatin in LAM and TSC (SOS)
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|ClinicalTrials.gov Identifier: NCT02061397|
Recruitment Status : Active, not recruiting
First Posted : February 12, 2014
Last Update Posted : February 6, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Lymphangioleiomyomatosis Tuberous Sclerosis Complex||Drug: Simvastatin||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)|
|Study Start Date :||March 2014|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
Experimental: single simvastatin treatment arm
Simvastatin 20 mg oral daily for 2 months; if tolerated, followed by simvastatin 40 mg oral daily for 2 months
Eligible patients will be assigned to receive 20 mg of simvastatin once daily for a period of two months. If tolerated, the dosage of simvastatin will be advanced to 40 mg once daily in months 3 and 4.
Other Name: trade name Zocor
- Safety of simvastatin in the treatment of LAM-S and LAM-TS patients on a stable (for at least 3 months) dose of sirolimus or everolimus. [ Time Frame: 3 months ]Safety (including any major changes or deterioration in patient health) will be measure by reductions from baseline in physical examination, pulmonary function tests and/or blood values (CBC, serum chemistry) and in the reporting of new or aggravated adverse events. A Data Safety Monitoring Board will review data, particularly significant and serious adverse events, for trends and recommendations regarding the safe treatment of patients during the study.
- FEV1, FVC, DLCO, VEGF-D, and QOL; signs of clinical benefit. [ Time Frame: 3 months ]Pulmonary measures (FEV1, FVC, DLCO), VEGF-D, and QOL; signs of clinical benefit.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02061397
|United States, Pennsylvania|
|University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Vera P Krymskaya, PhD, MBA||University of Pennsylvania|
|Study Director:||Maryl Kreider, MD, MSCE||University of Pennsylvania|
|Study Chair:||Frank McCormack, MD||University of Cincinnati|