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Lung Disease | Study of the Disease Process of Lymphangioleiomyomatosis

Lung Disease research study

What is the primary objective of this study?

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.

Who is eligible to participate?

- INCLUSION CRITERIA: General admission criteria for patients include one or both of the following: Findings on lung biopsy diagnostic of LAM; Findings on chest x-ray and/or chest computed axial tomography consistent with LAM. Patients with TSC and pulmonary LAM will be included in the study. Normal non-smokers in the control group are defined as individuals who have not smoked for greater than or equal to 1 year and have no systemic or pulmonary disease. Normal smokers defined as individuals with no systemic or pulmonary disease, who have smoked for greater than or equal to 1 year and have normal chest x-ray and normal pulmonary function tests may be included if needed as controls for a similar population of patients with LAM. Pregnant and or nursing women can be included in accordance with Federal Regulations at Subpart B of 45 CFR 46 Subjects who are pregnant and or nursing will be excluded from procedures during their pregnancy that are greater than minimal risk, until they are no longer pregnant and/or nursing. Procedures that will not be completed while the subject is pregnant and/or nursing including: PFTs, Six Minute Walk Test, thoracentesis, bronchoscopy, and measurements with imaging modalities requiring contrast or with radiation exposure such as Chest x-ray, CT scan, MRI, bone densitometry (DEXA). Allowing subjects to be included in the study may glean important information about individuals with uncommon pulmonary disease during and post pregnancy. EXCLUSION CRITERIA: Exclusion criteria for patients include: Age less than 16. Advanced stage of a pulmonary or a systemic illness in which the risk of the study is judged to be significant even in the absence of a clear contraindication to the procedures. Exclusion criteria for patients for the formal exercise study and the stress echocardiogram include patients on continuous oxygen. Patients may perform an exercise test that will assess the patient's exercise capacity with activities of daily living.

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Lung Disease

Pneumothorax

Tuberous Sclerosis

Lymphangioleiomyomatosis

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Device:Toshibia Aquilion One CTThe Toshiba Aquilion ONE CT system is currently being used for studies in both general CT radiology and CT cardiac imaging. One of the unique aspects of the Aquilion ONE CT system is its ability to acquire whole organ volume images in a single rotation by utilizing an x-ray detector that is configured as 320 detector rows with a 0.5 mm width, providing a z-axis coverage of 16 cm of anatomy. In line with the evolutionary changes to CT systems, the Aquilion ONE will be upgraded with new technology that will expand its capabilities. The changes being made to the Aquilion ONE will provide enhancements to image acquisition capabilities, reduce ionizing radiation dose, and improve subject access to the system. All of these features assist in enhancing the safety of the currently installed Aquilion ONE CT system.

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

LAM patientsThe study population will include unlimited number of individuals with definitive or probable diagnosis of LAM. This study enrolled normal volunteers during the years 1995 to 1999. The patients with tissue diagnosis of LAM may be admitted for evaluation every six months, or as deemed necessary for research. If a patient is admitted more frequently than twice a year, the visit will not include procedures involving radiation unless medically-indicated. If tests are done for medical indications, documentation of the medical reason will be made in the patient s medical record. In these cases, the tests may also be used for research purposes as defined by this protocol.

Study Status

Recruiting

Start Date: December 18, 1995

Completed Date: September 30, 2030

Phase: N/A

Type: Interventional

Design:

Primary Outcome: Define the clinical course of the disease LAM and elucidate the pathogenesis of LAM at cellular and molecular levels

Secondary Outcome:

Study sponsors, principal investigator, and references

Principal Investigator: Joel Moss, M.D.

Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Collaborator:

More information:https://clinicaltrials.gov/show/NCT00001465

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