Plain-English translation of NCT00001465 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This is a research study designed to help doctors better understand lymphangioleiomyomatosis, a serious lung disease that mostly affects women. Researchers will follow patients over time, collecting medical information and samples to identify the genes and proteins that cause lung damage in this disease. The goal is to develop more effective treatments for patients who don't respond well to current medications like sirolimus.
Currently, sirolimus is the main treatment available, but it doesn't work for everyone and the disease progresses differently in different people. Researchers need to understand the underlying biological causes of the disease so they can create better, more personalized treatments.
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You would visit the study team every six months, or more often if medically necessary. During visits, researchers will collect information about your health, perform lung tests, and may take blood or tissue samples to study the genes and proteins involved in your disease. Some visits may include exercise tests or heart imaging, depending on your individual health status. This is a long-term study designed to track how your disease progresses over time.
AI-generated summary from trial data · Aug 22, 2026 · Not medical advice
United States
Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Enrollment target
~2,000 participants
Started
December 1995
Age range
16 Years – 100 Years
Last updated on clinicaltrials.gov in September 2026.
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Central contact
Tatyana A Worthy, R.N.
National Heart, Lung, and Blood Institute (NHLBI)
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.