Table 1.

Interventional Clinical Trials^*

Trial Number	Title	Purpose	Intervention	Status	Duration	Primary Outcome Measures	Location
NCT01799538	Trial of Aromatase Inhibition in Lymphangioleiomyomatosis (TRAIL)	To determine whether estrogen suppression by an aromatase inhibitor in postmenopausal women with LAM will prevent or delay progression of lung disease	Letrozole: 2.5 mg daily dose	Unknown	12 Months	The effect on Forced Expiratory Volume in one second (FEV1)	University of Cincinnati, OH, USA
NCT01059318	A Study to Determine the Effectiveness of Escalating Doses of RAD001 (Everolimus) in Patients with Lymphangioleiomyomatosis	The safety of escalating doses of RAD001 (everolimus) in patients with LAM	Escalating daily doses of Everolimus (RAD001): 2.5mg, 5mg, 10mg	Completed	26 Weeks	Assess safety, pharmacokinetics of everolimus and pharmacodynamics of serum VEGF-D	Novartis Pharmaceuticals
NCT00790400	Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis (SAIL)	The safety and tolerability of the combination of sirolimus and hydroxychloroquine	Combination of sirolimus and escalating daily doses of hydroxychloroquine: 200 mg or 400 mg	Recruiting	6 Months	Safety of combinational treatment of everolimus and hydroxychloroquine	Brigham and Women's Hospital, Boston, MA, USA
NCT01687179	Safety of Simvastatin in LAM and TSC (SOS)	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	Combination of sirolimus or everolimus with escalating daily doses of simvastatin: 20 mg and 40 mg	Recruiting	4 Months	Safety of simvastatin in the treatment of LAM-S and LAM-TS patients on a stable dose of sirolimus or everolimus	University of Pennsylvania, Philadelphia, USA
NCT01353209	Doxycycline In Lymphangioleiomyomatosis (LAM)	Effect of doxycycline on the progression of LAM	Doxycycline: 50mg	Unknown	24 Months	Mean rate of change of FEV1	University of Nottigham, UK
NCT00414648	The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)	Tolerabilty and safety of Saracatinib in patients with LAM	Escalating daily doses of Saracatinib: 50 mg, 125 mg, and 175 mg	Recruiting	4 Weeks treatment + 8 Weeks follow up	Dose Determination	Baylor College of Medicine, Houston, TX, USA
NCT00989742	Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)	The safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with TSC or S-LAM	Everolimus (RAD001): 10mg daily dose	Active, not recruiting (has results)	12 Months	Angiomyolipoma Response Rate	Novartis Pharmaceuticals
NCT02061397	RAD001 Therapy of Angiomyolipoma in Patients With TS Complex and Sporadic LAM	Reduction of Angiomyolipoma volume in patients with TSC and LAM	Everolimus (RAD001), 5 and 10 mg/day or 30, 50, 70mg/week	Completed	24 Months	Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline	Children's Hospital Medical Center, Cincinnati, OH, USA
NCT02009241	Pulmonary Rehabilitation in Lymphangioleiomyomatosis	Determine the effect of pulmonary rehabilitation on exercise capacity, dyspnea, quality of life, muscle force, functional limitation and dynamic hyperiflation	Pulmonary Rehabilitation program consisting of 30 minutes of treadmill aerobic exercise training and 30 minutes of muscle strength training	Recruiting	12 Weeks	Endurance time during constant work rate cycle ergometry	University of San Paulo, Brasil
NCT02116712	Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis	Effect of Albuterol given in a metered dose inhaler or with a nebulizer will be compared lung function	Albuterol inhaler (two or four puffs) or Albuterol nebulizer	Recruiting	3 Days	Improvement in lung function	National Heart, Lung, and Blood Institute (NHLBI), USA

^*For more details, see http://clinicaltrials.gov/