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.

Condition
Lung Disease Pneumothorax Tuberous Sclerosis

Further Study Details: 

Expected Total Enrollment:  999

Individuals with pulmonary lymphangioleiomyomatosis develop severe destructive lung disease. Most of them are females of childbearing age. Currently, there is no proven effective therapy and the prognosis is variable. This study is designed to (a) define the clinical course of the disease and (b) elucidate the pathogenesis of the disease at the cellular and molecular levels, in order to develop more effective therapy. To accomplish this, we intend to identify the proteins and genes that contribute to the process of lung destruction in affected individuals.

Genders Eligible for Study:  Both

Criteria

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.
EXCLUSION CRITERIA:
Exclusion criteria for patients and normal volunteers include:
Age less than 18.
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 research volunteers for PET scan include:
Age less than 18 or greater than 80.
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.
LAM occurs almost exclusively in females of child-bearing age: thus, the normal volunteers will also be female.
Patients and normal volunteers of child-bearing age (defined as less than 56 years) who participate in the PET study must not be pregnant nor breast feeding. Appropriate birth control methods must be reported, and a negative pregnancy test must be obtained within 24 hours of the PET scan.

Maryland
      National Heart, Lung and Blood Institute (NHLBI), 9000 Rockville Pike,  Bethesda,  Maryland,  20892,  United States; Recruiting

Study ID Numbers:  950186; 95-H-0186
Record last reviewed:  August 24, 2004
Last Updated:  March 1, 2005
Record first received:  November 3, 1999
ClinicalTrials.gov Identifier:  NCT00001465
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08