At a glance
ClinicalIndex Comparison Record- ✓Age 18 or older
- ✓Diagnosis of LAM confirmed by compatible chest CT plus biopsy/cytology consistent with LAM, or presence of tuberous sclerosis, angiomyolipoma or chylous pleural effusion, or VEGF-D ≥800 pg/ml
- ✓FEV1 ≤70% of predicted value after bronchodilator
- ✓Able to attend scheduled study visits and perform pulmonary function tests
- ✕Known allergy to sirolimus
- ✕History of heart attack, angina, or stroke due to clogging, narrowing, and hardening of the arteries and blood vessels
- ✕Significant hematologic or hepatic abnormality: transaminases >3× upper limit of normal, hematocrit <30%, platelets <80,000/mm³, absolute neutrophil count <1,000/mm³, or WBC <3,000/mm³
- ✕Intercurrent infection at time of treatment initiation
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus Trial
In Brief
A Phase 3 clinical trial evaluating Sirolimus and Placebo for Lymphangioleiomyomatosis. Completed, enrolled 89 participants across 13 sites in 3 countries.
Detailed Summary
Lymphangioleiomyomatosis (LAM) is a rare lung disease of women that is caused by genetic mutations. It results in the uncontrolled growth of an unusual type of smooth muscle cell in the lung. These cells invade lung tissue, including the airways, blood vessels, and lymph vessels, and restrict the flow of air, blood, and lymph, respectively. Respiratory failure, lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the disease. This study will evaluate the safety and effectiveness of sirolimus, an inhibitor of the mTOR pathway, in stabilizing or improving lung function in people with LAM.
Study Details
Timeline
Interventions
A sirolimus dose of 2 tablets (1 mg/tablet) per day for 1 year.
A placebo dose of 2 tablets per day for 1 year.