Condition (MeSH)
Lymphangioleiomyomatosis
ClinicalTrials.gov lists 48 trials for Lymphangioleiomyomatosis or a narrower condition beneath it. National Heart, Lung, and Blood Institute (NHLBI) has the most among industry and government lead sponsors, with 8.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
48 of the 48 trials are tagged with Lymphangioleiomyomatosis itself; the rest are tagged with a narrower condition beneath it.
When did Lymphangioleiomyomatosis trials start? (25)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2025 | 2 |
| 2024 | 1 |
| 2023 | 2 |
| 2022 | 1 |
| 2021 | 3 |
| 2020 | 1 |
| 2019 | 3 |
| 2018 | 5 |
| 2017 | 2 |
| 2016 | 5 |
Showing 10 of 25 earlier years.
No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Lymphangioleiomyomatosis trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 6 |
| Phase 1/2 | 5 |
| Phase 2 | 10 |
| Phase 3 | 3 |
| Phase 4 | 2 |
| Not applicable | 3 |
| Observational | 19 |
How many Lymphangioleiomyomatosis trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 10 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 2 |
| Completed | 28 |
| Terminated | 2 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Lymphangioleiomyomatosis trials? (3)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | 8 |
| Novartis Pharmaceuticals | 3 |
| CENTOGENE GmbH Rostock | 1 |
Which drugs are being tested for Lymphangioleiomyomatosis? (3)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Sirolimus | 6 |
| EVEROLIMUS | 5 |
| Imatinib | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| L01XE31 - Nintedanib | 1 |
| Letrozol (Femara, Novartis) | 1 |
| Rapamycin, sirolimus | 1 |
| albuterol nebulizer | 1 |
| Sirolimus Oral Product | 1 |
Showing 5 of 12 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Lymphangioleiomyomatosis trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pulmonary | 15 | 1 |
| Pain | 8 | 1 |
| Epilepsy | 6 | 1 |
| Pneumonia | 6 | 3 |
| Infection | 5 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Lymphangioleiomyomatosis? (77)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Nintedanib for patients with lymphangioleiomyomatosis: a phase 2, open-label, single-arm study. | The Lancet. Respiratory medicine | 2024 | Yes |
| Safety and Efficacy of Combined Resveratrol and Sirolimus in Lymphangioleiomyomatosis. | Chest | 2023 | Yes |
| Determinants of Progression and Mortality in Lymphangioleiomyomatosis. | Chest | 2023 | Yes |
| Novel Pulmonary Cyst Characteristics Associated with the Diffusing Capacity of the Lung in Lymphangioleiomyomatosis: A Cross-Sectional Clinical Trial. | Annals of the American Thoracic Society | 2023 | Yes |
| Ultra-Small Lung Cysts Impair Diffusion Without Obstructing Air Flow in Lymphangioleiomyomatosis. | Chest | 2021 | Yes |
Showing 5 of 77 publications.
Prevalence (26)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Lymphangioleiomyomatosis | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Lymphangioleiomyomatosis | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Lymphangioleiomyomatosis | Annual incidence | <1 / 1 000 000 | United States | US | Exact |
| Lymphangioleiomyomatosis | Point prevalence | 1-9 / 1 000 000 | United States | US | Exact |
| Lymphangioleiomyomatosis | Annual incidence | <1 / 1 000 000 | United States | US | Exact |
Showing 5 of 26 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Lymphangioleiomyomatosis | 538 | Exact | Orphadata Science knowledge files, Product 1 |
| Lymphangioleiomyomatosis | 538 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.