Lung Disease / Toshibia Aquilion One CT / Pneumothorax · Observational Study

Study of the Disease Process of Lymphangioleiomyomatosis

ClinicalTrials.gov · August 20, 2026

Preliminary Why this matters to you Expert discussion

Early or partial results. Treat as a signal, not a conclusion.

AI Summary

This is an active observational cohort study registered to characterize the clinical course and molecular pathogenesis of lymphangioleiomyomatosis (LAM) in approximately 2000 participants. No results are reported in this registry record; the study remains ongoing and recruitment is active.

What was studied

Observational. Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis; age from 16 Years; to 100 Years. Intervention: LAM. n = 2,000. 1 site: United States.

Key findings

Safety

Safety was not reported in the material analysed. Check the source before drawing any conclusion about harm.

Who this may help

The source did not state who this applies to in practice.

Plain-language summary

This is an ongoing observational registry study with no reported results; it aims to characterize disease biology and natural history of LAM but cannot yet support clinical inference.

Study design

As stated by the source record.

MethodsCohort model. Prospective time perspective. Primary outcome measure: Define the clinical course of the disease LAM andelucidate the pathogenesis of LAM at cellular and molecular levels (assessed at on going)

DesignObservational

PopulationLung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis; age from 16 Years; to 100 Years

Sample size2,000

InterventionLAM

Setting1 site: United States

Reported figures

Quoted from the source exactly as published.

Planned enrollment2000

Primary outcome measuresDefine the clinical course of the disease LAM and elucidate the pathogenesis of LAM at cellular and molecular levels

Study typeObservational

Caveats

No results are posted in this registry record; study is ongoing and recruiting.
As an observational study without a control arm, it cannot establish causation or measure treatment efficacy.
Specific eligibility criteria, enrollment progress, and outcome metrics are not detailed in this registry summary.
No effect sizes, confidence intervals, or p-values are available; cannot assess magnitude of any associations.

Evidence assessment

Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.

Study design

moderate

Observational design; no control group or intervention arm.

Directness

moderate

Directly studies LAM disease course and pathogenesis in affected individuals.

Consistency

insufficient

Single ongoing study; no comparative data or replication reported.

Risk of bias

insufficient

Registry record does not describe bias assessment or mitigation methods.

Sample adequacy

moderate

Planned enrollment 2000 participants; adequacy depends on outcome and effect detection.

What is missing. This record has no key findings. That is a gap in the analysis, not a judgement about the study.

Source abstract

Registry record from ClinicalTrials.gov (NCT00001465). This is a study registration, not published results. Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI). Recruitment status: RECRUITING. Study type: OBSERVATIONAL. Enrollment: 2000 participants (ESTIMATED). Conditions: Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis. Interventions: DEVICE: Toshibia Aquilion One CT. Primary outcome measures: Define the clinical course of the disease LAM andelucidate the pathogenesis of LAM at cellular and molecular levels , on going. Brief summary: Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor. This study is designed to determine the disease processes involved at the level of cells and molecules, in order to develop more effective therapy. Researchers intend to identify the proteins and genes that contribute to the process of lung destruction in affected individuals.

Follow from this

Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.

Lung DiseaseToshibia Aquilion One CTPneumothoraxTuberous SclerosisNCT00001465National Heart, Lung, and Blood Institute (NHLBI)