Phase 2
N=10
Safety of Simvastatin in LAM and TSC
Lymphangioleiomyomatosis · Tuberous Sclerosis Complex
Bottom Line
View on ClinicalTrials.gov: NCT02061397 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Aug 2020
Primary outcome: Primary: Safety of Simvastatin in the Treatment of LAM-S and LAM-TS Patients — 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Simvastatin (Drug); Sirolimus Oral Product (Drug); Everolimus Oral Product (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Female
- Sponsor
- University of Pennsylvania
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety of Simvastatin in the Treatment of LAM-S and LAM-TS Patients |
— | — |
| SECONDARY Percent Predicted FEV1 |
-2.9 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Female, age 18 and older with clinically definitive diagnosis (biopsy proven or compatible chest CT/MRI scan) of sporadic LAM (LAM-S) or LAM associated with TS (LAM-TS).
- Treated with a stable (at least 3 months) dose of sirolimus or everolimus
- Negative pregnancy test (women of child bearing potential) at screening.
- Women of childbearing potential must be using barrier, medically acceptable contraceptive precautions.
- Signed and dated informed consent.
Exclusion Criteria
- Age 2 times upper limit of normal-ULN), kidney (serum creatinine > 1.5 times ULN), or blood (leucopenia (ANC<2000), anemia, Hgb < 11 gm/dl).
- History of inflammatory muscle disease or myopathy.
- Bleeding diathesis or anticoagulant therapy.
- Uncontrolled hyperlipidemia or diabetes.
- Pregnant, breast feeding, or plan to become pregnant within the next 6 months
- Inadequate contraception (must agree to barrier method)
- History of organ transplant.
- Active on transplant list.
- Severe or uncontrolled medical conditions which would cause an unacceptable safety risk or compromise compliance with the protocol.
- Unstable seizures (recent changes in pattern or anti-epileptics).
- Mental illness or cognitive deficit precluding informed consent..
- Inability to attend scheduled clinic visits or comply with study procedures.
Data sourced from ClinicalTrials.gov (NCT02061397). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.