CompletedPhase 3

Efficacy and Safety of Sirolimus in LAM

United States, Canada, Japan89 participantsStarted 2006-12

Plain-language 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.

Who can participate

Age range

18 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age 18 or older * Signed and dated informed consent * Diagnosis of LAM based on compatible chest CT scan and a) biopsy or cytology consistent with LAM, or b) presence of tuberous sclerosis, angiomyolipoma or chylous pleural effusion; or c) a VEGF-D level of at least 800 pg/ml * Forced expiratory volume in one second (FEV1) of 70% or less of predicted value after administration of a bronchodilator Exclusion Criteria: * 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 (transaminase levels greater than three times the upper limit of normal, HCT less than 30%, platelets less than 80,000/cubic mm, adjusted absolute neutrophil count less than 1,000/cubic mm, total white blood cell count less than 3,000/cubic mm) * Intercurrent infection at the time treatment with sirolimus begins * Any surgery involving entry into a body cavity or requiring three or more sutures within 8 weeks of initiation of study drug * Use of an investigational drug within the 30 days prior to random assignment * Uncontrolled hyperlipidemia * Previous lung transplant or currently on lung transplant list * Unable to attend scheduled study visits * Unable to perform pulmonary function tests * Creatinine levels greater than 2.5 mg/dl * Chylous ascites severe enough to affect diaphragmatic function * Pleural effusion severe enough to affect pulm…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month

Timeframe: Baseline to Month 12

Trial details

NCT IDNCT00414648

SponsorUniversity of Cincinnati

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2010-09

Results submitted2021-12-15

View on ClinicalTrials.gov More Lymphangioleiomyomatosis trials