A Phase 2 interventional study of Sirolimus 2mg in Lymphangioleiomyomatosis, sponsored by National Heart, Lung, and Blood Institute (NHLBI). Completed at 1 site in United States. Open to female participants aged 18 Years to 90 Years. Per ClinicalTrials.gov, last updated 2026-02-18.
Sponsored by National Heart, Lung, and Blood Institute (NHLBI) · Phase 2, Interventional, and Other
Among 28 studies in Lymphangioleiomyomatosis with a site in United States — 6 open to participants now, 8 under way, 1 of them phase 3. Lymphangioleiomyomatosis · United States.
Among the 28 studies in Lymphangioleiomyomatosis with a site in United States, this one is the 7th largest by enrollment of the 21 interventional ones, is the only one that started in 2017, is one of 8 in phase 2, and was the 23rd registered.
49 studies on the registry are indexed under Lymphangioleiomyomatosis; 11 are open to participants now.
This study's enrollment of 33 is above the median of 25 across 30 interventional studies indexed under Lymphangioleiomyomatosis.
Browse Lymphangioleiomyomatosis studies →6 of the 28 studies in Lymphangioleiomyomatosis with a site in United States are open now, in 2 countries. Each count lists the studies.
Of the 10 Lymphangioleiomyomatosis studies recruiting now, 6 (60%) set no upper age limit and 1 (10%) stop at 75 or younger; 4 take children under 18; 1 take healthy volunteers.
This study: 18 Years to 90 Years · female · no healthy volunteers.
The full table on the hub →28 studies; the count open now beside each. This study's row is marked.
First in 1995; this study’s year is marked. 1 started in 2017.
Median enrollment 24 across the 21 interventional studies, 400 across the 7 observational. Of the 20 that completed or were terminated, 12 (60%) have results posted.
2 of 28 have an industry lead sponsor. The 10 most frequent:
National Heart, Lung, and Blood Institute (NHLBI) is the lead sponsor of 1,143 studies on the registry; 71 are open to participants now.
Of its 58 completed or terminated interventional studies of FDA-regulated products, 51 (88%) have results posted.
National Heart, Lung, and Blood Institute (NHLBI) has 8 studies filed under Lymphangioleiomyomatosis — fewer than ten, so there is no comparison to draw.
2 of the studies in Lymphangioleiomyomatosis with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06889168Columbia UniversityEvaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM) | 2026-12 |
| NCT01799538National Heart, Lung, and Blood Institute (NHLBI)Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis | 2027-11-01 |
Among the 28 studies in Lymphangioleiomyomatosis with a site in United States, from their registry updates:
Background:
Lymphangioleiomyomatosis (LAM) is a rare, progressive disease. It usually affects women in the prime of their lives. It typically results in lung destruction. Studies have shown that a drug called sirolimus stabilizes lung function in people with LAM. But researchers do not know what drug dose and blood serum levels are needed to reach this stability. Researchers want to learn more about the right dose of sirolimus for people with LAM.
Objective:
To determine if blood and urine markers after 1 dose and again after 9 months can be used to evaluate the correct dose of sirolimus for people with LAM.
Eligibility:
Women ages 18-90 with LAM whose doctors have decided they should start taking sirolimus to treat it.
Design:
At visit 1, participants will take their first dose of sirolimus by mouth at the clinic. They will have blood and urine collected.
Participants will take 1 tablet of the study drug each day.
Visit 2 will be 3 months after visit 1. Participants will have blood and urine collected.
Visit 3 will be 9 months after visit 1. Participants will have blood and urine collected.
Participant samples will be stored in a secure place. No personal data will be connected to them.
Sirolimus (rapamycin), which acts as a targeted inhibitor of the protein mechanistic target of rapamycin (mTOR), has been shown to be effective in patients with lymphangioleiomyomatosis (LAM). It stabilizes lung function, resolves chylous effusions and lymphangioleiomas and shrinks angiomyolipomas. The current study is to understand better the short-term action of the drug by following the effects on potential biomarkers in blood and urine. Patients with LAM will have samples taken prior to administration of first dose of the drug, at 1 hr and then at 23 hours after the drug (trough level). At 3 and 9 months, samples will be obtained at trough and 1 hour after the dose. Molecular and cellular analyses will be performed to look for potential biomarkers.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
EXCLUSION CRITERIA
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants diagnosed with Lymphangioleiomyomatosis will be administered Sirolimus 2 mg by mouth daily to be prescribed by treating physician.
Drug: Sirolimus 2mg
Patients with LAM, whose treating physicians have decided that they need to start treatment with sirolimus will be referred to the NIH Clinical Center for these studies.
Also known as: Rapamune
Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants
Plasma from Lymphangioleiomyomatosis (LAM) participants was profiled using the aptamer-based Somascan 11k assay (Somalogic). The primary outcome is the number of plasma proteins that exhibit a statistically significant change from each participant's baseline following sirolimus administration, defined by pre-specified multiplicity-adjusted thresholds (e.g., FDR q\<0.05 with absolute log2 fold-change ≥0.5), representing candidate sirolimus-sensitive biomarkers.
Time frame: Day 0 (pre-treatment, 1 hour post-Sirolimus, 23 hours post-Sirolimus), Month 3 (23 hours post-Sirolimus, 1 hour post next dose), Month 9 (23 hours post-Sirolimus, 1 hour post next dose)
| Milestone | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| Started | 33 |
| Completed | 24 |
| Not completed | 9 |
| Withdrew: Physician decision | 5 |
| Withdrew: Adverse event | 1 |
| Withdrew: Non-compliance | 3 |
Plasma from Lymphangioleiomyomatosis (LAM) participants was profiled using the aptamer-based Somascan 11k assay (Somalogic). The primary outcome is the number of plasma proteins that exhibit a statistically significant change from each participant's baseline following sirolimus administration, defined by pre-specified multiplicity-adjusted thresholds (e.g., FDR q\<0.05 with absolute log2 fold-change ≥0.5), representing candidate sirolimus-sensitive biomarkers.
| plasma proteins | Participant With LAM Receiving Sirolimus |
|---|---|
| Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants | 662 |
Collected over Up to 9 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 0/32 (0%) | 0/32 (0%) | 1/32 (3.1%) |
| Event | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| PneumothoraxRespiratory, thoracic and mediastinal disorders | 1/32 |
| Age, Categorical(Participants) | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 33 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| Female | 33 |
| Male | 0 |
| Ethnicity (NIH/OMB)(Participants) | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 32 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 3 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 25 |
| More than one race | 1 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(participants) | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus |
|---|---|
| United States | 33 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — All IPD that underlie results in a publication.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form
Time frame: Data will be made available as soon as possible or at the time of associated publication, whichever comes first, according to Institute policy.
Access criteria: BioData Catalyst is supported by NHLBI and access to data is controlled by the NHLBI Data Access Committee (DAC) utilizing the database (dbGaP)
Ran from Dec 2017 to primary completion in Mar 2025, 87 months.
Results were first posted in Feb 2026, 11 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Sirolimus
National Heart, Lung, and Blood Institute (NHLBI) →