RecruitingNCT00001465
Respiratory
Lymphangioleiomyomatosis
Also known as LAM, TSC-LAM, sporadic LAM
Lymphangioleiomyomatosis is a rare, slowly progressive cystic lung disease caused by mutations in the TSC1 or TSC2 genes, leading to abnormal smooth muscle-like cell proliferation in the lungs. It almost exclusively affects women and may oc
ORPHA:538 ↗Gene TSC2Gene TSC1Prevalence Approximately 3–5 per million womenOnset Adult (typically women of childbearing age)
10
studies recruiting now
as of 7 Sept 2026
51
studies registered in total
as of 7 Sept 2026
2
countries with a recruiting site
as of 7 Sept 2026
21 Mar 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 1 / Phase 2NCT01799538
Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis
RecruitingNCT05676099
TSC Biosample Repository and Natural History Database
RecruitingNCT06160310
Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)
RecruitingPhase 1NCT06889168
Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 10 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: LAM Foundation Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Lymphangioleiomyomatosis
Lymphangioleiomyomatosis is a rare, slowly progressive cystic lung disease caused by mutations in the TSC1 or TSC2 genes, leading to abnormal smooth muscle-like cell proliferation in the lungs. It almost exclusively affects women and may occur sporadically or in association with tuberous sclerosis complex. Over time, the cystic destruction of lung tissue results in declining lung function and, in some cases, respiratory failure.
Common clinical features
Progressive dyspnoea on exertionRecurrent spontaneous pneumothoraxChylothorax (chylous pleural effusion)Chronic coughHaemoptysisAbdominal angiomyolipomasFatigue and reduced exercise tolerance
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment and 8 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Sirolimus (Fyarro)
Phase 3Everolimus (Afinitor)
Phase 2Letrozole (Femara)
Phase 2Nintedanib (Nintedanib component of ofev)
Phase 2Saracatinib
Phase 2Celecoxib (Celebra)
Phase 2Loratadine (Alavert)
Phase 1/2Imatinib Mesylate (Gleevec)
Phase 1/2Albuterol (Aerolin)
Before you apply
Things trial teams commonly ask about for Lymphangioleiomyomatosis. Not eligibility rules; those are set by each study.
- Trials often require confirmed diagnosis via HRCT showing bilateral pulmonary cysts plus serum VEGF-D elevation or TSC mutation; ensure documentation is up to date.
- Many studies exclude women who are pregnant or breastfeeding; clarify reproductive status before screening.
- Baseline pulmonary function tests (FEV1, DLCO) are key eligibility metrics; obtain recent spirometry within study-specified windows.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).